[{"id":"accelerate-platform","name":"ACCELERATE","tldr":"ACCELERATE is a Brussels-based forum where children's cancer doctors, drug companies, the EMA and FDA and parents agree which new cancer drugs should be tested in children and how, so that medicines developed for adults are not left untested in childhood cancers.","route":"/institutions/accelerate-platform/","kind":"institution","logo":"/logos/accelerate-platform.png","avatar":"org","sub":"Brussels, BE","facets":{"kind":["Institution"],"cancers":["Neuroblastoma","Diffuse midline glioma, H3 K27-altered","Paediatric low-grade glioma","Acute lymphoblastic leukaemia"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Institution","tip":"The centres and universities that matter, mapped and ranked."},"cancers":[{"label":"Neuroblastoma","href":"/cancers/neuroblastoma/","tip":"Neuroblastoma is a childhood nerve-cell cancer where anti-GD2 antibodies and, recently, GD2 CAR-T have improved survival in high-risk disease."},{"label":"Diffuse midline glioma, H3 K27-altered","href":"/cancers/dipg-dmg/","tip":"Diffuse midline glioma grows through the brainstem and cannot be removed surgically. A single change in a histone protein (H3 K27M) rewires how the tumour reads its DNA. Radiotherapy was long the only help; in 2025 the first drug aimed at this tumour, dordaviprone (ONC201), was approved after durable shrinkage in some patients, and GD2 CAR-T cells have produced striking early responses."},{"label":"Paediatric low-grade glioma","href":"/cancers/paediatric-low-grade-glioma/","tip":"Paediatric low-grade gliomas are slow-growing brain tumours driven almost always by a single overactive signal, the MAPK pathway, most often through a BRAF gene change. Because the switch is known, pills that block it (dabrafenib with trametinib, and tovorafenib) now shrink tumours far more often than chemotherapy, and children are increasingly spared radiation to the developing brain."},{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."}],"tags":[]},"sortKeys":{"year":0}},{"id":"action-dmg","name":"ACTION","tldr":"ACTION is the first placebo-controlled phase 3 trial ever run in diffuse midline glioma, the childhood brain-stem tumour that radiotherapy alone has never cured. It asks whether taking dordaviprone after radiotherapy lengthens life.","route":"/trials/action-dmg/","status":"recruiting","kind":"trial","cancerIcon":"dipg-dmg","sub":"NCT05580562","facets":{"kind":["Trial"],"cancers":["Diffuse midline glioma, H3 K27-altered"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Trial","tip":"Landmark and current trials that define the standard of care."},"cancers":[{"label":"Diffuse midline glioma, H3 K27-altered","href":"/cancers/dipg-dmg/","tip":"Diffuse midline glioma grows through the brainstem and cannot be removed surgically. A single change in a histone protein (H3 K27M) rewires how the tumour reads its DNA. Radiotherapy was long the only help; in 2025 the first drug aimed at this tumour, dordaviprone (ONC201), was approved after durable shrinkage in some patients, and GD2 CAR-T cells have produced striking early responses."}],"tags":[{"label":"cns","href":"/tagged/cns/","tip":"Every record tagged cns."}]},"sortKeys":{"year":0}},{"id":"aml-paediatric","name":"Acute myeloid leukaemia in children","tldr":"Acute myeloid leukaemia in children carries gene fusions rather than the mutations of ageing, is treated with four or five intensive courses of chemotherapy, and cures around two thirds of children. Adding gemtuzumab ozogamicin lowered relapse in the AAML0531 trial, and the menin inhibitor revumenib is the first targeted drug approved for the KMT2A-rearranged form common in young children.","route":"/cancers/aml-paediatric/","kind":"cancer","cancerIcon":"aml-paediatric","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"subtype-page","href":"/tagged/subtype-page/","tip":"Every record tagged subtype-page."}]},"sortKeys":{"year":0}},{"id":"rejuv-ayac-distinct-group","name":"Adolescents and young adults: a group with its own cancers, its own gap and its own needs","tldr":"People diagnosed between 15 and 39 get different cancers from children and from older adults, and for years their survival improved more slowly than either. Since 2000 that has changed: five-year survival gains for this group have paralleled those of childhood cancers. The obstacles that remain are trial enrolment, access and insurance, and support that fits the age.","route":"/technologies/rejuv-ayac-distinct-group/","status":"established","kind":"technology","schematic":{"id":"rejuv-ayac-distinct-group","sections":["rejuvenation","supportive-care"]},"facets":{"kind":["Technology"],"cancers":["Hodgkin lymphoma","Testicular germ cell tumours","Thyroid cancer","Melanoma","Ewing sarcoma","Osteosarcoma","Acute lymphoblastic leukaemia","Childhood cancers"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Technology","tip":"Every way we see, measure, or attack a tumour, explained."},"cancers":[{"label":"Hodgkin lymphoma","href":"/cancers/hodgkin-lymphoma/","tip":"Hodgkin lymphoma is one of the most curable cancers, where the goal is now to cure with less toxicity, using brentuximab and, from 2026, first-line nivolumab."},{"label":"Testicular germ cell tumours","href":"/cancers/testicular/","tip":"Testicular germ cell tumours are the most curable adult solid cancer: cisplatin-based chemotherapy cures the large majority even when the disease has spread to distant sites. Today's research is about giving less treatment to the majority who are cured, rescuing the minority who relapse, and limiting lifelong survivorship harms."},{"label":"Thyroid cancer","href":"/cancers/thyroid/","tip":"Thyroid cancer is usually curable with surgery and radioactive iodine, the original theranostic. Rare aggressive forms respond to RET and BRAF inhibitors."},{"label":"Melanoma","href":"/cancers/melanoma/","tip":"The skin cancer that proved immunotherapy works: half of advanced patients now live 10 years. Also the first with an approved TIL therapy, an oncolytic virus, and a positive phase 3 personalised vaccine."}],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."}]},"sortKeys":{"year":0}},{"id":"alexs-lemonade-stand","name":"Alex's Lemonade Stand Foundation (ALSF)","tldr":"A childhood-cancer charity started by a four-year-old patient's lemonade stand that now funds hundreds of grants and runs an open-science data lab for paediatric cancer genomics.","route":"/collections/alexs-lemonade-stand/","kind":"collection","logo":"/logos/alexs-lemonade-stand.png","avatar":"org","sub":"ALSF","facets":{"kind":["Collection"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Collection","tip":"The open databases and registries the field runs on."},"cancers":[],"tags":[{"label":"patient-org","href":"/tagged/patient-org/","tip":"Every record tagged patient-org."}]},"sortKeys":{"year":0}},{"id":"amar-gajjar","name":"Amar Gajjar","tldr":"Led the St. Jude medulloblastoma trials that introduced molecular subgroups into risk-adapted treatment.","route":"/people/amar-gajjar/","kind":"person","round":true,"avatar":"person","sub":"Chair, Department of Pediatric Medicine, St. Jude Children's Research Hospital · St. Jude Children's Research Hospital","facets":{"kind":["Person"],"cancers":["Medulloblastoma"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[{"label":"Medulloblastoma","href":"/cancers/medulloblastoma/","tip":"Medulloblastoma is the most common malignant childhood brain tumour, arising in the cerebellum. Surgery, radiation to the whole brain and spine, and chemotherapy cure about 70%, at a heavy cost to thinking and growth; treatment is now being tailored to four molecular subgroups so that the low-risk children get less."}],"tags":[{"label":"medulloblastoma","href":"/tagged/medulloblastoma/","tip":"Every record tagged medulloblastoma."},{"label":"st-jude","href":"/tagged/st-jude/","tip":"Every record tagged st-jude."}]},"sortKeys":{"year":0}},{"id":"atrt","name":"Atypical teratoid/rhabdoid tumour (ATRT)","tldr":"ATRT is an aggressive brain tumour of babies and toddlers caused by loss of a single gene, SMARCB1, part of the machinery that opens and closes DNA. Intensive chemotherapy with stem-cell rescue, and radiotherapy where age allows, now cure a meaningful share of children who once had little chance, and drugs aimed at the epigenetic consequence of SMARCB1 loss (EZH2 inhibitors) are in trials.","route":"/cancers/atrt/","kind":"cancer","cancerIcon":"atrt","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"cns","href":"/tagged/cns/","tip":"Every record tagged cns."}]},"sortKeys":{"year":0}},{"id":"rejuv-paed-bone","name":"Bone after childhood cancer: peak bone mass, osteonecrosis and what rebuilds","tldr":"A child treated during the years in which bone is laid down may never reach the peak bone mass they would have had, which is a different problem from an adult losing bone already built. Thirty per cent of adult survivors of childhood leukaemia had low bone density, most strongly associated with growth hormone deficiency and smoking, both treatable.","route":"/technologies/rejuv-paed-bone/","status":"established","kind":"technology","schematic":{"id":"rejuv-paed-bone","sections":["rejuvenation","supportive-care"]},"facets":{"kind":["Technology"],"cancers":["Acute lymphoblastic leukaemia","Standard-risk B-cell acute lymphoblastic leukaemia in children","High-risk acute lymphoblastic leukaemia in children","Childhood cancers","Hodgkin lymphoma"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Technology","tip":"Every way we see, measure, or attack a tumour, explained."},"cancers":[{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."},{"label":"Standard-risk B-cell acute lymphoblastic leukaemia in children","href":"/cancers/all-paediatric-standard-risk/","tip":"Standard-risk acute lymphoblastic leukaemia is the commonest and most curable childhood cancer: a child aged one to nine with a modest white cell count and favourable genetics. Two to three years of chemotherapy cures about nine in ten, and adding the immune drug blinatumomab to the chemotherapy in the AALL1731 trial cut relapses further."},{"label":"High-risk acute lymphoblastic leukaemia in children","href":"/cancers/all-paediatric-high-risk/","tip":"High-risk childhood leukaemia means a child aged ten or over, a very high white cell count, T-cell disease, spread to the brain or testes, or adverse genetics, and it is treated with longer and more intensive chemotherapy. Most children are still cured; the T-cell form gained the drug nelarabine after the AALL0434 trial, and cranial radiotherapy has been dropped for almost everyone."},{"label":"Childhood cancers","href":"/cancers/childhood-cancers/","tip":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available."}],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."}]},"sortKeys":{"year":0}},{"id":"rejuv-paed-breast-after-chest-radiotherapy","name":"Breast cancer after chest radiotherapy in childhood, and the screening that follows","tldr":"A girl who had radiotherapy to the chest carries a risk of breast cancer by age 50 of about 30 per cent. It is not only about dose: a low dose to the whole lung gave a higher standardised incidence than a high dose to a smaller field, because volume matters. Surveillance is recommended from early adulthood, decades before ordinary screening starts.","route":"/technologies/rejuv-paed-breast-after-chest-radiotherapy/","status":"standard-of-care","kind":"technology","schematic":{"id":"rejuv-paed-breast-after-chest-radiotherapy","sections":["rejuvenation","supportive-care","early-detection","imaging"]},"facets":{"kind":["Technology"],"cancers":["Hodgkin lymphoma","Childhood cancers","Breast cancer","Ewing sarcoma","Wilms tumour"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Technology","tip":"Every way we see, measure, or attack a tumour, explained."},"cancers":[{"label":"Hodgkin lymphoma","href":"/cancers/hodgkin-lymphoma/","tip":"Hodgkin lymphoma is one of the most curable cancers, where the goal is now to cure with less toxicity, using brentuximab and, from 2026, first-line nivolumab."},{"label":"Childhood cancers","href":"/cancers/childhood-cancers/","tip":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available."},{"label":"Breast cancer","href":"/cancers/breast-cancer/","tip":"Breast cancer is not one disease, and the result that decides which page you need is the receptor result: whether the cancer carries oestrogen and progesterone receptors, and whether it carries too much HER2. Those two answers give four boxes, not three, with a page for each. Grade and stage do not change which page you read; they change what happens on it."},{"label":"Ewing sarcoma","href":"/cancers/ewing-sarcoma/","tip":"Ewing sarcoma is a bone and soft-tissue cancer of teenagers driven by a single fusion gene, EWSR1-FLI1. Intensive chemotherapy with surgery or radiation cures most localised cases; disease that has spread at diagnosis, and relapse, remain hard to treat, and no drug against the fusion protein itself has yet succeeded."}],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."}]},"sortKeys":{"year":0}},{"id":"bccss","name":"British Childhood Cancer Survivor Study (BCCSS)","tldr":"Britain's own survivor cohort, built on national registration rather than on hospital volunteers, which is why it can follow people for half a century and count deaths that nobody reported. Its central finding is that what kills survivors changes with time: recurrence early, second cancers and heart disease late.","route":"/collections/bccss/","kind":"collection","logo":"https://www.google.com/s2/favicons?domain=research.birmingham.ac.uk&sz=128","avatar":"org","sub":"Centre for Childhood Cancer Survivor Studies, University of Birmingham","facets":{"kind":["Collection"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Collection","tip":"The open databases and registries the field runs on."},"cancers":[],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."},{"label":"cohort","href":"/tagged/cohort/","tip":"Every record tagged cohort."},{"label":"uk","href":"/tagged/uk/","tip":"Every record tagged uk."}]},"sortKeys":{"year":0}},{"id":"burkitt-lymphoma","name":"Burkitt lymphoma","tldr":"Burkitt lymphoma is the fastest-growing human tumour, driven by a single rearrangement that switches on the MYC gene. That speed makes it exquisitely sensitive to chemotherapy: short, intense courses, now with the antibody rituximab, cure the great majority of children in well-resourced settings. The remaining task is to bring the same cure to the African children who make up most cases.","route":"/cancers/burkitt-lymphoma/","kind":"cancer","cancerIcon":"burkitt-lymphoma","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"haematologic","href":"/tagged/haematologic/","tip":"Every record tagged haematologic."},{"label":"global-health","href":"/tagged/global-health/","tip":"Every record tagged global-health."}]},"sortKeys":{"year":0}},{"id":"cns-germ-cell-tumours","name":"Central nervous system germ cell tumours (germinoma and non-germinomatous)","tldr":"Germ cell tumours of the brain grow near the pineal gland or above the pituitary in teenagers. The commonest kind, germinoma, is so sensitive to radiation and chemotherapy that most patients are cured; the other kinds need stronger chemotherapy and radiotherapy, and doctors measure two proteins in the blood and spinal fluid to tell them apart and to follow treatment.","route":"/cancers/cns-germ-cell-tumours/","kind":"cancer","cancerIcon":"cns-germ-cell-tumours","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"subtype-page","href":"/tagged/subtype-page/","tip":"Every record tagged subtype-page."},{"label":"cns","href":"/tagged/cns/","tip":"Every record tagged cns."}]},"sortKeys":{"year":0}},{"id":"ccss","name":"Childhood Cancer Survivor Study (CCSS)","tldr":"The largest study of what happens to children after cancer is cured. Following tens of thousands of survivors for decades, it showed that heart damage, second cancers and other late effects were common after older treatments, and that gentler modern protocols have already halved late deaths.","route":"/trials/ccss/","status":"active","kind":"trial","cancerIcon":"all-leukemia","sub":"NCT01120353","facets":{"kind":["Trial"],"cancers":["Acute lymphoblastic leukaemia","Hodgkin lymphoma","Medulloblastoma","Wilms tumour","Neuroblastoma","Rhabdomyosarcoma","Osteosarcoma","Ewing sarcoma"],"year":["2016"]},"cols":{"kind":{"facet":"kind","value":"Trial","tip":"Landmark and current trials that define the standard of care."},"cancers":[{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."},{"label":"Hodgkin lymphoma","href":"/cancers/hodgkin-lymphoma/","tip":"Hodgkin lymphoma is one of the most curable cancers, where the goal is now to cure with less toxicity, using brentuximab and, from 2026, first-line nivolumab."},{"label":"Medulloblastoma","href":"/cancers/medulloblastoma/","tip":"Medulloblastoma is the most common malignant childhood brain tumour, arising in the cerebellum. Surgery, radiation to the whole brain and spine, and chemotherapy cure about 70%, at a heavy cost to thinking and growth; treatment is now being tailored to four molecular subgroups so that the low-risk children get less."},{"label":"Wilms tumour","href":"/cancers/wilms-tumor/","tip":"Wilms tumour is a kidney cancer of young children and one of paediatric oncology's success stories: surgery plus a few months of chemotherapy cures about nine in ten. Today's trials aim to give the lowest-risk children almost no chemotherapy while finding the few with aggressive biology."}],"year":{"facet":"year","value":"2016"},"tags":[{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."}]},"sortKeys":{"year":2016}},{"id":"childhood-cancers","name":"Childhood cancers (all types)","tldr":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available.","route":"/cancers/childhood-cancers/","kind":"cancer","cancerIcon":"childhood-cancers","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"umbrella","href":"/tagged/umbrella/","tip":"Every record tagged umbrella."}]},"sortKeys":{"year":0}},{"id":"pleuropulmonary-blastoma","name":"Childhood lung and airway tumours (pleuropulmonary blastoma, tracheobronchial tumours)","tldr":"Primary lung tumours in children are rare and unlike adult lung cancer. Pleuropulmonary blastoma starts as a lung cyst in infants and results from a faulty DICER1 gene that also predisposes to thyroid, ovarian and kidney tumours; removing cysts early, guided by an international registry and family gene testing, prevents progression to the aggressive solid forms.","route":"/cancers/pleuropulmonary-blastoma/","kind":"cancer","cancerIcon":"pleuropulmonary-blastoma","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"thoracic","href":"/tagged/thoracic/","tip":"Every record tagged thoracic."}]},"sortKeys":{"year":0}},{"id":"ching-hon-pui","name":"Ching-Hon Pui","tldr":"Led the St. Jude Total Therapy studies that cure over 90% of children with leukaemia without cranial radiation.","route":"/people/ching-hon-pui/","kind":"person","round":true,"avatar":"person","sub":"Chair, Department of Oncology, St. Jude Children's Research Hospital · St. Jude Children's Research Hospital","facets":{"kind":["Person"],"cancers":["Acute lymphoblastic leukaemia"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."}],"tags":[{"label":"all","href":"/tagged/all/","tip":"Every record tagged all."},{"label":"st-jude","href":"/tagged/st-jude/","tip":"Every record tagged st-jude."}]},"sortKeys":{"year":0}},{"id":"chordoma","name":"Chordoma","tldr":"Chordoma is a slow-growing bone cancer (a sarcoma) of the skull base and spine that arises from leftover embryonic notochord cells. Complete surgery followed by high-dose proton or carbon-ion radiotherapy controls most tumours, and the whole disease depends on a single transcription factor, brachyury, which vaccines and degraders are now trying to hit.","route":"/cancers/chordoma/","kind":"cancer","cancerIcon":"chordoma","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"rare","href":"/tagged/rare/","tip":"Every record tagged rare."},{"label":"sarcoma","href":"/tagged/sarcoma/","tip":"Every record tagged sarcoma."}]},"sortKeys":{"year":0}},{"id":"aaml0531","name":"COG AAML0531","tldr":"Adding the antibody-drug conjugate gemtuzumab ozogamicin to chemotherapy lowered the chance of relapse in children with acute myeloid leukaemia. Years after the drug had been withdrawn from the US market, this trial helped bring it back for children.","route":"/trials/aaml0531/","status":"positive","kind":"trial","cancerIcon":"aml","sub":"NCT00372593","facets":{"kind":["Trial"],"cancers":["Acute myeloid leukaemia","Acute myeloid leukaemia in children"],"year":["2014"]},"cols":{"kind":{"facet":"kind","value":"Trial","tip":"Landmark and current trials that define the standard of care."},"cancers":[{"label":"Acute myeloid leukaemia","href":"/cancers/aml/","tip":"Acute myeloid leukaemia is an aggressive blood cancer where, after 40 years of the same chemotherapy, a wave of targeted drugs (FLT3, IDH, BCL-2, menin) arrived."},{"label":"Acute myeloid leukaemia in children","href":"/cancers/aml-paediatric/","tip":"Acute myeloid leukaemia in children carries gene fusions rather than the mutations of ageing, is treated with four or five intensive courses of chemotherapy, and cures around two thirds of children. Adding gemtuzumab ozogamicin lowered relapse in the AAML0531 trial, and the menin inhibitor revumenib is the first targeted drug approved for the KMT2A-rearranged form common in young children."}],"year":{"facet":"year","value":"2014"},"tags":[{"label":"haematologic","href":"/tagged/haematologic/","tip":"Every record tagged haematologic."}]},"sortKeys":{"year":2014}},{"id":"acns0331","name":"COG ACNS0331","tldr":"This trial asked whether children with average-risk medulloblastoma could safely receive less radiation. Shrinking the boost to the tumour bed was safe; cutting the dose to the whole brain and spine in young children was not, so 23.4 Gy remains the floor for most.","route":"/trials/acns0331/","status":"mixed","kind":"trial","cancerIcon":"medulloblastoma","sub":"NCT00085735","facets":{"kind":["Trial"],"cancers":["Medulloblastoma","WNT-activated medulloblastoma","Group 3 and group 4 medulloblastoma"],"year":["2021"]},"cols":{"kind":{"facet":"kind","value":"Trial","tip":"Landmark and current trials that define the standard of care."},"cancers":[{"label":"Medulloblastoma","href":"/cancers/medulloblastoma/","tip":"Medulloblastoma is the most common malignant childhood brain tumour, arising in the cerebellum. Surgery, radiation to the whole brain and spine, and chemotherapy cure about 70%, at a heavy cost to thinking and growth; treatment is now being tailored to four molecular subgroups so that the low-risk children get less."},{"label":"WNT-activated medulloblastoma","href":"/cancers/medulloblastoma-wnt/","tip":"WNT-activated medulloblastoma is the rarest and most curable of the four molecular groups of medulloblastoma, a brain tumour of the cerebellum. It is driven by a mutation in the beta-catenin gene that switches the WNT growth pathway on. Almost every child is cured with standard therapy, so current trials are asking how much radiotherapy and chemotherapy can be taken away."},{"label":"Group 3 and group 4 medulloblastoma","href":"/cancers/medulloblastoma-group-3-4/","tip":"Group 3 and group 4 medulloblastoma are the two commonest forms of this cerebellar brain tumour and the ones without a druggable driver. Group 3 strikes young children, often with extra copies of MYC and spread through the spinal fluid; group 4 affects older boys. Both get surgery, craniospinal radiotherapy and chemotherapy; trials showed the radiation dose cannot be cut for young children."}],"year":{"facet":"year","value":"2021"},"tags":[{"label":"cns","href":"/tagged/cns/","tip":"Every record tagged cns."},{"label":"de-escalation","href":"/tagged/de-escalation/","tip":"Every record tagged de-escalation."}]},"sortKeys":{"year":2021}},{"id":"aren0533","name":"COG AREN0533","tldr":"A risk-adapted Wilms tumour trial: children whose lung metastases vanished after six weeks of chemotherapy were spared lung radiation, while those with stubborn nodules or a high-risk chromosome pattern got stronger chemotherapy and did better than in the past.","route":"/trials/aren0533/","status":"positive","kind":"trial","cancerIcon":"wilms-tumor","sub":"NCT00379340","facets":{"kind":["Trial"],"cancers":["Wilms tumour"],"year":["2018"]},"cols":{"kind":{"facet":"kind","value":"Trial","tip":"Landmark and current trials that define the standard of care."},"cancers":[{"label":"Wilms tumour","href":"/cancers/wilms-tumor/","tip":"Wilms tumour is a kidney cancer of young children and one of paediatric oncology's success stories: surgery plus a few months of chemotherapy cures about nine in ten. Today's trials aim to give the lowest-risk children almost no chemotherapy while finding the few with aggressive biology."}],"year":{"facet":"year","value":"2018"},"tags":[{"label":"kidney","href":"/tagged/kidney/","tip":"Every record tagged kidney."}]},"sortKeys":{"year":2018}},{"id":"craniopharyngioma","name":"Craniopharyngioma","tldr":"Craniopharyngioma is a benign but destructive brain tumour growing from embryonic remnants beside the pituitary gland and hypothalamus. Surgery, or limited surgery plus radiotherapy, cures most people, but the price can be lifelong hormone deficiency and severe obesity. The adult (papillary) form carries a BRAF mutation and shrinks markedly with BRAF and MEK inhibitors, its first drug treatment.","route":"/cancers/craniopharyngioma/","kind":"cancer","cancerIcon":"craniopharyngioma","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"cns","href":"/tagged/cns/","tip":"Every record tagged cns."}]},"sortKeys":{"year":0}},{"id":"rejuv-history-cure-is-not-enough","name":"Cure is not enough: when late effects stopped being an afterthought","tldr":"Children started surviving cancer in the 1960s, and within a decade the doctors who had cured them began writing down what the cure had cost. The phrase that stuck, from a 1975 paper by Giulio D'Angio, is that cure is not enough.","route":"/terms/rejuv-history-cure-is-not-enough/","kind":"term","term":{"category":"Clinic basics"},"facets":{"kind":["Term"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Term","tip":"Glossary with plain-English TL;DRs and Wikipedia links."},"cancers":[],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"history","href":"/tagged/history/","tip":"Every record tagged history."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."}]},"sortKeys":{"year":0}},{"id":"darren-hargrave","name":"Darren R. Hargrave","tldr":"London paediatric neuro-oncologist who led the trial showing that dabrafenib and trametinib work in children with BRAF V600-mutant high-grade glioma.","route":"/people/darren-hargrave/","kind":"person","round":true,"avatar":"person","sub":"Paediatric neuro-oncologist, Great Ormond Street Hospital and UCL Great Ormond Street Institute of Child Health · Great Ormond Street Hospital for Children","facets":{"kind":["Person"],"cancers":["Paediatric high-grade glioma","Childhood cancers"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[{"label":"Paediatric high-grade glioma","href":"/cancers/paediatric-high-grade-glioma/","tip":"High-grade gliomas in children look like adult glioblastoma under the microscope but are driven by different genes, so they are now classified separately. Surgery and radiotherapy remain the mainstay and chemotherapy adds little; the real gains are in small subsets with a targetable gene change, such as BRAF V600E tumours and the fusion-driven tumours of infants."},{"label":"Childhood cancers","href":"/cancers/childhood-cancers/","tip":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available."}],"tags":[{"label":"brain","href":"/tagged/brain/","tip":"Every record tagged brain."},{"label":"targeted-therapy","href":"/tagged/targeted-therapy/","tip":"Every record tagged targeted-therapy."},{"label":"trialist","href":"/tagged/trialist/","tip":"Every record tagged trialist."}]},"sortKeys":{"year":0}},{"id":"dipg-dmg","name":"Diffuse midline glioma, H3 K27-altered (including DIPG)","tldr":"Diffuse midline glioma grows through the brainstem and cannot be removed surgically. A single change in a histone protein (H3 K27M) rewires how the tumour reads its DNA. Radiotherapy was long the only help; in 2025 the first drug aimed at this tumour, dordaviprone (ONC201), was approved after durable shrinkage in some patients, and GD2 CAR-T cells have produced striking early responses.","route":"/cancers/dipg-dmg/","kind":"cancer","cancerIcon":"dipg-dmg","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"cns","href":"/tagged/cns/","tip":"Every record tagged cns."}]},"sortKeys":{"year":0}},{"id":"rejuv-ayac-education-and-work","name":"Education, work and the years that were interrupted","tldr":"Cancer in the years when education and first jobs happen costs more than the time taken. In the largest cohort, 23 per cent of childhood cancer survivors had used special education services against 8 per cent of siblings, and survivors of several cancers were less likely to finish high school. The important finding is that where the educational support was given, the gap closed.","route":"/technologies/rejuv-ayac-education-and-work/","status":"established","kind":"technology","schematic":{"id":"rejuv-ayac-education-and-work","sections":["rejuvenation","supportive-care"]},"facets":{"kind":["Technology"],"cancers":["Acute lymphoblastic leukaemia","Childhood cancers","Medulloblastoma","Paediatric low-grade glioma","Hodgkin lymphoma","Neuroblastoma"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Technology","tip":"Every way we see, measure, or attack a tumour, explained."},"cancers":[{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."},{"label":"Childhood cancers","href":"/cancers/childhood-cancers/","tip":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available."},{"label":"Medulloblastoma","href":"/cancers/medulloblastoma/","tip":"Medulloblastoma is the most common malignant childhood brain tumour, arising in the cerebellum. Surgery, radiation to the whole brain and spine, and chemotherapy cure about 70%, at a heavy cost to thinking and growth; treatment is now being tailored to four molecular subgroups so that the low-risk children get less."},{"label":"Paediatric low-grade glioma","href":"/cancers/paediatric-low-grade-glioma/","tip":"Paediatric low-grade gliomas are slow-growing brain tumours driven almost always by a single overactive signal, the MAPK pathway, most often through a BRAF gene change. Because the switch is known, pills that block it (dabrafenib with trametinib, and tovorafenib) now shrink tumours far more often than chemotherapy, and children are increasingly spared radiation to the developing brain."}],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."}]},"sortKeys":{"year":0}},{"id":"ependymoma","name":"Ependymoma","tldr":"Ependymomas grow from the cells lining the fluid spaces of the brain and spinal cord, mostly in children under five. Removing the whole tumour followed by focused radiotherapy controls most cases; molecular groups defined in 2021 behave differently, with posterior fossa group A relapsing often, and there is no approved drug.","route":"/cancers/ependymoma/","kind":"cancer","cancerIcon":"ependymoma","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"cns","href":"/tagged/cns/","tip":"Every record tagged cns."}]},"sortKeys":{"year":0}},{"id":"euramos-1","name":"EURAMOS-1","tldr":"The largest osteosarcoma trial ever run, across four cooperative groups on two continents. Neither adding interferon for good responders nor adding ifosfamide and etoposide for poor responders improved outcomes, so three-drug MAP chemotherapy remained the standard and the field turned to new biology.","route":"/trials/euramos-1/","status":"negative","kind":"trial","cancerIcon":"osteosarcoma","sub":"NCT00134030","facets":{"kind":["Trial"],"cancers":["Osteosarcoma"],"year":["2016"]},"cols":{"kind":{"facet":"kind","value":"Trial","tip":"Landmark and current trials that define the standard of care."},"cancers":[{"label":"Osteosarcoma","href":"/cancers/osteosarcoma/","tip":"Osteosarcoma is the most common bone cancer, mostly in teenagers. Chemotherapy plus surgery cures about two-thirds when it has not spread; because no new drug has beaten that chemotherapy in a large trial in 30 years, the next gains are being sought in cellular therapy against GD2, HER2 and B7-H3."}],"year":{"facet":"year","value":"2016"},"tags":[{"label":"bone","href":"/tagged/bone/","tip":"Every record tagged bone."}]},"sortKeys":{"year":2016}},{"id":"ewing-sarcoma","name":"Ewing sarcoma","tldr":"Ewing sarcoma is a bone and soft-tissue cancer of teenagers driven by a single fusion gene, EWSR1-FLI1. Intensive chemotherapy with surgery or radiation cures most localised cases; disease that has spread at diagnosis, and relapse, remain hard to treat, and no drug against the fusion protein itself has yet succeeded.","route":"/cancers/ewing-sarcoma/","kind":"cancer","cancerIcon":"ewing-sarcoma","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"sarcoma","href":"/tagged/sarcoma/","tip":"Every record tagged sarcoma."},{"label":"aya","href":"/tagged/aya/","tip":"Every record tagged aya."}]},"sortKeys":{"year":0}},{"id":"rejuv-paed-fertility-male","name":"Fertility in boys and young men treated for cancer, including testicular tissue banking","tldr":"Male survivors were about half as likely as their brothers to father a child, and the causes are specific: testicular radiotherapy above 7.5 gray, and high cumulative cyclophosphamide, ifosfamide, procarbazine or cisplatin. A young man with none of those was no less likely than his brother. Sperm banking works; tissue banking before puberty has produced no births.","route":"/technologies/rejuv-paed-fertility-male/","status":"established","kind":"technology","schematic":{"id":"rejuv-paed-fertility-male","sections":["rejuvenation","supportive-care","hormonal"]},"facets":{"kind":["Technology"],"cancers":["Childhood cancers","Acute lymphoblastic leukaemia","Hodgkin lymphoma","Ewing sarcoma","Germ cell tumours of childhood and adolescence","Testicular germ cell tumours"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Technology","tip":"Every way we see, measure, or attack a tumour, explained."},"cancers":[{"label":"Childhood cancers","href":"/cancers/childhood-cancers/","tip":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available."},{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."},{"label":"Hodgkin lymphoma","href":"/cancers/hodgkin-lymphoma/","tip":"Hodgkin lymphoma is one of the most curable cancers, where the goal is now to cure with less toxicity, using brentuximab and, from 2026, first-line nivolumab."},{"label":"Ewing sarcoma","href":"/cancers/ewing-sarcoma/","tip":"Ewing sarcoma is a bone and soft-tissue cancer of teenagers driven by a single fusion gene, EWSR1-FLI1. Intensive chemotherapy with surgery or radiation cures most localised cases; disease that has spread at diagnosis, and relapse, remain hard to treat, and no drug against the fusion protein itself has yet succeeded."}],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."}]},"sortKeys":{"year":0}},{"id":"rejuv-paed-fertility-female","name":"Fertility in girls and young women treated for cancer, including ovarian tissue freezing","tldr":"Most female survivors treated with chemotherapy and no radiotherapy to the pelvis or brain can become pregnant: the large cohort that asked found chemotherapy-specific effects were few. The exceptions are busulfan, high-dose lomustine, pelvic and cranial radiotherapy and transplant conditioning. Before puberty, freezing ovarian tissue is the only option.","route":"/technologies/rejuv-paed-fertility-female/","status":"standard-of-care","kind":"technology","schematic":{"id":"rejuv-paed-fertility-female","sections":["rejuvenation","supportive-care","hormonal"]},"facets":{"kind":["Technology"],"cancers":["Childhood cancers","Acute lymphoblastic leukaemia","Hodgkin lymphoma","Ewing sarcoma","Osteosarcoma","Germ cell tumours of childhood and adolescence"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Technology","tip":"Every way we see, measure, or attack a tumour, explained."},"cancers":[{"label":"Childhood cancers","href":"/cancers/childhood-cancers/","tip":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available."},{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."},{"label":"Hodgkin lymphoma","href":"/cancers/hodgkin-lymphoma/","tip":"Hodgkin lymphoma is one of the most curable cancers, where the goal is now to cure with less toxicity, using brentuximab and, from 2026, first-line nivolumab."},{"label":"Ewing sarcoma","href":"/cancers/ewing-sarcoma/","tip":"Ewing sarcoma is a bone and soft-tissue cancer of teenagers driven by a single fusion gene, EWSR1-FLI1. Intensive chemotherapy with surgery or radiation cures most localised cases; disease that has spread at diagnosis, and relapse, remain hard to treat, and no drug against the fusion protein itself has yet succeeded."}],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."}]},"sortKeys":{"year":0}},{"id":"firefly-1","name":"FIREFLY-1","tldr":"FIREFLY-1 showed that a once-weekly pill, tovorafenib, shrinks most relapsed childhood low-grade gliomas driven by BRAF changes, including the common KIAA1549-BRAF fusion that older BRAF drugs could not treat safely. It led to the first approval of a drug for this disease.","route":"/trials/firefly-1/","status":"positive","kind":"trial","cancerIcon":"paediatric-low-grade-glioma","sub":"NCT04775485","facets":{"kind":["Trial"],"cancers":["Paediatric low-grade glioma"],"year":["2023"]},"cols":{"kind":{"facet":"kind","value":"Trial","tip":"Landmark and current trials that define the standard of care."},"cancers":[{"label":"Paediatric low-grade glioma","href":"/cancers/paediatric-low-grade-glioma/","tip":"Paediatric low-grade gliomas are slow-growing brain tumours driven almost always by a single overactive signal, the MAPK pathway, most often through a BRAF gene change. Because the switch is known, pills that block it (dabrafenib with trametinib, and tovorafenib) now shrink tumours far more often than chemotherapy, and children are increasingly spared radiation to the developing brain."}],"year":{"facet":"year","value":"2023"},"tags":[{"label":"cns","href":"/tagged/cns/","tip":"Every record tagged cns."}]},"sortKeys":{"year":2023}},{"id":"paediatric-germ-cell-tumours","name":"Germ cell tumours of childhood and adolescence (extracranial and CNS)","tldr":"Germ cell tumours arise from the cells meant to become eggs or sperm and can appear in the gonads, lower back, chest or brain. They are among the most curable childhood cancers because they respond to cisplatin chemotherapy and release blood markers that make monitoring easy. The work now is to cure with less: surgery alone for low-risk tumours, gentler platinum drugs, and protecting hearing.","route":"/cancers/paediatric-germ-cell-tumours/","kind":"cancer","cancerIcon":"paediatric-germ-cell-tumours","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"germ-cell","href":"/tagged/germ-cell/","tip":"Every record tagged germ-cell."}]},"sortKeys":{"year":0}},{"id":"giles-robinson","name":"Giles W. Robinson","tldr":"Memphis paediatric neuro-oncologist who led the study showing that the Hedgehog inhibitor vismodegib helps only adults and older children whose medulloblastoma belongs to the SHH subgroup, an early example of subgroup-directed brain tumour therapy.","route":"/people/giles-robinson/","kind":"person","round":true,"avatar":"person","sub":"Paediatric neuro-oncologist, St. Jude Children's Research Hospital · St. Jude Children's Research Hospital","facets":{"kind":["Person"],"cancers":["SHH-activated medulloblastoma","Medulloblastoma","Childhood cancers"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[{"label":"SHH-activated medulloblastoma","href":"/cancers/medulloblastoma-shh/","tip":"SHH-activated medulloblastoma is driven by the sonic hedgehog growth pathway, the signal that normally tells the developing cerebellum to grow. In infants it is often cured with chemotherapy alone and no radiotherapy; in adults it responds for a time to hedgehog-blocking pills such as vismodegib; and when it carries a TP53 mutation in an older child, often inherited, it resists everything."},{"label":"Medulloblastoma","href":"/cancers/medulloblastoma/","tip":"Medulloblastoma is the most common malignant childhood brain tumour, arising in the cerebellum. Surgery, radiation to the whole brain and spine, and chemotherapy cure about 70%, at a heavy cost to thinking and growth; treatment is now being tailored to four molecular subgroups so that the low-risk children get less."},{"label":"Childhood cancers","href":"/cancers/childhood-cancers/","tip":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available."}],"tags":[{"label":"brain","href":"/tagged/brain/","tip":"Every record tagged brain."},{"label":"targeted-therapy","href":"/tagged/targeted-therapy/","tip":"Every record tagged targeted-therapy."},{"label":"trialist","href":"/tagged/trialist/","tip":"Every record tagged trialist."}]},"sortKeys":{"year":0}},{"id":"medulloblastoma-group-3-4","name":"Group 3 and group 4 medulloblastoma (non-WNT/non-SHH)","tldr":"Group 3 and group 4 medulloblastoma are the two commonest forms of this cerebellar brain tumour and the ones without a druggable driver. Group 3 strikes young children, often with extra copies of MYC and spread through the spinal fluid; group 4 affects older boys. Both get surgery, craniospinal radiotherapy and chemotherapy; trials showed the radiation dose cannot be cut for young children.","route":"/cancers/medulloblastoma-group-3-4/","kind":"cancer","cancerIcon":"medulloblastoma-group-3-4","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"subtype-page","href":"/tagged/subtype-page/","tip":"Every record tagged subtype-page."},{"label":"cns","href":"/tagged/cns/","tip":"Every record tagged cns."}]},"sortKeys":{"year":0}},{"id":"rejuv-paed-growth-and-height","name":"Growth and final height after treatment in childhood, and growth hormone","tldr":"Radiotherapy that reaches the pituitary stops the growth hormone signal, and radiotherapy to the spine stops the spine growing. In a Dutch cohort of 573 survivors, 8.9 per cent ended up more than two standard deviations below mean adult height, the largest losses after total body irradiation and craniospinal radiotherapy. Replacement restores some height.","route":"/technologies/rejuv-paed-growth-and-height/","status":"standard-of-care","kind":"technology","schematic":{"id":"rejuv-paed-growth-and-height","sections":["rejuvenation","supportive-care","hormonal"]},"facets":{"kind":["Technology"],"cancers":["Childhood cancers","Medulloblastoma","Acute lymphoblastic leukaemia","Paediatric low-grade glioma","Paediatric high-grade glioma"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Technology","tip":"Every way we see, measure, or attack a tumour, explained."},"cancers":[{"label":"Childhood cancers","href":"/cancers/childhood-cancers/","tip":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available."},{"label":"Medulloblastoma","href":"/cancers/medulloblastoma/","tip":"Medulloblastoma is the most common malignant childhood brain tumour, arising in the cerebellum. Surgery, radiation to the whole brain and spine, and chemotherapy cure about 70%, at a heavy cost to thinking and growth; treatment is now being tailored to four molecular subgroups so that the low-risk children get less."},{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."},{"label":"Paediatric low-grade glioma","href":"/cancers/paediatric-low-grade-glioma/","tip":"Paediatric low-grade gliomas are slow-growing brain tumours driven almost always by a single overactive signal, the MAPK pathway, most often through a BRAF gene change. Because the switch is known, pills that block it (dabrafenib with trametinib, and tovorafenib) now shrink tumours far more often than chemotherapy, and children are increasingly spared radiation to the developing brain."}],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."}]},"sortKeys":{"year":0}},{"id":"h3-k27m","name":"H3 K27M mutation","tldr":"H3 K27M is a single change in a histone that defines diffuse midline glioma, including the brain-stem tumour DIPG. In 2025 dordaviprone became the first drug approved for tumours carrying it.","route":"/biomarkers/h3-k27m/","kind":"biomarker","target":{"id":"h3-3a","name":"Histone H3.3 (H3-3A)","targetClass":"other","tldr":"H3.3 is one of the histone proteins DNA wraps around; a single change at position 27 (K27M) locks brain-stem and midline gliomas in an immature state and defines the diagnosis."},"sub":"H3-3A","facets":{"kind":["Biomarker"],"cancers":["Diffuse midline glioma, H3 K27-altered","Paediatric high-grade glioma"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Biomarker","tip":"The readouts a pathology report gives (PD-L1 CPS, HER2 IHC 3+, MSI-high), each under its gene or protein, with the thresholds approvals use and the tests that measure them."},"cancers":[{"label":"Diffuse midline glioma, H3 K27-altered","href":"/cancers/dipg-dmg/","tip":"Diffuse midline glioma grows through the brainstem and cannot be removed surgically. A single change in a histone protein (H3 K27M) rewires how the tumour reads its DNA. Radiotherapy was long the only help; in 2025 the first drug aimed at this tumour, dordaviprone (ONC201), was approved after durable shrinkage in some patients, and GD2 CAR-T cells have produced striking early responses."},{"label":"Paediatric high-grade glioma","href":"/cancers/paediatric-high-grade-glioma/","tip":"High-grade gliomas in children look like adult glioblastoma under the microscope but are driven by different genes, so they are now classified separately. Surgery and radiotherapy remain the mainstay and chemotherapy adds little; the real gains are in small subsets with a targetable gene change, such as BRAF V600E tumours and the fusion-driven tumours of infants."}],"tags":[{"label":"biomarker","href":"/tagged/biomarker/","tip":"Every record tagged biomarker."},{"label":"glioma","href":"/tagged/glioma/","tip":"Every record tagged glioma."}]},"sortKeys":{"year":0}},{"id":"rejuv-paed-hearing","name":"Hearing after platinum and cranial radiotherapy in a developing child","tldr":"Hearing loss in a child still learning to speak and read costs more than the same loss in an adult. In the St Jude Lifetime Cohort, severe hearing impairment affected 34.9 per cent of platinum-treated survivors and 38.3 per cent of those irradiated at the cochlea, against 8.8 per cent of unexposed survivors, and tracked deficits in reasoning, fluency and mathematics.","route":"/technologies/rejuv-paed-hearing/","status":"standard-of-care","kind":"technology","schematic":{"id":"rejuv-paed-hearing","sections":["rejuvenation","supportive-care","chemotherapy"]},"facets":{"kind":["Technology"],"cancers":["Neuroblastoma","Medulloblastoma","Osteosarcoma","Hepatoblastoma","Germ cell tumours of childhood and adolescence","Childhood cancers"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Technology","tip":"Every way we see, measure, or attack a tumour, explained."},"cancers":[{"label":"Neuroblastoma","href":"/cancers/neuroblastoma/","tip":"Neuroblastoma is a childhood nerve-cell cancer where anti-GD2 antibodies and, recently, GD2 CAR-T have improved survival in high-risk disease."},{"label":"Medulloblastoma","href":"/cancers/medulloblastoma/","tip":"Medulloblastoma is the most common malignant childhood brain tumour, arising in the cerebellum. Surgery, radiation to the whole brain and spine, and chemotherapy cure about 70%, at a heavy cost to thinking and growth; treatment is now being tailored to four molecular subgroups so that the low-risk children get less."},{"label":"Osteosarcoma","href":"/cancers/osteosarcoma/","tip":"Osteosarcoma is the most common bone cancer, mostly in teenagers. Chemotherapy plus surgery cures about two-thirds when it has not spread; because no new drug has beaten that chemotherapy in a large trial in 30 years, the next gains are being sought in cellular therapy against GD2, HER2 and B7-H3."},{"label":"Hepatoblastoma","href":"/cancers/hepatoblastoma/","tip":"Hepatoblastoma is a childhood liver cancer, mostly of toddlers, cured in most standard-risk cases with cisplatin chemotherapy and surgery, including liver transplant when the tumour cannot be cut out. Sodium thiosulfate given after cisplatin halves the permanent hearing loss cisplatin causes, and became the first approved otoprotectant in 2022."}],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."}]},"sortKeys":{"year":0}},{"id":"hepatoblastoma","name":"Hepatoblastoma","tldr":"Hepatoblastoma is a childhood liver cancer, mostly of toddlers, cured in most standard-risk cases with cisplatin chemotherapy and surgery, including liver transplant when the tumour cannot be cut out. Sodium thiosulfate given after cisplatin halves the permanent hearing loss cisplatin causes, and became the first approved otoprotectant in 2022.","route":"/cancers/hepatoblastoma/","kind":"cancer","cancerIcon":"hepatoblastoma","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[]},"sortKeys":{"year":0}},{"id":"all-paediatric-high-risk","name":"High-risk acute lymphoblastic leukaemia in children (high-risk B-ALL and T-ALL)","tldr":"High-risk childhood leukaemia means a child aged ten or over, a very high white cell count, T-cell disease, spread to the brain or testes, or adverse genetics, and it is treated with longer and more intensive chemotherapy. Most children are still cured; the T-cell form gained the drug nelarabine after the AALL0434 trial, and cranial radiotherapy has been dropped for almost everyone.","route":"/cancers/all-paediatric-high-risk/","kind":"cancer","cancerIcon":"all-paediatric-high-risk","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"subtype-page","href":"/tagged/subtype-page/","tip":"Every record tagged subtype-page."}]},"sortKeys":{"year":0}},{"id":"neuroblastoma-high-risk","name":"High-risk neuroblastoma","tldr":"High-risk neuroblastoma has spread widely in a child over 18 months old or carries extra copies of the MYCN gene. Treatment lasts about 18 months and uses every tool: chemotherapy, surgery, high-dose chemotherapy with stem cell rescue, radiotherapy, and the anti-GD2 antibody dinutuximab, which raised survival in ANBL0032; eflornithine, given afterwards, was approved in 2023 to lower relapse.","route":"/cancers/neuroblastoma-high-risk/","kind":"cancer","cancerIcon":"neuroblastoma-high-risk","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"subtype-page","href":"/tagged/subtype-page/","tip":"Every record tagged subtype-page."}]},"sortKeys":{"year":0}},{"id":"histiocyte-society","name":"Histiocyte Society","tldr":"The international society of doctors and scientists who study histiocytic disorders; its LCH trials, run since the 1990s, set the worldwide standard for treating Langerhans cell histiocytosis in children.","route":"/institutions/histiocyte-society/","kind":"institution","logo":"/logos/histiocyte-society.png","avatar":"org","sub":"Pitman, US","facets":{"kind":["Institution"],"cancers":["Langerhans cell histiocytosis","Erdheim-Chester disease, Rosai-Dorfman disease and other histiocytic neoplasms"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Institution","tip":"The centres and universities that matter, mapped and ranked."},"cancers":[{"label":"Langerhans cell histiocytosis","href":"/cancers/langerhans-cell-histiocytosis/","tip":"Langerhans cell histiocytosis is a disorder in which a small group of immune cells with a faulty growth signal (most often a BRAF mutation) pile up in bone, skin, pituitary or organs. It ranges from a single bone lesion that heals after biopsy to a life-threatening disease of infants. A year of gentle chemotherapy cures most children, and BRAF or MEK inhibitors rescue those with resistant disease."},{"label":"Erdheim-Chester disease, Rosai-Dorfman disease and other histiocytic neoplasms","href":"/cancers/histiocytoses/","tip":"Histiocytoses are diseases in which immune scavenger cells build up in bone, heart, brain, kidneys and skin. They used to be treated as inflammatory conditions with steroids and interferon. The discovery that most carry mutations in the same growth pathway as melanoma turned them into targetable cancers: BRAF and MEK inhibitor pills now produce responses in nearly every treated patient."}],"tags":[]},"sortKeys":{"year":0}},{"id":"rejuv-ayac-diagnostic-delay","name":"How long it takes to diagnose cancer in a young person, and what the evidence actually says","tldr":"Young people often say their cancer took a long time to diagnose, and the research agrees that time to diagnosis varies widely by tumour type and age. What the research does not support is a single number: a systematic review found the studies used different definitions and skewed data that could not be combined, so no meta-analysis was possible.","route":"/technologies/rejuv-ayac-diagnostic-delay/","status":"established","kind":"technology","schematic":{"id":"rejuv-ayac-diagnostic-delay","sections":["rejuvenation","supportive-care","early-detection"]},"facets":{"kind":["Technology"],"cancers":["Ewing sarcoma","Osteosarcoma","Hodgkin lymphoma","Paediatric high-grade glioma","Testicular germ cell tumours","Childhood cancers"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Technology","tip":"Every way we see, measure, or attack a tumour, explained."},"cancers":[{"label":"Ewing sarcoma","href":"/cancers/ewing-sarcoma/","tip":"Ewing sarcoma is a bone and soft-tissue cancer of teenagers driven by a single fusion gene, EWSR1-FLI1. Intensive chemotherapy with surgery or radiation cures most localised cases; disease that has spread at diagnosis, and relapse, remain hard to treat, and no drug against the fusion protein itself has yet succeeded."},{"label":"Osteosarcoma","href":"/cancers/osteosarcoma/","tip":"Osteosarcoma is the most common bone cancer, mostly in teenagers. Chemotherapy plus surgery cures about two-thirds when it has not spread; because no new drug has beaten that chemotherapy in a large trial in 30 years, the next gains are being sought in cellular therapy against GD2, HER2 and B7-H3."},{"label":"Hodgkin lymphoma","href":"/cancers/hodgkin-lymphoma/","tip":"Hodgkin lymphoma is one of the most curable cancers, where the goal is now to cure with less toxicity, using brentuximab and, from 2026, first-line nivolumab."},{"label":"Paediatric high-grade glioma","href":"/cancers/paediatric-high-grade-glioma/","tip":"High-grade gliomas in children look like adult glioblastoma under the microscope but are driven by different genes, so they are now classified separately. Surgery and radiotherapy remain the mainstay and chemotherapy adds little; the real gains are in small subsets with a targetable gene change, such as BRAF V600E tumours and the fusion-driven tumours of infants."}],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."}]},"sortKeys":{"year":0}},{"id":"rejuv-paed-chronic-disease-burden","name":"How much illness childhood cancer survivors carry, and at what age","tldr":"The figures, with the cohort and the age attached, because they are misquoted more than any others. On self-report at a mean age of 26, 62.3 per cent of survivors had a chronic condition. On clinical testing, the cumulative prevalence of any chronic condition by age 45 was 95.5 per cent, and by age 50 a survivor had 17.1 conditions against 9.2 in matched controls.","route":"/technologies/rejuv-paed-chronic-disease-burden/","status":"established","kind":"technology","schematic":{"id":"rejuv-paed-chronic-disease-burden","sections":["rejuvenation","supportive-care"]},"facets":{"kind":["Technology"],"cancers":["Childhood cancers","Acute lymphoblastic leukaemia","Hodgkin lymphoma","Neuroblastoma","Wilms tumour","Medulloblastoma","Osteosarcoma","Ewing sarcoma"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Technology","tip":"Every way we see, measure, or attack a tumour, explained."},"cancers":[{"label":"Childhood cancers","href":"/cancers/childhood-cancers/","tip":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available."},{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."},{"label":"Hodgkin lymphoma","href":"/cancers/hodgkin-lymphoma/","tip":"Hodgkin lymphoma is one of the most curable cancers, where the goal is now to cure with less toxicity, using brentuximab and, from 2026, first-line nivolumab."},{"label":"Neuroblastoma","href":"/cancers/neuroblastoma/","tip":"Neuroblastoma is a childhood nerve-cell cancer where anti-GD2 antibodies and, recently, GD2 CAR-T have improved survival in high-risk disease."}],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."}]},"sortKeys":{"year":0}},{"id":"all-infant","name":"Infant acute lymphoblastic leukaemia (KMT2A-rearranged, under one year)","tldr":"Leukaemia diagnosed in the first year of life is a different disease from leukaemia in older children: most cases carry a broken KMT2A gene and respond poorly to chemotherapy, and fewer than half of infants were cured for twenty years. One course of the immune drug blinatumomab after induction raised two-year disease-free survival from about half to over 80 percent in a pilot study.","route":"/cancers/all-infant/","kind":"cancer","cancerIcon":"all-infant","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"subtype-page","href":"/tagged/subtype-page/","tip":"Every record tagged subtype-page."}]},"sortKeys":{"year":0}},{"id":"inflammatory-myofibroblastic-tumour","name":"Inflammatory myofibroblastic tumour (IMT)","tldr":"IMT is a rare tumour, grouped with the sarcomas, of spindle cells mixed with inflammatory cells, most often in the lung or abdomen of children and young adults. Surgery cures most, and about half carry an ALK gene fusion, so the ALK-blocking pill crizotinib is approved for those that cannot be removed, one of the first targeted approvals for a childhood solid tumour.","route":"/cancers/inflammatory-myofibroblastic-tumour/","kind":"cancer","cancerIcon":"inflammatory-myofibroblastic-tumour","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"rare","href":"/tagged/rare/","tip":"Every record tagged rare."},{"label":"sarcoma","href":"/tagged/sarcoma/","tip":"Every record tagged sarcoma."}]},"sortKeys":{"year":0}},{"id":"inge-van-der-sluis","name":"Inge M. van der Sluis","tldr":"Dutch paediatric oncologist who led the study showing that adding one course of blinatumomab to chemotherapy sharply improves survival for babies with KMT2A-rearranged leukaemia.","route":"/people/inge-van-der-sluis/","kind":"person","round":true,"avatar":"person","sub":"Paediatric oncologist, Princess Máxima Center for Pediatric Oncology · Princess Máxima Center for Pediatric Oncology","facets":{"kind":["Person"],"cancers":["Infant acute lymphoblastic leukaemia","Acute lymphoblastic leukaemia","Childhood cancers"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[{"label":"Infant acute lymphoblastic leukaemia","href":"/cancers/all-infant/","tip":"Leukaemia diagnosed in the first year of life is a different disease from leukaemia in older children: most cases carry a broken KMT2A gene and respond poorly to chemotherapy, and fewer than half of infants were cured for twenty years. One course of the immune drug blinatumomab after induction raised two-year disease-free survival from about half to over 80 percent in a pilot study."},{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."},{"label":"Childhood cancers","href":"/cancers/childhood-cancers/","tip":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available."}],"tags":[{"label":"leukaemia","href":"/tagged/leukaemia/","tip":"Every record tagged leukaemia."},{"label":"immunotherapy","href":"/tagged/immunotherapy/","tip":"Every record tagged immunotherapy."},{"label":"trialist","href":"/tagged/trialist/","tip":"Every record tagged trialist."}]},"sortKeys":{"year":0}},{"id":"itcc","name":"Innovative Therapies for Children with Cancer (ITCC)","tldr":"Europe's network of children's hospitals that run the first trials of new cancer drugs in children, so that European children can access experimental medicines close to home.","route":"/companies/itcc/","kind":"company","logo":"/logos/itcc.png","avatar":"org","sub":"Villejuif, FR","facets":{"kind":["Company"],"cancers":["Neuroblastoma","Diffuse midline glioma, H3 K27-altered","Paediatric low-grade glioma","Rhabdomyosarcoma"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Company","tip":"Who is building what."},"cancers":[{"label":"Neuroblastoma","href":"/cancers/neuroblastoma/","tip":"Neuroblastoma is a childhood nerve-cell cancer where anti-GD2 antibodies and, recently, GD2 CAR-T have improved survival in high-risk disease."},{"label":"Diffuse midline glioma, H3 K27-altered","href":"/cancers/dipg-dmg/","tip":"Diffuse midline glioma grows through the brainstem and cannot be removed surgically. A single change in a histone protein (H3 K27M) rewires how the tumour reads its DNA. Radiotherapy was long the only help; in 2025 the first drug aimed at this tumour, dordaviprone (ONC201), was approved after durable shrinkage in some patients, and GD2 CAR-T cells have produced striking early responses."},{"label":"Paediatric low-grade glioma","href":"/cancers/paediatric-low-grade-glioma/","tip":"Paediatric low-grade gliomas are slow-growing brain tumours driven almost always by a single overactive signal, the MAPK pathway, most often through a BRAF gene change. Because the switch is known, pills that block it (dabrafenib with trametinib, and tovorafenib) now shrink tumours far more often than chemotherapy, and children are increasingly spared radiation to the developing brain."},{"label":"Rhabdomyosarcoma","href":"/cancers/rhabdomyosarcoma/","tip":"A childhood soft-tissue sarcoma, a cancer of muscle-like cells found anywhere from the eye socket to the bladder. Most children are cured with chemotherapy, surgery and radiation, and a fusion gene (PAX-FOXO1) now decides how intensively to treat."}],"tags":[{"label":"cooperative-group","href":"/tagged/cooperative-group/","tip":"Every record tagged cooperative-group."}]},"sortKeys":{"year":0}},{"id":"inter-b-nhl-ritux-2010","name":"Inter-B-NHL Ritux 2010","tldr":"Adding the antibody rituximab to intensive chemotherapy in children with high-risk Burkitt and related lymphomas cut treatment failures by about two-thirds, making an already curable disease more so. It is the model of a joint European-North American children's cancer trial.","route":"/trials/inter-b-nhl-ritux-2010/","status":"positive","kind":"trial","cancerIcon":"burkitt-lymphoma","sub":"NCT01516580","facets":{"kind":["Trial"],"cancers":["Burkitt lymphoma","Diffuse large B-cell lymphoma"],"year":["2020"]},"cols":{"kind":{"facet":"kind","value":"Trial","tip":"Landmark and current trials that define the standard of care."},"cancers":[{"label":"Burkitt lymphoma","href":"/cancers/burkitt-lymphoma/","tip":"Burkitt lymphoma is the fastest-growing human tumour, driven by a single rearrangement that switches on the MYC gene. That speed makes it exquisitely sensitive to chemotherapy: short, intense courses, now with the antibody rituximab, cure the great majority of children in well-resourced settings. The remaining task is to bring the same cure to the African children who make up most cases."},{"label":"Diffuse large B-cell lymphoma","href":"/cancers/dlbcl/","tip":"Diffuse large B-cell lymphoma (DLBCL) is an aggressive but curable lymphoma. CAR-T cures about 40% of relapsed patients, and off-the-shelf bispecifics are now approved."}],"year":{"facet":"year","value":"2020"},"tags":[{"label":"haematologic","href":"/tagged/haematologic/","tip":"Every record tagged haematologic."}]},"sortKeys":{"year":2020}},{"id":"neuroblastoma-intermediate-risk","name":"Intermediate-risk neuroblastoma","tldr":"Intermediate-risk neuroblastoma sits between the tumours that go away on their own and the high-risk disease that needs everything. A few cycles of moderate chemotherapy followed by surgery cure most children, and trials have spent twenty years showing how few cycles are enough.","route":"/cancers/neuroblastoma-intermediate-risk/","kind":"cancer","cancerIcon":"neuroblastoma-intermediate-risk","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"subtype-page","href":"/tagged/subtype-page/","tip":"Every record tagged subtype-page."}]},"sortKeys":{"year":0}},{"id":"ighg","name":"International Guideline Harmonization Group for late effects of childhood cancer","tldr":"Three countries wrote three different sets of follow-up rules for the same survivors, and the rules disagreed about who to screen, how and how often. Since 2010 this group has been settling those disagreements one organ at a time, in public, with the evidence graded and the disagreement named.","route":"/collections/ighg/","kind":"collection","logo":"https://www.google.com/s2/favicons?domain=ighg.org&sz=128","avatar":"org","sub":"International Guideline Harmonization Group, with PanCare and the Children's Oncology Group","facets":{"kind":["Collection"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Collection","tip":"The open databases and registries the field runs on."},"cancers":[],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."},{"label":"guideline","href":"/tagged/guideline/","tip":"Every record tagged guideline."}]},"sortKeys":{"year":0}},{"id":"jacques-grill","name":"Jacques Grill","tldr":"Paris paediatric neuro-oncologist who led HERBY, the trial that showed adding bevacizumab does not help children with high-grade glioma, and who leads European trials in diffuse midline glioma.","route":"/people/jacques-grill/","kind":"person","round":true,"avatar":"person","sub":"Paediatric neuro-oncologist, Gustave Roussy · Gustave Roussy","facets":{"kind":["Person"],"cancers":["Paediatric high-grade glioma","Glioma & glioblastoma","Childhood cancers"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[{"label":"Paediatric high-grade glioma","href":"/cancers/paediatric-high-grade-glioma/","tip":"High-grade gliomas in children look like adult glioblastoma under the microscope but are driven by different genes, so they are now classified separately. Surgery and radiotherapy remain the mainstay and chemotherapy adds little; the real gains are in small subsets with a targetable gene change, such as BRAF V600E tumours and the fusion-driven tumours of infants."},{"label":"Glioma & glioblastoma","href":"/cancers/glioblastoma/","tip":"Gliomas are now diagnosed by molecular class, and three classes got their first targeted drugs in 2024-25 (vorasidenib for IDH-mutant glioma, tovorafenib for BRAF-altered paediatric glioma, dordaviprone for H3 K27M). Glioblastoma is the hardest to treat: surgery, radiotherapy with temozolomide and tumour treating fields are its backbone, with CAR-T into the brain and focused ultrasound in trials."},{"label":"Childhood cancers","href":"/cancers/childhood-cancers/","tip":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available."}],"tags":[{"label":"brain","href":"/tagged/brain/","tip":"Every record tagged brain."},{"label":"trialist","href":"/tagged/trialist/","tip":"Every record tagged trialist."}]},"sortKeys":{"year":0}},{"id":"jeffrey-dome","name":"Jeffrey S. Dome","tldr":"Led the COG renal tumour committee that used biology to tailor Wilms tumour treatment.","route":"/people/jeffrey-dome/","kind":"person","round":true,"avatar":"person","sub":"Senior Vice President, Center for Cancer and Blood Disorders, Children's National Hospital, Washington DC · Children's National Hospital","facets":{"kind":["Person"],"cancers":["Wilms tumour"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[{"label":"Wilms tumour","href":"/cancers/wilms-tumor/","tip":"Wilms tumour is a kidney cancer of young children and one of paediatric oncology's success stories: surgery plus a few months of chemotherapy cures about nine in ten. Today's trials aim to give the lowest-risk children almost no chemotherapy while finding the few with aggressive biology."}],"tags":[{"label":"wilms-tumor","href":"/tagged/wilms-tumor/","tip":"Every record tagged wilms-tumor."},{"label":"cooperative-group","href":"/tagged/cooperative-group/","tip":"Every record tagged cooperative-group."}]},"sortKeys":{"year":0}},{"id":"john-maris","name":"John M. Maris","tldr":"Neuroblastoma geneticist whose lab found the ALK mutations and immunotherapy targets now in paediatric trials.","route":"/people/john-maris/","kind":"person","round":true,"avatar":"person","sub":"Giulio D'Angio Chair in Neuroblastoma Research, Children's Hospital of Philadelphia · Children's Hospital of Philadelphia","facets":{"kind":["Person"],"cancers":["Neuroblastoma"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[{"label":"Neuroblastoma","href":"/cancers/neuroblastoma/","tip":"Neuroblastoma is a childhood nerve-cell cancer where anti-GD2 antibodies and, recently, GD2 CAR-T have improved survival in high-risk disease."}],"tags":[{"label":"neuroblastoma","href":"/tagged/neuroblastoma/","tip":"Every record tagged neuroblastoma."},{"label":"genomics","href":"/tagged/genomics/","tip":"Every record tagged genomics."}]},"sortKeys":{"year":0}},{"id":"kara-kelly","name":"Kara M. Kelly","tldr":"Leads the COG Hodgkin lymphoma committee and the trials bringing brentuximab and nivolumab to children with the disease.","route":"/people/kara-kelly/","kind":"person","round":true,"avatar":"person","sub":"Chair of Pediatric Oncology, Roswell Park Comprehensive Cancer Center; Division Chief, Pediatric Hematology/Oncology, University at Buffalo · Roswell Park Comprehensive Cancer Center","facets":{"kind":["Person"],"cancers":["Hodgkin lymphoma"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[{"label":"Hodgkin lymphoma","href":"/cancers/hodgkin-lymphoma/","tip":"Hodgkin lymphoma is one of the most curable cancers, where the goal is now to cure with less toxicity, using brentuximab and, from 2026, first-line nivolumab."}],"tags":[{"label":"hodgkin","href":"/tagged/hodgkin/","tip":"Every record tagged hodgkin."},{"label":"cooperative-group","href":"/tagged/cooperative-group/","tip":"Every record tagged cooperative-group."}]},"sortKeys":{"year":0}},{"id":"rejuv-paed-kidneys","name":"Kidneys after cisplatin, ifosfamide, radiotherapy and nephrectomy in childhood","tldr":"A Cochrane review of 61 studies found reported rates of kidney damage after childhood cancer treatment ranging from nought to 84 per cent, which is a statement about the literature rather than about kidneys. On systematic clinical testing of one large cohort, kidney dysfunction was present in 5 per cent, among the least common of the organ problems measured.","route":"/technologies/rejuv-paed-kidneys/","status":"established","kind":"technology","schematic":{"id":"rejuv-paed-kidneys","sections":["rejuvenation","supportive-care"]},"facets":{"kind":["Technology"],"cancers":["Wilms tumour","Neuroblastoma","Osteosarcoma","Ewing sarcoma","Germ cell tumours of childhood and adolescence","Childhood cancers"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Technology","tip":"Every way we see, measure, or attack a tumour, explained."},"cancers":[{"label":"Wilms tumour","href":"/cancers/wilms-tumor/","tip":"Wilms tumour is a kidney cancer of young children and one of paediatric oncology's success stories: surgery plus a few months of chemotherapy cures about nine in ten. Today's trials aim to give the lowest-risk children almost no chemotherapy while finding the few with aggressive biology."},{"label":"Neuroblastoma","href":"/cancers/neuroblastoma/","tip":"Neuroblastoma is a childhood nerve-cell cancer where anti-GD2 antibodies and, recently, GD2 CAR-T have improved survival in high-risk disease."},{"label":"Osteosarcoma","href":"/cancers/osteosarcoma/","tip":"Osteosarcoma is the most common bone cancer, mostly in teenagers. Chemotherapy plus surgery cures about two-thirds when it has not spread; because no new drug has beaten that chemotherapy in a large trial in 30 years, the next gains are being sought in cellular therapy against GD2, HER2 and B7-H3."},{"label":"Ewing sarcoma","href":"/cancers/ewing-sarcoma/","tip":"Ewing sarcoma is a bone and soft-tissue cancer of teenagers driven by a single fusion gene, EWSR1-FLI1. Intensive chemotherapy with surgery or radiation cures most localised cases; disease that has spread at diagnosis, and relapse, remain hard to treat, and no drug against the fusion protein itself has yet succeeded."}],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."}]},"sortKeys":{"year":0}},{"id":"kimberly-dunsmore","name":"Kimberly P. Dunsmore","tldr":"Paediatric oncologist who chaired COG AALL0434, the largest trial ever run in childhood T-cell leukaemia, which showed that adding nelarabine improves disease-free survival.","route":"/people/kimberly-dunsmore/","kind":"person","round":true,"avatar":"person","sub":"Paediatric oncologist and Children's Oncology Group trial chair · Carilion Clinic / Virginia Tech Carilion School of Medicine","facets":{"kind":["Person"],"cancers":["High-risk acute lymphoblastic leukaemia in children","Acute lymphoblastic leukaemia","Childhood cancers"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[{"label":"High-risk acute lymphoblastic leukaemia in children","href":"/cancers/all-paediatric-high-risk/","tip":"High-risk childhood leukaemia means a child aged ten or over, a very high white cell count, T-cell disease, spread to the brain or testes, or adverse genetics, and it is treated with longer and more intensive chemotherapy. Most children are still cured; the T-cell form gained the drug nelarabine after the AALL0434 trial, and cranial radiotherapy has been dropped for almost everyone."},{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."},{"label":"Childhood cancers","href":"/cancers/childhood-cancers/","tip":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available."}],"tags":[{"label":"leukaemia","href":"/tagged/leukaemia/","tip":"Every record tagged leukaemia."},{"label":"trialist","href":"/tagged/trialist/","tip":"Every record tagged trialist."}]},"sortKeys":{"year":0}},{"id":"langerhans-cell-histiocytosis","name":"Langerhans cell histiocytosis (LCH)","tldr":"Langerhans cell histiocytosis is a disorder in which a small group of immune cells with a faulty growth signal (most often a BRAF mutation) pile up in bone, skin, pituitary or organs. It ranges from a single bone lesion that heals after biopsy to a life-threatening disease of infants. A year of gentle chemotherapy cures most children, and BRAF or MEK inhibitors rescue those with resistant disease.","route":"/cancers/langerhans-cell-histiocytosis/","kind":"cancer","cancerIcon":"langerhans-cell-histiocytosis","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"haematologic","href":"/tagged/haematologic/","tip":"Every record tagged haematologic."}]},"sortKeys":{"year":0}},{"id":"rejuv-paed-late-mortality","name":"Late deaths after childhood cancer, what causes them, and the proof that gentler treatment worked","tldr":"Five-year survivors still die earlier than their peers, but much less than they did. Fifteen-year mortality among American five-year survivors fell from 12.4 per cent for children treated in the early 1970s to 6.0 per cent for those treated in the 1990s, and the fall tracks the radiotherapy and anthracycline that were taken out of the protocols.","route":"/technologies/rejuv-paed-late-mortality/","status":"established","kind":"technology","schematic":{"id":"rejuv-paed-late-mortality","sections":["rejuvenation","supportive-care"]},"facets":{"kind":["Technology"],"cancers":["Childhood cancers","Acute lymphoblastic leukaemia","Hodgkin lymphoma","Wilms tumour","Medulloblastoma"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Technology","tip":"Every way we see, measure, or attack a tumour, explained."},"cancers":[{"label":"Childhood cancers","href":"/cancers/childhood-cancers/","tip":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available."},{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."},{"label":"Hodgkin lymphoma","href":"/cancers/hodgkin-lymphoma/","tip":"Hodgkin lymphoma is one of the most curable cancers, where the goal is now to cure with less toxicity, using brentuximab and, from 2026, first-line nivolumab."},{"label":"Wilms tumour","href":"/cancers/wilms-tumor/","tip":"Wilms tumour is a kidney cancer of young children and one of paediatric oncology's success stories: surgery plus a few months of chemotherapy cures about nine in ten. Today's trials aim to give the lowest-risk children almost no chemotherapy while finding the few with aggressive biology."}],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."}]},"sortKeys":{"year":0}},{"id":"lch-iii","name":"LCH-III","tldr":"The Histiocyte Society's third international trial showed that treating multisystem Langerhans cell histiocytosis for a full year, rather than six months, roughly halves the chance of the disease coming back, while adding methotrexate added nothing but toxicity.","route":"/trials/lch-iii/","status":"positive","kind":"trial","cancerIcon":"langerhans-cell-histiocytosis","sub":"NCT00276757","facets":{"kind":["Trial"],"cancers":["Langerhans cell histiocytosis","Multisystem Langerhans cell histiocytosis","Single-system Langerhans cell histiocytosis"],"year":["2013"]},"cols":{"kind":{"facet":"kind","value":"Trial","tip":"Landmark and current trials that define the standard of care."},"cancers":[{"label":"Langerhans cell histiocytosis","href":"/cancers/langerhans-cell-histiocytosis/","tip":"Langerhans cell histiocytosis is a disorder in which a small group of immune cells with a faulty growth signal (most often a BRAF mutation) pile up in bone, skin, pituitary or organs. It ranges from a single bone lesion that heals after biopsy to a life-threatening disease of infants. A year of gentle chemotherapy cures most children, and BRAF or MEK inhibitors rescue those with resistant disease."},{"label":"Multisystem Langerhans cell histiocytosis","href":"/cancers/lch-multisystem/","tip":"Multisystem Langerhans cell histiocytosis is the severe form of this rare histiocytosis, in which the abnormal cells involve several organs at once, most dangerously the liver, spleen and bone marrow of infants. It is treated with a year of vinblastine and prednisone, with stronger drugs or BRAF-targeted tablets for children who do not respond quickly; survival is now high but late effects remain."},{"label":"Single-system Langerhans cell histiocytosis","href":"/cancers/lch-single-system/","tip":"Single-system Langerhans cell histiocytosis is the milder form of this rare histiocytosis, in which the abnormal immune cells affect only one organ, usually a bone or the skin, in a child or young adult. Single bone lesions often heal after biopsy or curettage, skin disease may fade by itself, and gentle vinblastine and prednisone is kept for multiple bone lesions or lesions near the brain."}],"year":{"facet":"year","value":"2013"},"tags":[{"label":"histiocytosis","href":"/tagged/histiocytosis/","tip":"Every record tagged histiocytosis."}]},"sortKeys":{"year":2013}},{"id":"rejuv-paed-uk-long-term-follow-up","name":"Long-term follow-up in the United Kingdom: what a survivor is actually offered","tldr":"Britain sorts survivors into three levels of follow-up by how intensive their treatment was: a postal or telephone review at one end, a specialist late-effects clinic at the other. A Scottish cohort applied the levels retrospectively and found they worked: late effects affected 11.6 per cent of level one survivors and 65.2 per cent of level three.","route":"/technologies/rejuv-paed-uk-long-term-follow-up/","status":"standard-of-care","kind":"technology","schematic":{"id":"rejuv-paed-uk-long-term-follow-up","sections":["rejuvenation","supportive-care"]},"facets":{"kind":["Technology"],"cancers":["Childhood cancers","Acute lymphoblastic leukaemia","Hodgkin lymphoma","Medulloblastoma","Wilms tumour"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Technology","tip":"Every way we see, measure, or attack a tumour, explained."},"cancers":[{"label":"Childhood cancers","href":"/cancers/childhood-cancers/","tip":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available."},{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."},{"label":"Hodgkin lymphoma","href":"/cancers/hodgkin-lymphoma/","tip":"Hodgkin lymphoma is one of the most curable cancers, where the goal is now to cure with less toxicity, using brentuximab and, from 2026, first-line nivolumab."},{"label":"Medulloblastoma","href":"/cancers/medulloblastoma/","tip":"Medulloblastoma is the most common malignant childhood brain tumour, arising in the cerebellum. Surgery, radiation to the whole brain and spine, and chemotherapy cure about 70%, at a heavy cost to thinking and growth; treatment is now being tailored to four molecular subgroups so that the low-risk children get less."}],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."}]},"sortKeys":{"year":0}},{"id":"neuroblastoma-low-risk","name":"Low-risk neuroblastoma (INRG very low and low risk, including stage MS)","tldr":"Low-risk neuroblastoma is the form found in infants and young children whose tumour has not spread beyond its site or, in the special stage MS pattern, has spread only to the liver, skin and a little marrow. Many of these tumours shrink and disappear on their own, so treatment is surgery, or simply watching, and almost every child survives.","route":"/cancers/neuroblastoma-low-risk/","kind":"cancer","cancerIcon":"neuroblastoma-low-risk","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"subtype-page","href":"/tagged/subtype-page/","tip":"Every record tagged subtype-page."}]},"sortKeys":{"year":0}},{"id":"medulloblastoma","name":"Medulloblastoma","tldr":"Medulloblastoma is the most common malignant childhood brain tumour, arising in the cerebellum. Surgery, radiation to the whole brain and spine, and chemotherapy cure about 70%, at a heavy cost to thinking and growth; treatment is now being tailored to four molecular subgroups so that the low-risk children get less.","route":"/cancers/medulloblastoma/","kind":"cancer","cancerIcon":"medulloblastoma","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"cns","href":"/tagged/cns/","tip":"Every record tagged cns."}]},"sortKeys":{"year":0}},{"id":"rejuv-paed-neurocognitive","name":"Memory, attention and learning after treatment of a childhood cancer","tldr":"The commonest pattern after cranial radiotherapy in a young child is not forgetting what was learned but learning more slowly than other children, so the gap widens with every year at school. In 44 children treated for medulloblastoma the measured loss was 2.55 IQ points a year, and raw scores were still rising: they were gaining skills, just more slowly than the test expected for their age.","route":"/technologies/rejuv-paed-neurocognitive/","status":"established","kind":"technology","schematic":{"id":"rejuv-paed-neurocognitive","sections":["rejuvenation","supportive-care"]},"facets":{"kind":["Technology"],"cancers":["Childhood cancers","Medulloblastoma","Acute lymphoblastic leukaemia","Paediatric low-grade glioma","Paediatric high-grade glioma","Acute myeloid leukaemia in children"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Technology","tip":"Every way we see, measure, or attack a tumour, explained."},"cancers":[{"label":"Childhood cancers","href":"/cancers/childhood-cancers/","tip":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available."},{"label":"Medulloblastoma","href":"/cancers/medulloblastoma/","tip":"Medulloblastoma is the most common malignant childhood brain tumour, arising in the cerebellum. Surgery, radiation to the whole brain and spine, and chemotherapy cure about 70%, at a heavy cost to thinking and growth; treatment is now being tailored to four molecular subgroups so that the low-risk children get less."},{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."},{"label":"Paediatric low-grade glioma","href":"/cancers/paediatric-low-grade-glioma/","tip":"Paediatric low-grade gliomas are slow-growing brain tumours driven almost always by a single overactive signal, the MAPK pathway, most often through a BRAF gene change. Because the switch is known, pills that block it (dabrafenib with trametinib, and tovorafenib) now shrink tumours far more often than chemotherapy, and children are increasingly spared radiation to the developing brain."}],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."}]},"sortKeys":{"year":0}},{"id":"michael-taylor","name":"Michael D. Taylor","tldr":"Neurosurgeon-scientist who defined the four molecular subgroups of medulloblastoma that now guide therapy.","route":"/people/michael-taylor/","kind":"person","logo":"/portraits/michael-taylor.jpg","round":true,"avatar":"person","sub":"Professor of Pediatrics and Neurosurgery, Baylor College of Medicine and Texas Children's Hospital · Texas Children's Cancer and Hematology Center","facets":{"kind":["Person"],"cancers":["Medulloblastoma"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[{"label":"Medulloblastoma","href":"/cancers/medulloblastoma/","tip":"Medulloblastoma is the most common malignant childhood brain tumour, arising in the cerebellum. Surgery, radiation to the whole brain and spine, and chemotherapy cure about 70%, at a heavy cost to thinking and growth; treatment is now being tailored to four molecular subgroups so that the low-risk children get less."}],"tags":[{"label":"medulloblastoma","href":"/tagged/medulloblastoma/","tip":"Every record tagged medulloblastoma."},{"label":"genomics","href":"/tagged/genomics/","tip":"Every record tagged genomics."}]},"sortKeys":{"year":0}},{"id":"michelle-monje","name":"Michelle Monje","tldr":"Founded cancer neuroscience and led the GD2 CAR-T trial that produced the first regressions of diffuse midline glioma.","route":"/people/michelle-monje/","kind":"person","logo":"/portraits/michelle-monje.jpg","round":true,"avatar":"person","sub":"Professor of Neurology and Neurological Sciences, Stanford University; HHMI Investigator · Stanford Health Care / Stanford Cancer Institute","facets":{"kind":["Person"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[],"tags":[{"label":"glioma","href":"/tagged/glioma/","tip":"Every record tagged glioma."},{"label":"car-t","href":"/tagged/car-t/","tip":"Every record tagged car-t."},{"label":"cancer-neuroscience","href":"/tagged/cancer-neuroscience/","tip":"Every record tagged cancer-neuroscience."}]},"sortKeys":{"year":0}},{"id":"lch-multisystem","name":"Multisystem Langerhans cell histiocytosis (with or without risk-organ involvement)","tldr":"Multisystem Langerhans cell histiocytosis is the severe form of this rare histiocytosis, in which the abnormal cells involve several organs at once, most dangerously the liver, spleen and bone marrow of infants. It is treated with a year of vinblastine and prednisone, with stronger drugs or BRAF-targeted tablets for children who do not respond quickly; survival is now high but late effects remain.","route":"/cancers/lch-multisystem/","kind":"cancer","cancerIcon":"lch-multisystem","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"subtype-page","href":"/tagged/subtype-page/","tip":"Every record tagged subtype-page."}]},"sortKeys":{"year":0}},{"id":"mycn-amp","name":"MYCN amplification","tldr":"MYCN amplification, more than four times the normal copy number of the gene on FISH, marks the most aggressive fifth of neuroblastomas and puts a child in the high-risk group whatever their age or stage. No drug targets it; it decides how much treatment is given.","route":"/biomarkers/mycn-amp/","kind":"biomarker","target":{"id":"mycn","name":"MYCN (N-myc)","targetClass":"transcription","tldr":"MYCN is a growth-driving gene that some neuroblastomas copy many times over; that amplification is one of the strongest signs the tumour is aggressive and sets the intensity of treatment."},"sub":"MYCN","facets":{"kind":["Biomarker"],"cancers":["Neuroblastoma","High-risk neuroblastoma","Intermediate-risk neuroblastoma"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Biomarker","tip":"The readouts a pathology report gives (PD-L1 CPS, HER2 IHC 3+, MSI-high), each under its gene or protein, with the thresholds approvals use and the tests that measure them."},"cancers":[{"label":"Neuroblastoma","href":"/cancers/neuroblastoma/","tip":"Neuroblastoma is a childhood nerve-cell cancer where anti-GD2 antibodies and, recently, GD2 CAR-T have improved survival in high-risk disease."},{"label":"High-risk neuroblastoma","href":"/cancers/neuroblastoma-high-risk/","tip":"High-risk neuroblastoma has spread widely in a child over 18 months old or carries extra copies of the MYCN gene. Treatment lasts about 18 months and uses every tool: chemotherapy, surgery, high-dose chemotherapy with stem cell rescue, radiotherapy, and the anti-GD2 antibody dinutuximab, which raised survival in ANBL0032; eflornithine, given afterwards, was approved in 2023 to lower relapse."},{"label":"Intermediate-risk neuroblastoma","href":"/cancers/neuroblastoma-intermediate-risk/","tip":"Intermediate-risk neuroblastoma sits between the tumours that go away on their own and the high-risk disease that needs everything. A few cycles of moderate chemotherapy followed by surgery cure most children, and trials have spent twenty years showing how few cycles are enough."}],"tags":[{"label":"biomarker","href":"/tagged/biomarker/","tip":"Every record tagged biomarker."},{"label":"no-approval","href":"/tagged/no-approval/","tip":"Every record tagged no-approval."}]},"sortKeys":{"year":0}},{"id":"nai-kong-cheung","name":"Nai-Kong V. Cheung","tldr":"Developed the anti-GD2 antibodies 3F8 and naxitamab that treat relapsed neuroblastoma.","route":"/people/nai-kong-cheung/","kind":"person","round":true,"avatar":"person","sub":"Enid A. Haupt Chair in Pediatric Oncology, Memorial Sloan Kettering Cancer Center · Memorial Sloan Kettering Cancer Center","facets":{"kind":["Person"],"cancers":["Neuroblastoma"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[{"label":"Neuroblastoma","href":"/cancers/neuroblastoma/","tip":"Neuroblastoma is a childhood nerve-cell cancer where anti-GD2 antibodies and, recently, GD2 CAR-T have improved survival in high-risk disease."}],"tags":[{"label":"neuroblastoma","href":"/tagged/neuroblastoma/","tip":"Every record tagged neuroblastoma."},{"label":"immunotherapy","href":"/tagged/immunotherapy/","tip":"Every record tagged immunotherapy."}]},"sortKeys":{"year":0}},{"id":"pediatric-match","name":"NCI-COG Pediatric MATCH (APEC1621)","tldr":"Pediatric MATCH was the first nationwide precision-medicine trial for children: every child with a relapsed solid tumour could have their tumour sequenced and, if a matching drug existed, join a trial arm for it. It proved the plumbing works, even though most single drugs given alone did little.","route":"/trials/pediatric-match/","status":"active","kind":"trial","schematic":{"id":"cgp","sections":["diagnostics"]},"sub":"NCT03155620","facets":{"kind":["Trial"],"cancers":[],"year":["2022"]},"cols":{"kind":{"facet":"kind","value":"Trial","tip":"Landmark and current trials that define the standard of care."},"cancers":[],"year":{"facet":"year","value":"2022"},"tags":[{"label":"precision-medicine","href":"/tagged/precision-medicine/","tip":"Every record tagged precision-medicine."}]},"sortKeys":{"year":2022}},{"id":"neuroblastoma","name":"Neuroblastoma (paediatric)","tldr":"Neuroblastoma is a childhood nerve-cell cancer where anti-GD2 antibodies and, recently, GD2 CAR-T have improved survival in high-risk disease.","route":"/cancers/neuroblastoma/","kind":"cancer","cancerIcon":"neuroblastoma","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[]},"sortKeys":{"year":0}},{"id":"nut-carcinoma","name":"NUT carcinoma (midline carcinoma with NUTM1 rearrangement)","tldr":"NUT carcinoma is a fast-growing cancer of the midline of the body driven by a single fused gene, BRD4-NUTM1, that locks cells in an immature state. Chemotherapy and surgery rarely control it for long, but drugs that block the BET proteins the fusion depends on have produced responses and are the focus of trials.","route":"/cancers/nut-carcinoma/","kind":"cancer","cancerIcon":"nut-carcinoma","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"rare","href":"/tagged/rare/","tip":"Every record tagged rare."},{"label":"head-and-neck","href":"/tagged/head-and-neck/","tip":"Every record tagged head-and-neck."}]},"sortKeys":{"year":0}},{"id":"osteosarcoma","name":"Osteosarcoma","tldr":"Osteosarcoma is the most common bone cancer, mostly in teenagers. Chemotherapy plus surgery cures about two-thirds when it has not spread; because no new drug has beaten that chemotherapy in a large trial in 30 years, the next gains are being sought in cellular therapy against GD2, HER2 and B7-H3.","route":"/cancers/osteosarcoma/","kind":"cancer","cancerIcon":"osteosarcoma","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"sarcoma","href":"/tagged/sarcoma/","tip":"Every record tagged sarcoma."},{"label":"aya","href":"/tagged/aya/","tip":"Every record tagged aya."}]},"sortKeys":{"year":0}},{"id":"paediatric-high-grade-glioma","name":"Paediatric high-grade glioma (excluding diffuse midline glioma)","tldr":"High-grade gliomas in children look like adult glioblastoma under the microscope but are driven by different genes, so they are now classified separately. Surgery and radiotherapy remain the mainstay and chemotherapy adds little; the real gains are in small subsets with a targetable gene change, such as BRAF V600E tumours and the fusion-driven tumours of infants.","route":"/cancers/paediatric-high-grade-glioma/","kind":"cancer","cancerIcon":"paediatric-high-grade-glioma","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"subtype-page","href":"/tagged/subtype-page/","tip":"Every record tagged subtype-page."},{"label":"cns","href":"/tagged/cns/","tip":"Every record tagged cns."}]},"sortKeys":{"year":0}},{"id":"paediatric-low-grade-glioma","name":"Paediatric low-grade glioma","tldr":"Paediatric low-grade gliomas are slow-growing brain tumours driven almost always by a single overactive signal, the MAPK pathway, most often through a BRAF gene change. Because the switch is known, pills that block it (dabrafenib with trametinib, and tovorafenib) now shrink tumours far more often than chemotherapy, and children are increasingly spared radiation to the developing brain.","route":"/cancers/paediatric-low-grade-glioma/","kind":"cancer","cancerIcon":"paediatric-low-grade-glioma","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"cns","href":"/tagged/cns/","tip":"Every record tagged cns."}]},"sortKeys":{"year":0}},{"id":"pancaresurfup","name":"PanCareSurFup and the European survivor cohorts","tldr":"Europe's answer to the American cohorts: thirteen data providers in twelve countries pooled their records to build what its own authors call the largest cohort of children with cancer to date, 83,333 five-year survivors, so that second cancers and heart disease could be counted on a continent that keeps its data in national pieces.","route":"/collections/pancaresurfup/","kind":"collection","logo":"https://www.google.com/s2/favicons?domain=pancare.eu&sz=128","avatar":"org","sub":"PanCare network, with the PanCareSurFup data centre in Mainz","facets":{"kind":["Collection"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Collection","tip":"The open databases and registries the field runs on."},"cancers":[],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."},{"label":"cohort","href":"/tagged/cohort/","tip":"Every record tagged cohort."},{"label":"europe","href":"/tagged/europe/","tip":"Every record tagged europe."}]},"sortKeys":{"year":0}},{"id":"all-ph-like","name":"Philadelphia chromosome-like acute lymphoblastic leukaemia (Ph-like or BCR::ABL1-like ALL)","tldr":"Ph-like leukaemia behaves like Philadelphia chromosome-positive leukaemia, with the same kind of overactive growth signalling, but lacks the BCR::ABL1 gene itself. It is caused by a scattered set of gene fusions and mutations, many of them blockable by existing kinase pills such as dasatinib or ruxolitinib, and it is now screened for at diagnosis so those drugs can be tried.","route":"/cancers/all-ph-like/","kind":"cancer","cancerIcon":"all-ph-like","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"subtype-page","href":"/tagged/subtype-page/","tip":"Every record tagged subtype-page."}]},"sortKeys":{"year":0}},{"id":"all-paediatric-ph-positive","name":"Philadelphia chromosome-positive acute lymphoblastic leukaemia in children (Ph-positive ALL)","tldr":"Philadelphia chromosome-positive leukaemia carries the same faulty BCR::ABL1 gene as chronic myeloid leukaemia. Until 2000 most children with it needed a bone marrow transplant; adding the targeted pill imatinib to chemotherapy, and then dasatinib, means most are now cured without one.","route":"/cancers/all-paediatric-ph-positive/","kind":"cancer","cancerIcon":"all-paediatric-ph-positive","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"subtype-page","href":"/tagged/subtype-page/","tip":"Every record tagged subtype-page."}]},"sortKeys":{"year":0}},{"id":"race-for-children-act","name":"RACE for Children Act","tldr":"A US law that makes drug companies test new targeted cancer drugs in children whenever the drug's target matters in a childhood cancer, instead of letting them skip children because their cancers are rare.","route":"/terms/race-for-children-act/","kind":"term","molecule":"tovorafenib","modality":"Small-molecule type II RAF inhibitor","facets":{"kind":["Term"],"cancers":["Paediatric low-grade glioma","Diffuse midline glioma, H3 K27-altered","Neuroblastoma"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Term","tip":"Glossary with plain-English TL;DRs and Wikipedia links."},"cancers":[{"label":"Paediatric low-grade glioma","href":"/cancers/paediatric-low-grade-glioma/","tip":"Paediatric low-grade gliomas are slow-growing brain tumours driven almost always by a single overactive signal, the MAPK pathway, most often through a BRAF gene change. Because the switch is known, pills that block it (dabrafenib with trametinib, and tovorafenib) now shrink tumours far more often than chemotherapy, and children are increasingly spared radiation to the developing brain."},{"label":"Diffuse midline glioma, H3 K27-altered","href":"/cancers/dipg-dmg/","tip":"Diffuse midline glioma grows through the brainstem and cannot be removed surgically. A single change in a histone protein (H3 K27M) rewires how the tumour reads its DNA. Radiotherapy was long the only help; in 2025 the first drug aimed at this tumour, dordaviprone (ONC201), was approved after durable shrinkage in some patients, and GD2 CAR-T cells have produced striking early responses."},{"label":"Neuroblastoma","href":"/cancers/neuroblastoma/","tip":"Neuroblastoma is a childhood nerve-cell cancer where anti-GD2 antibodies and, recently, GD2 CAR-T have improved survival in high-risk disease."}],"tags":[{"label":"regulatory","href":"/tagged/regulatory/","tip":"Every record tagged regulatory."}]},"sortKeys":{"year":0}},{"id":"rare-childhood-cancers","name":"Rare cancers of childhood (NCI PDQ umbrella)","tldr":"Some childhood cancers are so rare that no single hospital sees enough to learn from. The NCI groups them together: heart tumours, airway papillomas, cancers of the thyroid, adrenal, nose and throat, melanoma and carcinomas more typical of adults. The answer has been international registries and expert networks that pool every case, so treatment guidance exists even without trials.","route":"/cancers/rare-childhood-cancers/","kind":"cancer","cancerIcon":"rare-childhood-cancers","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"umbrella","href":"/tagged/umbrella/","tip":"Every record tagged umbrella."}]},"sortKeys":{"year":0}},{"id":"all-paediatric-relapsed","name":"Relapsed and refractory acute lymphoblastic leukaemia in children","tldr":"When childhood leukaemia comes back, chemotherapy alone cures fewer than half. Three immune treatments changed this: blinatumomab, which links the child's T-cells to leukaemia cells and beat chemotherapy in two trials; tisagenlecleucel, the first approved CAR T-cell therapy, which put over eight in ten pretreated children into remission; and the antibody-drug conjugate inotuzumab ozogamicin.","route":"/cancers/all-paediatric-relapsed/","kind":"cancer","cancerIcon":"all-paediatric-relapsed","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"subtype-page","href":"/tagged/subtype-page/","tip":"Every record tagged subtype-page."}]},"sortKeys":{"year":0}},{"id":"retinoblastoma","name":"Retinoblastoma","tldr":"An eye cancer of infants caused by loss of the RB1 gene, the first tumour-suppressor gene ever found. In rich countries almost every child survives and most eyes are saved by chemotherapy delivered through the eye's artery; in low-income countries, where most cases occur, survival depends on finding it early, and that is the global gap.","route":"/cancers/retinoblastoma/","kind":"cancer","cancerIcon":"retinoblastoma","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"eye","href":"/tagged/eye/","tip":"Every record tagged eye."},{"label":"hereditary","href":"/tagged/hereditary/","tip":"Every record tagged hereditary."}]},"sortKeys":{"year":0}},{"id":"rhabdomyosarcoma","name":"Rhabdomyosarcoma","tldr":"A childhood soft-tissue sarcoma, a cancer of muscle-like cells found anywhere from the eye socket to the bladder. Most children are cured with chemotherapy, surgery and radiation, and a fusion gene (PAX-FOXO1) now decides how intensively to treat.","route":"/cancers/rhabdomyosarcoma/","kind":"cancer","cancerIcon":"rhabdomyosarcoma","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"sarcoma","href":"/tagged/sarcoma/","tip":"Every record tagged sarcoma."}]},"sortKeys":{"year":0}},{"id":"rob-pieters","name":"Rob Pieters","tldr":"Dutch paediatric oncologist who led the international Interfant trials for babies with leukaemia and helped create the Princess Máxima Center, which brought all Dutch childhood cancer care into one hospital.","route":"/people/rob-pieters/","kind":"person","round":true,"avatar":"person","sub":"Paediatric oncologist and Chief Medical Officer, Princess Máxima Center for Pediatric Oncology · Princess Máxima Center for Pediatric Oncology","facets":{"kind":["Person"],"cancers":["Infant acute lymphoblastic leukaemia","Acute lymphoblastic leukaemia","Childhood cancers"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[{"label":"Infant acute lymphoblastic leukaemia","href":"/cancers/all-infant/","tip":"Leukaemia diagnosed in the first year of life is a different disease from leukaemia in older children: most cases carry a broken KMT2A gene and respond poorly to chemotherapy, and fewer than half of infants were cured for twenty years. One course of the immune drug blinatumomab after induction raised two-year disease-free survival from about half to over 80 percent in a pilot study."},{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."},{"label":"Childhood cancers","href":"/cancers/childhood-cancers/","tip":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available."}],"tags":[{"label":"leukaemia","href":"/tagged/leukaemia/","tip":"Every record tagged leukaemia."},{"label":"trialist","href":"/tagged/trialist/","tip":"Every record tagged trialist."}]},"sortKeys":{"year":0}},{"id":"robbie-majzner","name":"Robbie G. Majzner","tldr":"Led the GD2 CAR-T trial in diffuse midline glioma and defined antigen density as a barrier to CAR-T in solid tumours.","route":"/people/robbie-majzner/","kind":"person","round":true,"avatar":"person","sub":"Director of Solid Tumor Immunotherapy, Department of Oncology, St. Jude Children's Research Hospital · St. Jude Children's Research Hospital","facets":{"kind":["Person"],"cancers":["Neuroblastoma","Osteosarcoma"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[{"label":"Neuroblastoma","href":"/cancers/neuroblastoma/","tip":"Neuroblastoma is a childhood nerve-cell cancer where anti-GD2 antibodies and, recently, GD2 CAR-T have improved survival in high-risk disease."},{"label":"Osteosarcoma","href":"/cancers/osteosarcoma/","tip":"Osteosarcoma is the most common bone cancer, mostly in teenagers. Chemotherapy plus surgery cures about two-thirds when it has not spread; because no new drug has beaten that chemotherapy in a large trial in 30 years, the next gains are being sought in cellular therapy against GD2, HER2 and B7-H3."}],"tags":[{"label":"cell-therapy","href":"/tagged/cell-therapy/","tip":"Every record tagged cell-therapy."},{"label":"car-t","href":"/tagged/car-t/","tip":"Every record tagged car-t."}]},"sortKeys":{"year":0}},{"id":"ruth-ladenstein","name":"Ruth Ladenstein","tldr":"Chaired the SIOPEN HR-NBL1 trial, the largest neuroblastoma trial ever, which set European standards for high-risk disease.","route":"/people/ruth-ladenstein/","kind":"person","round":true,"avatar":"person","sub":"Professor of Paediatrics, St. Anna Children's Hospital and Children's Cancer Research Institute, Vienna · St. Anna Children's Hospital and Children's Cancer Research Institute (CCRI)","facets":{"kind":["Person"],"cancers":["Neuroblastoma"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[{"label":"Neuroblastoma","href":"/cancers/neuroblastoma/","tip":"Neuroblastoma is a childhood nerve-cell cancer where anti-GD2 antibodies and, recently, GD2 CAR-T have improved survival in high-risk disease."}],"tags":[{"label":"neuroblastoma","href":"/tagged/neuroblastoma/","tip":"Every record tagged neuroblastoma."},{"label":"cooperative-group","href":"/tagged/cooperative-group/","tip":"Every record tagged cooperative-group."}]},"sortKeys":{"year":0}},{"id":"sarah-leary","name":"Sarah E. S. Leary","tldr":"Seattle paediatric neuro-oncologist who led COG ACNS0332, the trial that found adding carboplatin to radiotherapy improves survival in children with high-risk group 3 medulloblastoma but isotretinoin does not help.","route":"/people/sarah-leary/","kind":"person","round":true,"avatar":"person","sub":"Paediatric neuro-oncologist, Seattle Children's Hospital · Seattle Children's Hospital","facets":{"kind":["Person"],"cancers":["Group 3 and group 4 medulloblastoma","Medulloblastoma","Childhood cancers"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[{"label":"Group 3 and group 4 medulloblastoma","href":"/cancers/medulloblastoma-group-3-4/","tip":"Group 3 and group 4 medulloblastoma are the two commonest forms of this cerebellar brain tumour and the ones without a druggable driver. Group 3 strikes young children, often with extra copies of MYC and spread through the spinal fluid; group 4 affects older boys. Both get surgery, craniospinal radiotherapy and chemotherapy; trials showed the radiation dose cannot be cut for young children."},{"label":"Medulloblastoma","href":"/cancers/medulloblastoma/","tip":"Medulloblastoma is the most common malignant childhood brain tumour, arising in the cerebellum. Surgery, radiation to the whole brain and spine, and chemotherapy cure about 70%, at a heavy cost to thinking and growth; treatment is now being tailored to four molecular subgroups so that the low-risk children get less."},{"label":"Childhood cancers","href":"/cancers/childhood-cancers/","tip":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available."}],"tags":[{"label":"brain","href":"/tagged/brain/","tip":"Every record tagged brain."},{"label":"trialist","href":"/tagged/trialist/","tip":"Every record tagged trialist."}]},"sortKeys":{"year":0}},{"id":"rejuv-paed-second-cancers","name":"Second cancers after childhood cancer: the risk by treatment, and why it is falling","tldr":"The largest late risk a childhood cancer survivor carries. Thirty years after diagnosis, 20.5 per cent of survivors treated in the 1970s and early 1980s had developed a subsequent neoplasm. The fifteen-year risk of a second malignancy has since fallen from 2.1 to 1.3 per cent across treatment decades, and the fall tracks the radiotherapy taken out.","route":"/technologies/rejuv-paed-second-cancers/","status":"established","kind":"technology","schematic":{"id":"rejuv-paed-second-cancers","sections":["rejuvenation","supportive-care","early-detection"]},"facets":{"kind":["Technology"],"cancers":["Childhood cancers","Hodgkin lymphoma","Ewing sarcoma","Retinoblastoma","Acute lymphoblastic leukaemia","Wilms tumour","Medulloblastoma","Thyroid cancer"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Technology","tip":"Every way we see, measure, or attack a tumour, explained."},"cancers":[{"label":"Childhood cancers","href":"/cancers/childhood-cancers/","tip":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available."},{"label":"Hodgkin lymphoma","href":"/cancers/hodgkin-lymphoma/","tip":"Hodgkin lymphoma is one of the most curable cancers, where the goal is now to cure with less toxicity, using brentuximab and, from 2026, first-line nivolumab."},{"label":"Ewing sarcoma","href":"/cancers/ewing-sarcoma/","tip":"Ewing sarcoma is a bone and soft-tissue cancer of teenagers driven by a single fusion gene, EWSR1-FLI1. Intensive chemotherapy with surgery or radiation cures most localised cases; disease that has spread at diagnosis, and relapse, remain hard to treat, and no drug against the fusion protein itself has yet succeeded."},{"label":"Retinoblastoma","href":"/cancers/retinoblastoma/","tip":"An eye cancer of infants caused by loss of the RB1 gene, the first tumour-suppressor gene ever found. In rich countries almost every child survives and most eyes are saved by chemotherapy delivered through the eye's artery; in low-income countries, where most cases occur, survival depends on finding it early, and that is the global gap."}],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."}]},"sortKeys":{"year":0}},{"id":"rejuv-ayac-services","name":"Services built for teenagers and young adults, and what the national evaluation found","tldr":"England built specialist units for 13 to 24 year olds and then evaluated them nationally, which almost no health system does. The results were mixed enough that young people were asked to interpret them, and they pointed out that three years of follow-up was too short and that the study had defined specialist care by how many admissions a person had rather than how long they spent there.","route":"/technologies/rejuv-ayac-services/","status":"established","kind":"technology","schematic":{"id":"rejuv-ayac-services","sections":["rejuvenation","supportive-care"]},"facets":{"kind":["Technology"],"cancers":["Hodgkin lymphoma","Acute lymphoblastic leukaemia","Osteosarcoma","Ewing sarcoma","Testicular germ cell tumours","Childhood cancers"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Technology","tip":"Every way we see, measure, or attack a tumour, explained."},"cancers":[{"label":"Hodgkin lymphoma","href":"/cancers/hodgkin-lymphoma/","tip":"Hodgkin lymphoma is one of the most curable cancers, where the goal is now to cure with less toxicity, using brentuximab and, from 2026, first-line nivolumab."},{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."},{"label":"Osteosarcoma","href":"/cancers/osteosarcoma/","tip":"Osteosarcoma is the most common bone cancer, mostly in teenagers. Chemotherapy plus surgery cures about two-thirds when it has not spread; because no new drug has beaten that chemotherapy in a large trial in 30 years, the next gains are being sought in cellular therapy against GD2, HER2 and B7-H3."},{"label":"Ewing sarcoma","href":"/cancers/ewing-sarcoma/","tip":"Ewing sarcoma is a bone and soft-tissue cancer of teenagers driven by a single fusion gene, EWSR1-FLI1. Intensive chemotherapy with surgery or radiation cures most localised cases; disease that has spread at diagnosis, and relapse, remain hard to treat, and no drug against the fusion protein itself has yet succeeded."}],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."}]},"sortKeys":{"year":0}},{"id":"medulloblastoma-shh","name":"SHH-activated medulloblastoma","tldr":"SHH-activated medulloblastoma is driven by the sonic hedgehog growth pathway, the signal that normally tells the developing cerebellum to grow. In infants it is often cured with chemotherapy alone and no radiotherapy; in adults it responds for a time to hedgehog-blocking pills such as vismodegib; and when it carries a TP53 mutation in an older child, often inherited, it resists everything.","route":"/cancers/medulloblastoma-shh/","kind":"cancer","cancerIcon":"medulloblastoma-shh","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"subtype-page","href":"/tagged/subtype-page/","tip":"Every record tagged subtype-page."},{"label":"cns","href":"/tagged/cns/","tip":"Every record tagged cns."}]},"sortKeys":{"year":0}},{"id":"lch-single-system","name":"Single-system Langerhans cell histiocytosis (bone, skin or one other organ)","tldr":"Single-system Langerhans cell histiocytosis is the milder form of this rare histiocytosis, in which the abnormal immune cells affect only one organ, usually a bone or the skin, in a child or young adult. Single bone lesions often heal after biopsy or curettage, skin disease may fade by itself, and gentle vinblastine and prednisone is kept for multiple bone lesions or lesions near the brain.","route":"/cancers/lch-single-system/","kind":"cancer","cancerIcon":"lch-single-system","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"subtype-page","href":"/tagged/subtype-page/","tip":"Every record tagged subtype-page."}]},"sortKeys":{"year":0}},{"id":"sjlife","name":"St Jude Lifetime Cohort Study (SJLIFE)","tldr":"The study that brought survivors back to a hospital and tested them, rather than asking them what was wrong. It found that most of what it found had not been diagnosed: by age 45, on clinical testing, 95.5 per cent of survivors had a chronic health condition and 80.5 per cent had a serious, disabling or life-threatening one.","route":"/collections/sjlife/","kind":"collection","logo":"https://www.google.com/s2/favicons?domain=sjlife.stjude.org&sz=128","avatar":"org","sub":"St. Jude Children's Research Hospital","facets":{"kind":["Collection"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Collection","tip":"The open databases and registries the field runs on."},"cancers":[],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."},{"label":"cohort","href":"/tagged/cohort/","tip":"Every record tagged cohort."}]},"sortKeys":{"year":0}},{"id":"st-baldricks","name":"St. Baldrick's Foundation","tldr":"A head-shaving fundraiser that became the largest charitable funder of children's cancer research in the US, underwriting much of the cooperative-group trial infrastructure that treats most American children with cancer.","route":"/collections/st-baldricks/","kind":"collection","logo":"/logos/st-baldricks.png","avatar":"org","sub":"St. Baldrick's Foundation","facets":{"kind":["Collection"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Collection","tip":"The open databases and registries the field runs on."},"cancers":[],"tags":[{"label":"patient-org","href":"/tagged/patient-org/","tip":"Every record tagged patient-org."}]},"sortKeys":{"year":0}},{"id":"all-paediatric-standard-risk","name":"Standard-risk B-cell acute lymphoblastic leukaemia in children","tldr":"Standard-risk acute lymphoblastic leukaemia is the commonest and most curable childhood cancer: a child aged one to nine with a modest white cell count and favourable genetics. Two to three years of chemotherapy cures about nine in ten, and adding the immune drug blinatumomab to the chemotherapy in the AALL1731 trial cut relapses further.","route":"/cancers/all-paediatric-standard-risk/","kind":"cancer","cancerIcon":"all-paediatric-standard-risk","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"subtype-page","href":"/tagged/subtype-page/","tip":"Every record tagged subtype-page."}]},"sortKeys":{"year":0}},{"id":"stefan-bielack","name":"Stefan S. Bielack","tldr":"Leads the COSS group and co-led EURAMOS-1, the largest osteosarcoma trial ever run.","route":"/people/stefan-bielack/","kind":"person","round":true,"avatar":"person","sub":"Head of Paediatric Oncology, Klinikum Stuttgart Olgahospital; Chair, Cooperative Osteosarcoma Study Group · Klinikum Stuttgart / Olgahospital","facets":{"kind":["Person"],"cancers":["Osteosarcoma"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[{"label":"Osteosarcoma","href":"/cancers/osteosarcoma/","tip":"Osteosarcoma is the most common bone cancer, mostly in teenagers. Chemotherapy plus surgery cures about two-thirds when it has not spread; because no new drug has beaten that chemotherapy in a large trial in 30 years, the next gains are being sought in cellular therapy against GD2, HER2 and B7-H3."}],"tags":[{"label":"osteosarcoma","href":"/tagged/osteosarcoma/","tip":"Every record tagged osteosarcoma."},{"label":"cooperative-group","href":"/tagged/cooperative-group/","tip":"Every record tagged cooperative-group."}]},"sortKeys":{"year":0}},{"id":"stephan-grupp","name":"Stephan A. Grupp","tldr":"Treated the first child with CAR-T cells and led ELIANA, the trial behind the first CAR-T approval.","route":"/people/stephan-grupp/","kind":"person","round":true,"avatar":"person","sub":"Chief, Cellular Therapy and Transplant Section, Children's Hospital of Philadelphia; Professor of Pediatrics, University of Pennsylvania · Children's Hospital of Philadelphia","facets":{"kind":["Person"],"cancers":["Acute lymphoblastic leukaemia"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."}],"tags":[{"label":"cell-therapy","href":"/tagged/cell-therapy/","tip":"Every record tagged cell-therapy."},{"label":"car-t","href":"/tagged/car-t/","tip":"Every record tagged car-t."}]},"sortKeys":{"year":0}},{"id":"stephen-hunger","name":"Stephen P. Hunger","tldr":"Chaired the COG leukaemia committee through the era that pushed childhood ALL cure rates above 90%.","route":"/people/stephen-hunger/","kind":"person","round":true,"avatar":"person","sub":"Chief, Division of Oncology, Children's Hospital of Philadelphia; Jeffrey E. Perelman Distinguished Chair in Pediatrics · Children's Hospital of Philadelphia","facets":{"kind":["Person"],"cancers":["Acute lymphoblastic leukaemia"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."}],"tags":[{"label":"all","href":"/tagged/all/","tip":"Every record tagged all."},{"label":"cooperative-group","href":"/tagged/cooperative-group/","tip":"Every record tagged cooperative-group."}]},"sortKeys":{"year":0}},{"id":"sumit-gupta","name":"Sumit Gupta","tldr":"Led COG AALL1731, which showed adding blinatumomab to chemotherapy improves survival for children with standard-risk leukaemia.","route":"/people/sumit-gupta/","kind":"person","round":true,"avatar":"person","sub":"Staff Oncologist and Senior Scientist, The Hospital for Sick Children (SickKids), Toronto · The Hospital for Sick Children (SickKids)","facets":{"kind":["Person"],"cancers":["Acute lymphoblastic leukaemia"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."}],"tags":[{"label":"all","href":"/tagged/all/","tip":"Every record tagged all."},{"label":"immunotherapy","href":"/tagged/immunotherapy/","tip":"Every record tagged immunotherapy."}]},"sortKeys":{"year":0}},{"id":"tadpole","name":"TADPOLE (CDRB436G2201)","tldr":"The first randomised trial to show that a targeted drug pair beats chemotherapy in children with a brain tumour. Children whose low-grade glioma carries a BRAF V600 mutation had far more tumour shrinkage and a much longer time before progression on dabrafenib plus trametinib than on standard carboplatin and vincristine.","route":"/trials/tadpole/","status":"positive","kind":"trial","cancerIcon":"paediatric-low-grade-glioma","sub":"NCT02684058","facets":{"kind":["Trial"],"cancers":["Paediatric low-grade glioma"],"year":["2023"]},"cols":{"kind":{"facet":"kind","value":"Trial","tip":"Landmark and current trials that define the standard of care."},"cancers":[{"label":"Paediatric low-grade glioma","href":"/cancers/paediatric-low-grade-glioma/","tip":"Paediatric low-grade gliomas are slow-growing brain tumours driven almost always by a single overactive signal, the MAPK pathway, most often through a BRAF gene change. Because the switch is known, pills that block it (dabrafenib with trametinib, and tovorafenib) now shrink tumours far more often than chemotherapy, and children are increasingly spared radiation to the developing brain."}],"year":{"facet":"year","value":"2023"},"tags":[{"label":"cns","href":"/tagged/cns/","tip":"Every record tagged cns."}]},"sortKeys":{"year":2023}},{"id":"rejuv-paed-teeth-and-face","name":"Teeth, jaws and facial growth after treatment in childhood","tldr":"Treatment given while the teeth are forming can stop them forming. In the largest survey, survivors were three times more likely than siblings to report small teeth, three times more likely to report abnormal roots and nearly ten times more likely to report a dry mouth, and the risk was concentrated in children treated with alkylating drugs before the age of five.","route":"/technologies/rejuv-paed-teeth-and-face/","status":"established","kind":"technology","schematic":{"id":"rejuv-paed-teeth-and-face","sections":["rejuvenation","supportive-care"]},"facets":{"kind":["Technology"],"cancers":["Childhood cancers","Acute lymphoblastic leukaemia","Neuroblastoma","Rhabdomyosarcoma","Retinoblastoma","Medulloblastoma"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Technology","tip":"Every way we see, measure, or attack a tumour, explained."},"cancers":[{"label":"Childhood cancers","href":"/cancers/childhood-cancers/","tip":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available."},{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."},{"label":"Neuroblastoma","href":"/cancers/neuroblastoma/","tip":"Neuroblastoma is a childhood nerve-cell cancer where anti-GD2 antibodies and, recently, GD2 CAR-T have improved survival in high-risk disease."},{"label":"Rhabdomyosarcoma","href":"/cancers/rhabdomyosarcoma/","tip":"A childhood soft-tissue sarcoma, a cancer of muscle-like cells found anywhere from the eye socket to the bladder. Most children are cured with chemotherapy, surgery and radiation, and a fusion gene (PAX-FOXO1) now decides how intensively to treat."}],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."}]},"sortKeys":{"year":0}},{"id":"rejuv-paed-cog-ltfu-guidelines","name":"The Children's Oncology Group Long-Term Follow-Up Guidelines","tldr":"The most widely used rulebook in childhood cancer survivorship, and the one that made follow-up exposure-based rather than diagnosis-based: what you were given decides what you are screened for. It comes with Health Links, plain-language sheets written for the survivor rather than the doctor, and it is free to download.","route":"/technologies/rejuv-paed-cog-ltfu-guidelines/","status":"standard-of-care","kind":"technology","schematic":{"id":"rejuv-paed-cog-ltfu-guidelines","sections":["rejuvenation","supportive-care"]},"facets":{"kind":["Technology"],"cancers":["Childhood cancers","Acute lymphoblastic leukaemia","Hodgkin lymphoma","Neuroblastoma","Wilms tumour","Medulloblastoma"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Technology","tip":"Every way we see, measure, or attack a tumour, explained."},"cancers":[{"label":"Childhood cancers","href":"/cancers/childhood-cancers/","tip":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available."},{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."},{"label":"Hodgkin lymphoma","href":"/cancers/hodgkin-lymphoma/","tip":"Hodgkin lymphoma is one of the most curable cancers, where the goal is now to cure with less toxicity, using brentuximab and, from 2026, first-line nivolumab."},{"label":"Neuroblastoma","href":"/cancers/neuroblastoma/","tip":"Neuroblastoma is a childhood nerve-cell cancer where anti-GD2 antibodies and, recently, GD2 CAR-T have improved survival in high-risk disease."}],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."}]},"sortKeys":{"year":0}},{"id":"rejuv-paed-transition-to-adult-care","name":"The handover from children's to adult services, where follow-up falls away","tldr":"This is where survivorship care is lost. Of 8,522 adult survivors asked, 88.8 per cent had seen a doctor in the previous two years but only 17.8 per cent had received care that addressed their cancer history with risk advice or screening. Among those who should have had an echocardiogram, 28.2 per cent had; among those due a mammogram, 40.8 per cent had.","route":"/technologies/rejuv-paed-transition-to-adult-care/","status":"emerging","kind":"technology","schematic":{"id":"rejuv-paed-transition-to-adult-care","sections":["rejuvenation","supportive-care"]},"facets":{"kind":["Technology"],"cancers":["Childhood cancers","Acute lymphoblastic leukaemia","Hodgkin lymphoma","Medulloblastoma","Neuroblastoma"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Technology","tip":"Every way we see, measure, or attack a tumour, explained."},"cancers":[{"label":"Childhood cancers","href":"/cancers/childhood-cancers/","tip":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available."},{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."},{"label":"Hodgkin lymphoma","href":"/cancers/hodgkin-lymphoma/","tip":"Hodgkin lymphoma is one of the most curable cancers, where the goal is now to cure with less toxicity, using brentuximab and, from 2026, first-line nivolumab."},{"label":"Medulloblastoma","href":"/cancers/medulloblastoma/","tip":"Medulloblastoma is the most common malignant childhood brain tumour, arising in the cerebellum. Surgery, radiation to the whole brain and spine, and chemotherapy cure about 70%, at a heavy cost to thinking and growth; treatment is now being tailored to four molecular subgroups so that the low-risk children get less."}],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."}]},"sortKeys":{"year":0}},{"id":"rejuv-paed-heart","name":"The heart after anthracyclines and chest radiotherapy in childhood","tldr":"Heart damage from childhood treatment appears quietly and decades later. When 1,853 adult survivors were examined rather than asked, 7.4 per cent had cardiomyopathy and 28 per cent had valve disease, and most of it was new at that visit: nearly all of them had no symptoms. High blood pressure multiplied the risk of heart failure nineteenfold, which makes it the most treatable thing on the page.","route":"/technologies/rejuv-paed-heart/","status":"standard-of-care","kind":"technology","schematic":{"id":"rejuv-paed-heart","sections":["rejuvenation","supportive-care","chemotherapy"]},"facets":{"kind":["Technology"],"cancers":["Childhood cancers","Acute lymphoblastic leukaemia","Acute myeloid leukaemia in children","Hodgkin lymphoma","Osteosarcoma","Ewing sarcoma","Wilms tumour","Neuroblastoma"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Technology","tip":"Every way we see, measure, or attack a tumour, explained."},"cancers":[{"label":"Childhood cancers","href":"/cancers/childhood-cancers/","tip":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available."},{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."},{"label":"Acute myeloid leukaemia in children","href":"/cancers/aml-paediatric/","tip":"Acute myeloid leukaemia in children carries gene fusions rather than the mutations of ageing, is treated with four or five intensive courses of chemotherapy, and cures around two thirds of children. Adding gemtuzumab ozogamicin lowered relapse in the AAML0531 trial, and the menin inhibitor revumenib is the first targeted drug approved for the KMT2A-rearranged form common in young children."},{"label":"Hodgkin lymphoma","href":"/cancers/hodgkin-lymphoma/","tip":"Hodgkin lymphoma is one of the most curable cancers, where the goal is now to cure with less toxicity, using brentuximab and, from 2026, first-line nivolumab."}],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."}]},"sortKeys":{"year":0}},{"id":"rejuv-paed-pituitary-and-puberty","name":"The pituitary, puberty and the hypothalamic axis after cranial radiotherapy in childhood","tldr":"Radiotherapy near the base of the brain damages the gland that runs growth, puberty, the thyroid and the stress response, in that order of sensitivity. In 748 survivors treated with cranial radiotherapy, 46.5 per cent had growth hormone deficiency, 10.8 per cent sex hormone deficiency, 7.5 per cent thyroid deficiency and 4 per cent adrenal deficiency, and most of it had not been treated.","route":"/technologies/rejuv-paed-pituitary-and-puberty/","status":"standard-of-care","kind":"technology","schematic":{"id":"rejuv-paed-pituitary-and-puberty","sections":["rejuvenation","supportive-care","hormonal"]},"facets":{"kind":["Technology"],"cancers":["Childhood cancers","Medulloblastoma","Paediatric low-grade glioma","Paediatric high-grade glioma","Acute lymphoblastic leukaemia","Nasopharyngeal carcinoma"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Technology","tip":"Every way we see, measure, or attack a tumour, explained."},"cancers":[{"label":"Childhood cancers","href":"/cancers/childhood-cancers/","tip":"Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available."},{"label":"Medulloblastoma","href":"/cancers/medulloblastoma/","tip":"Medulloblastoma is the most common malignant childhood brain tumour, arising in the cerebellum. Surgery, radiation to the whole brain and spine, and chemotherapy cure about 70%, at a heavy cost to thinking and growth; treatment is now being tailored to four molecular subgroups so that the low-risk children get less."},{"label":"Paediatric low-grade glioma","href":"/cancers/paediatric-low-grade-glioma/","tip":"Paediatric low-grade gliomas are slow-growing brain tumours driven almost always by a single overactive signal, the MAPK pathway, most often through a BRAF gene change. Because the switch is known, pills that block it (dabrafenib with trametinib, and tovorafenib) now shrink tumours far more often than chemotherapy, and children are increasingly spared radiation to the developing brain."},{"label":"Paediatric high-grade glioma","href":"/cancers/paediatric-high-grade-glioma/","tip":"High-grade gliomas in children look like adult glioblastoma under the microscope but are driven by different genes, so they are now classified separately. Surgery and radiotherapy remain the mainstay and chemotherapy adds little; the real gains are in small subsets with a targetable gene change, such as BRAF V600E tumours and the fusion-driven tumours of infants."}],"tags":[{"label":"rejuvenation","href":"/tagged/rejuvenation/","tip":"Every record tagged rejuvenation."},{"label":"survivorship","href":"/tagged/survivorship/","tip":"Every record tagged survivorship."},{"label":"late-effects","href":"/tagged/late-effects/","tip":"Every record tagged late-effects."}]},"sortKeys":{"year":0}},{"id":"timothy-robinson","name":"Timothy Robinson","tldr":"Chief Executive of Nationwide Children's Hospital in Columbus, Ohio, a large paediatric health system with a major childhood cancer programme.","route":"/people/timothy-robinson/","kind":"person","round":true,"avatar":"person","sub":"Chief Executive Officer, Nationwide Children's Hospital · Nationwide Children's Hospital","facets":{"kind":["Person"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[],"tags":[{"label":"leadership","href":"/tagged/leadership/","tip":"Every record tagged leadership."},{"label":"hospital-management","href":"/tagged/hospital-management/","tip":"Every record tagged hospital-management."}]},"sortKeys":{"year":0}},{"id":"vascular-tumours","name":"Vascular tumours (angiosarcoma, epithelioid haemangioendothelioma, kaposiform haemangioendothelioma)","tldr":"Vascular tumours range from angiosarcoma, an aggressive cancer of blood vessel lining cells, to the slow-growing EHE and the infant tumour KHE. Angiosarcoma responds to paclitaxel and, in the sun-damaged scalp form, to immunotherapy; EHE and KHE depend on growth signals that the mTOR blocker sirolimus quiets, and EHE without symptoms is watched.","route":"/cancers/vascular-tumours/","kind":"cancer","cancerIcon":"vascular-tumours","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"rare","href":"/tagged/rare/","tip":"Every record tagged rare."},{"label":"sarcoma","href":"/tagged/sarcoma/","tip":"Every record tagged sarcoma."}]},"sortKeys":{"year":0}},{"id":"waseem-qasim","name":"Waseem Qasim","tldr":"Delivered the first gene-edited off-the-shelf CAR-T cells to infants with leukaemia and the first base-edited CAR-T therapy.","route":"/people/waseem-qasim/","kind":"person","round":true,"avatar":"person","sub":"Professor of Cell and Gene Therapy, UCL Great Ormond Street Institute of Child Health · Great Ormond Street Hospital for Children","facets":{"kind":["Person"],"cancers":["Acute lymphoblastic leukaemia"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[{"label":"Acute lymphoblastic leukaemia","href":"/cancers/all-leukemia/","tip":"Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager."}],"tags":[{"label":"cell-therapy","href":"/tagged/cell-therapy/","tip":"Every record tagged cell-therapy."},{"label":"gene-editing","href":"/tagged/gene-editing/","tip":"Every record tagged gene-editing."}]},"sortKeys":{"year":0}},{"id":"wilms-tumor","name":"Wilms tumour (nephroblastoma)","tldr":"Wilms tumour is a kidney cancer of young children and one of paediatric oncology's success stories: surgery plus a few months of chemotherapy cures about nine in ten. Today's trials aim to give the lowest-risk children almost no chemotherapy while finding the few with aggressive biology.","route":"/cancers/wilms-tumor/","kind":"cancer","cancerIcon":"wilms-tumor","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[]},"sortKeys":{"year":0}},{"id":"medulloblastoma-wnt","name":"WNT-activated medulloblastoma","tldr":"WNT-activated medulloblastoma is the rarest and most curable of the four molecular groups of medulloblastoma, a brain tumour of the cerebellum. It is driven by a mutation in the beta-catenin gene that switches the WNT growth pathway on. Almost every child is cured with standard therapy, so current trials are asking how much radiotherapy and chemotherapy can be taken away.","route":"/cancers/medulloblastoma-wnt/","kind":"cancer","cancerIcon":"medulloblastoma-wnt","facets":{"kind":["Cancer"],"cancers":[],"year":[]},"cols":{"kind":{"facet":"kind","value":"Cancer","tip":"One page per disease: state of the art, history, and what is coming."},"cancers":[],"tags":[{"label":"subtype-page","href":"/tagged/subtype-page/","tip":"Every record tagged subtype-page."},{"label":"cns","href":"/tagged/cns/","tip":"Every record tagged cns."}]},"sortKeys":{"year":0}},{"id":"yael-mosse","name":"Yael P. Mossé","tldr":"Discovered ALK mutations in neuroblastoma and led the trials bringing crizotinib and lorlatinib to children.","route":"/people/yael-mosse/","kind":"person","round":true,"avatar":"person","sub":"Professor of Pediatrics and Patricia Brophy Endowed Chair in Neuroblastoma Research, Children's Hospital of Philadelphia · Children's Hospital of Philadelphia","facets":{"kind":["Person"],"cancers":["Neuroblastoma"],"year":[]},"cols":{"kind":{"facet":"kind","value":"Person","tip":"The clinicians and scientists doing the work: specialisms, bios, papers, and where to find them."},"cancers":[{"label":"Neuroblastoma","href":"/cancers/neuroblastoma/","tip":"Neuroblastoma is a childhood nerve-cell cancer where anti-GD2 antibodies and, recently, GD2 CAR-T have improved survival in high-risk disease."}],"tags":[{"label":"neuroblastoma","href":"/tagged/neuroblastoma/","tip":"Every record tagged neuroblastoma."},{"label":"alk","href":"/tagged/alk/","tip":"Every record tagged alk."}]},"sortKeys":{"year":0}}]