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Hazard ratios and medians are the language trial results are published in; this page is the same numbers in English. This page takes every trial result recorded in OnCo and says what it means for people: how many more out of 100 were helped, roughly how many need to be treated for one extra person to benefit, what a median is and is not, whether the endpoint is a surrogate or actual survival, and who the trial enrolled. The numbers come from the trial records and their sources; the words are ours.
Giving the immunotherapy doublet before surgery, instead of nivolumab after, cut the risk of recurrence by about two thirds and let most patients skip further treatment.
The trial behind India's first CAR-T approval: about three in four heavily pretreated patients with lymphoma or leukaemia responded to a therapy made in Mumbai.
The trial that turned menin inhibition from an idea into the first approved drug for KMT2A-rearranged leukaemia.
In children with average-risk leukaemia, adding blinatumomab pushed three-year disease-free survival from 88% to 96%, one of the largest gains in decades.
A phase 3 trial of Clofarabine, Cyclophosphamide, Cytarabine, Dasatinib, Daunorubicin, Dexamethasone, Doxorubicin, Etoposide, Leucovorin, Mercaptopurine, Methotrexate, Asparaginase, Prednisone, Thioguanine and Vincristine in Acute lymphoblastic leukaemia and Leukaemia, run by National Cancer Institute (NCI), completed.
Proved that adding the immunotherapy blinatumomab to standard treatment saves lives even in patients whose leukaemia was already undetectable.
The trial that showed a gentler CAR-T design could treat adult ALL with a fraction of the severe side effects.
Head to head, the third-generation TKI ponatinib doubled the rate of deep, undetectable remission over imatinib in newly diagnosed Ph-positive ALL.
The first industry-run CAR-T trial in India, testing a Bengaluru-made version of a Barcelona hospital's cell therapy in relapsed lymphoma and leukaemia; it led to the approval of Qartemi.
AALL1231 tried to improve treatment for T-cell leukaemia and lymphoma in children by adding bortezomib and by dropping routine radiotherapy to the brain; bortezomib clearly helped the lymphoma patients but not the whole group, and more than nine in ten children were spared cranial radiotherapy without more relapses.
A phase 3 trial of Omidubicel in Acute lymphoblastic leukaemia and Leukaemia, run by Gamida Cell ltd, completed.
A phase 2 trial of Azacitidine in acute myeloid leukaemia, run by Bristol-Myers Squibb, active and no longer recruiting.
ASCERTAIN-V is the study behind the first all-oral AML regimen, approved in May 2026.
A phase 2 trial of Imetelstat in myelodysplastic syndromes / neoplasms and acute myeloid leukaemia, run by GCP-Service International West GmbH, active and no longer recruiting.
A phase 3 trial of Gilteritinib, Cytarabine, Mitoxantrone, Etoposide and Fludarabine in acute myeloid leukaemia, run by Astellas Pharma Inc, now completed.
KOMET-001 was the registration trial for ziftomenib: a quarter of heavily pretreated patients with NPM1-mutated AML reached complete remission on a once-daily pill.
A phase 3 trial of Orca-T in acute myeloid leukaemia and myelodysplastic syndromes / neoplasms, run by Orca Biosystems, Inc., active and no longer recruiting.
A phase 3 trial of Asparaginase, Crisantaspase, Cytarabine, Daunorubicin, Etoposide, Mitoxantrone and Thioguanine in Acute myeloid leukaemia and Leukaemia, run by Children's Oncology Group, active and no longer recruiting.
A phase 3 trial of Magrolimab, Venetoclax, Azacitidine, Cytarabine, Daunorubicin and Idarubicin in Acute myeloid leukaemia, run by Gilead Sciences, recorded as terminated on the registry.
A phase 3 trial of Magrolimab, Venetoclax and Azacitidine in Acute myeloid leukaemia, run by Gilead Sciences, recorded as terminated on the registry.
A phase 2 trial of Azacitidine and Cusatuzumab in acute myeloid leukaemia, run by OncoVerity, Inc., active and no longer recruiting.
A TROP2 ADC helped in non-squamous lung cancer but not squamous, and the overall survival result fell short.
The trial that showed taking out one segment of a lung, rather than a whole lobe, is enough for a small peripheral lung cancer, and that the smaller operation leaves people alive longer.
Used just two cycles of chemotherapy to cover the first weeks while dual immunotherapy takes effect, an answer to the criticism that immunotherapy alone is too slow for people with heavy disease.
KEYNOTE-042 extended pembrolizumab alone to any lung cancer with at least 1 percent PD-L1, but the survival gain came almost entirely from the tumours with 50 percent or more, so chemotherapy is still added for the rest.
Added an anti-VEGF antibody to chemotherapy and immunotherapy, and is the one first-line regimen with evidence in people whose cancer is driven by an EGFR or ALK change.
The trial that put a PD-L1 antibody alongside the PD-1 antibodies as second-line treatment for lung cancer, in the largest second-line immunotherapy trial run.
Showed that a PD-1 antibody also beats chemotherapy in the commoner, non-squamous form of lung cancer, and that PD-L1 staining predicts how much.
An oral drug against three angiogenic receptors added to second-line chemotherapy; it worked, but only in adenocarcinoma, which is how it is licensed.
Adding an antibody against the VEGF receptor to second-line chemotherapy added about six weeks of life: enough for a licence, not enough for NICE.
Showed that the second-generation EGFR pill afatinib beats chemotherapy for lung cancers driven by an EGFR mutation, and that the benefit is largest for the two common mutations.
ADIUVO asked whether people whose low-grade adrenal cancer had been fully removed should take the toxic drug mitotane for two years to prevent recurrence; recurrence and survival were no different from watching and waiting, so observation with regular scans is the standard for this group.
This is the first trial to show that a drug given after surgery and radiotherapy stops the most dangerous skin squamous cancers coming back. Two years on, 87 out of 100 treated were still free of the cancer, against 64 out of 100 given a dummy.
Transplant patients are kept out of immunotherapy trials because the drugs can make the body reject the transplanted organ. This small study changed the anti-rejection drugs first, then gave the immunotherapy, and no one lost their kidney while nearly half saw their cancer shrink.
CK-301-101 was the first study of cosibelimab, and its skin squamous cell cancer groups, where about half of tumours shrank, led to the drug's US approval in December 2024.
Giving the immunotherapy before the operation left no living cancer cells in the removed specimen in half of the patients. For tumours that would otherwise cost someone an eye, an ear or part of the face, that changes what the operation has to take.
KEYNOTE-629 showed that pembrolizumab shrinks about a third of recurrent or metastatic skin squamous cell cancers and half of locally advanced ones, giving a second immunotherapy option after cemiplimab.
EMPOWER-CSCC-1 showed that cemiplimab shrinks almost half of advanced skin squamous cell cancers, most of them for years, and it gave this cancer its first approved drug.
A phase 3 trial of Lenvatinib in hepatocellular carcinoma, run by CStone Pharmaceuticals, active and no longer recruiting.
A phase 3 trial of Cabozantinib, Sorafenib, Atezolizumab in hepatocellular carcinoma, run by Exelixis, active and no longer recruiting.
Dual immune checkpoint blockade gave the longest median survival yet seen in a first-line liver cancer trial.
A single dose of a CTLA-4 antibody added to PD-L1 blockade doubled five-year survival in liver cancer without needing an anti-VEGF drug.
A phase 2 trial of Tremelimumab, Durvalumab, Bevacizumab in hepatocellular carcinoma, run by MedImmune LLC, active and no longer recruiting.
The trial that ended the sorafenib era: immunotherapy plus an anti-VEGF antibody became the new first-line standard for liver cancer.
REACH-2 showed that the antibody ramucirumab helped people with liver cancer live longer after sorafenib, but only when a blood marker, alpha-fetoprotein, was high, making it the first liver cancer drug approved on a biomarker.
Cabozantinib extended survival in previously treated liver cancer, including after two prior therapies.
Lenvatinib matched sorafenib on survival with more tumour shrinkage, giving a second first-line option after a decade.
RESORCE delivered the first second-line survival benefit in liver cancer.
A phase 3 trial of Nivolumab, Relatlimab + nivolumab in melanoma, run by Bristol-Myers Squibb, active and no longer recruiting.
A second LAG-3 blocker failed to beat pembrolizumab alone, a warning that the Opdualag result is not automatically a class effect.
A phase 2 trial of Ceralasertib, Durvalumab in melanoma, run by AstraZeneca, active and no longer recruiting.
A phase 2 trial of Infigratinib and Capmatinib in Melanoma, run by Pfizer, completed.
A phase 3 trial of Bempegaldesleukin and Nivolumab in Melanoma, run by Bristol-Myers Squibb, completed.
A phase 3 trial of Talimogene laherparepvec and Pembrolizumab in Melanoma, run by Amgen, recorded as terminated on the registry.
Proved that adding a LAG-3 blocker to PD-1 blockade delays progression, with far less toxicity than the ipilimumab combination.
A phase 2 trial of Denileukin diftitox in Melanoma and Advanced melanoma, run by Eisai Inc., completed.
The single-arm study that got the first cell therapy for a solid tumour approved: one in three patients responded after everything else had failed.
Settled the order question: for BRAF-mutant melanoma, start with immunotherapy and save the targeted pills for later.
XPORT-EC-042 tested maintenance selinexor against placebo in TP53-normal advanced endometrial cancer after response to platinum chemotherapy, chasing a subgroup finding from the earlier SIENDO trial. It missed its primary endpoint in July 2026: a trend favoured selinexor but did not reach statistical significance, a lesson in the limits of post-hoc biomarker subgroups.
A next-generation folate-receptor ADC that works even in tumours with low folate receptor, with responses in both ovarian and endometrial cancer.
A phase 2 trial of Trastuzumab duocarmazine in Endometrial cancer, run by Byondis B.V., completed.
DESTINY-PanTumor02 gave the HER2-directed antibody-drug conjugate Enhertu to 267 patients in seven cohorts of HER2-expressing solid tumours, endometrial and cervical cancer among them. Endometrial cancer responded best, and in April 2024 the FDA granted the first tumour-agnostic HER2 approval, for tumours with the strongest HER2 staining and no satisfactory alternative.
A third immunotherapy confirmed the benefit in dMMR disease and hinted that adding olaparib helps the mismatch-repair-proficient majority.
NRG-GY018 is the pembrolizumab twin of RUBY: immunotherapy plus chemotherapy cut the risk of progression by 70% in dMMR tumours and 46% in the rest.
Adding immunotherapy to first chemotherapy for advanced endometrial cancer extended life by more than a year on average, most dramatically in tumours with a broken DNA spell-checker.
A pill that blocks tumour blood vessels plus immunotherapy extended survival after chemotherapy, including in tumours that immunotherapy alone does not touch.
A phase 3 trial of Ixabepilone, Doxorubicin and Paclitaxel / nab-paclitaxel in Endometrial cancer, run by R-Pharm, recorded as terminated on the registry.
EXPLORER was the first human study of avapritinib, a pill designed to block the KIT D816V mutation that drives almost every case of advanced systemic mastocytosis; three quarters of evaluable patients responded and a third went into complete remission, and the drug was approved for the disease in 2021.
A new brentuximab-based intensive regimen matched Europe's most effective (and most toxic) chemotherapy with far fewer side effects.
Immunotherapy plus chemotherapy beat the previous best regimen with far less nerve damage, in the first Hodgkin trial to enrol children and adults together; approved March 2026.
AHOD1331 showed that replacing bleomycin with the antibody-drug conjugate brentuximab vedotin in the chemotherapy given to children and teenagers with advanced Hodgkin lymphoma cut relapses by more than half without extra side effects, and it became the new standard for high-risk disease.
A phase 1/2 trial of Brentuximab vedotin, Doxorubicin, Vinblastine and Dacarbazine in hodgkin lymphoma, run by Takeda, active and no longer recruiting.
Starting with the most intensive Hodgkin chemotherapy and switching to the gentler standard once the scan cleared gave the same results with markedly less anaemia, low platelets and infection.
Swapping bleomycin for the CD30 ADC in first-line chemotherapy improved survival in advanced Hodgkin lymphoma, the first frontline survival gain in decades.
People whose scan was clear after two rounds of the most intensive Hodgkin chemotherapy could stop after four rounds instead of six or eight, with the same disease control and far fewer severe infections.
Showed that patients whose PET scan is clear after two cycles can safely drop bleomycin and its lung toxicity.
ECHELON-2 was the first trial in decades to improve on CHOP chemotherapy for T-cell lymphoma: swapping vincristine for the antibody-drug conjugate brentuximab vedotin helped patients live longer, especially those with anaplastic large cell lymphoma.
The registrational study of a fourth-generation ALK pill designed to work after lorlatinib and to spare the brain-related side effects.
Showed that giving an ALK pill after surgery, instead of chemotherapy, sharply reduces recurrence, including in the brain.
eXalt3 showed that ensartinib, a second-generation ALK pill, kept ALK-positive lung cancer under control about twice as long as crizotinib, with useful activity against brain metastases.
The trial with the longest disease control ever seen for a targeted lung cancer pill: most patients still progression-free at five years.
ALTA-1L showed that brigatinib more than doubled the time ALK-positive lung cancer stayed under control compared with crizotinib, with strong activity against brain metastases.
ALEX showed that alectinib, a second-generation ALK drug that gets into the brain, kept ALK-positive lung cancer under control for about three years against less than one with crizotinib, and cut brain progression by three quarters.
ALTA tested two doses of brigatinib in ALK-positive lung cancer that had progressed on crizotinib; the higher dose, started after a lower-dose first week, gave more responses and longer control, including in the brain.
ASCEND-4 showed that ceritinib, a second-generation ALK pill, kept untreated ALK-positive lung cancer under control about twice as long as platinum chemotherapy, though stomach and gut side effects were common.
A phase 3 trial of Ceritinib, Pemetrexed and Docetaxel in Non-small-cell lung cancer, run by Novartis Pharmaceuticals, completed.
The trial that made an ALK pill, rather than chemotherapy, the first treatment for ALK-positive lung cancer.
A phase 2 trial of Romidepsin in Alveolar soft part sarcoma, Angiosarcoma, Epithelioid sarcoma, Chondrosarcoma, Osteosarcoma and Leiomyosarcoma, run by National Cancer Institute (NCI), completed.
A phase 2 trial of atezolizumab and cobimetinib in gallbladder cancer and other biliary tract cancers, run by National Cancer Institute (NCI), active and no longer recruiting.
In Japan, six months of the oral chemotherapy S-1 after surgery for bile duct, gallbladder or ampullary cancer improved three-year survival from 68 to 77 percent. S-1 is not licensed for this use in the UK or Europe, so the result cannot be applied directly to NHS patients.
ESPAC-3 compared the two adjuvant chemotherapies then available after pancreatic cancer surgery and found they gave the same survival, about 23 months, but gemcitabine caused half as many serious side effects; a companion cohort in ampullary and bile duct cancers is still the main randomised evidence for adjuvant chemotherapy in those rarer tumours.
The largest cancer screening trial ever run, testing whether a multi-cancer blood test reduces late-stage diagnoses; it reports that it did not, and its test-performance paper appeared in Nature Medicine on 22 September 2026.
POD1UM-303 showed that adding the immunotherapy retifanlimab to the standard carboplatin-paclitaxel chemotherapy for advanced anal cancer delayed progression by about two months in median terms and reduced the risk of progression by 37 percent, making it the first immunotherapy to improve first-line treatment of this cancer.
ANCHOR proved for the first time that treating precancerous anal lesions in people with HIV prevents anal cancer: those whose lesions were removed or ablated were 57 percent less likely to develop cancer than those who were simply monitored, which is the evidence behind screening and treating these lesions.
InterAACT was the first randomised trial ever run in advanced anal cancer; the two chemotherapy pairs shrank tumours equally often, but carboplatin with paclitaxel caused far fewer serious side effects and patients on it lived longer, so it became the standard chemotherapy backbone.
NCI9673 was the first completed immunotherapy trial in anal cancer: nivolumab on its own shrank tumours in a quarter of heavily treated patients, which made PD-1 blockade a standard later option, but the follow-on randomisation showed that adding ipilimumab did not help and added toxicity.
ACT II, the largest anal cancer trial, confirmed mitomycin-fluorouracil chemoradiation as the standard, showed that swapping in cisplatin or adding maintenance chemotherapy gave nothing, and taught doctors to wait six months before judging response.
TAPPAS tested whether adding an antibody against endoglin, a protein on the abnormal blood vessel cells that make up angiosarcoma, would improve on the standard tablet pazopanib; it did not, and the trial was stopped for futility.
The first targeted-therapy win in low-grade brain tumours: a pill that more than doubled the time before the tumour grew, delaying radiation and chemotherapy by years.
CATNON showed that a year of temozolomide after radiotherapy lengthens survival in anaplastic gliomas that lack the 1p/19q co-deletion, and that the benefit is concentrated in IDH-mutant tumours.
EORTC 22033 asked whether temozolomide could replace radiotherapy as the first treatment for high-risk low-grade glioma and found no difference in progression-free survival, with the molecular subtype mattering more than the choice of treatment.
NOA-08 showed that older people with glioblastoma can be treated with temozolomide tablets instead of six weeks of radiotherapy without living less long, and that the MGMT test tells you which to choose: chemotherapy if the gene is methylated, radiotherapy if it is not.
The 2005 trial that set the treatment every glioblastoma patient still receives. Nothing has replaced it in twenty years.
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.
ACNS0333 was the first trial built only for atypical teratoid/rhabdoid tumour, a brain cancer of very young children that used to be almost always fatal; its intensive plan of chemotherapy, high-dose chemotherapy with stem cell rescue and focused radiotherapy more than halved the risk of relapse or death compared with earlier treatment and became the template for care.
People born with Gorlin syndrome grow new basal cell carcinomas for life, and the pills that block the pathway cause side effects most cannot live with. Putting the drug on the skin instead was the obvious idea. A small trial hints that it works, but only in analyses decided after the results were seen.
Scraping the lump off first and then using the cream was tested against simply cutting it out, for the thicker kind of basal cell carcinoma. It did not come close: about one in five of those treated without surgery had the tumour back within five years, against one in fifty after the operation.
Before this trial there was nothing to offer someone whose basal cell carcinoma had outgrown or outlasted the hedgehog pill. Immunotherapy shrank the tumour in about a third of them, and it is now the standard second treatment.
Giving the hedgehog pill first to shrink a large facial basal cell carcinoma made the operation smaller or less disfiguring in four out of five people. Over three years, though, more than a third of those who benefited had the cancer back.
A phase 3 trial of Aminolevulinic acid and Methyl aminolevulinate in Basal cell carcinoma, run by Biofrontera Bioscience GmbH, completed.
This trial compared the three non-surgical treatments people are actually offered for a thin basal cell carcinoma. Photodynamic therapy, the one that was assumed to be best, came last. The imiquimod cream was the best of the three at one year and still the best at five, though by then a fifth of those treated with it had the tumour back.
The largest trial of a cream against an operation for the commonest cancer found the cream is worse, and stayed worse at five years. About one person in six treated with the cream still needed something else done, against one in fifty after surgery. It is still a reasonable choice for some people, but it is not equivalent.
This is the study that measured what taking the basal cell carcinoma pill is actually like, in more than a thousand ordinary patients rather than a hundred selected ones. It shrank most tumours. Almost everybody had side effects, and the average person stopped after about eight months.
BOLT showed that sonidegib, a second hedgehog pathway pill, shrinks about four in ten locally advanced basal cell carcinomas at its lower dose, and that the higher dose adds side effects rather than responses.
This is the only randomised trial of the microscope-guided skin cancer operation against the ordinary one. For a cancer that has already come back once, the microscope-guided operation clearly wins. For a cancer being treated the first time, the difference took ten years to appear and never reached statistical significance.
The trial that added immunotherapy to pre-surgery chemotherapy for triple-negative breast cancer and, uniquely, improved survival.
GeparSixto showed that adding carboplatin to chemotherapy given before surgery made triple-negative breast tumours disappear completely far more often, and in longer follow-up cut relapses, which put platinum into the standard neoadjuvant regimen for this type.
KEYNOTE-407 showed that adding pembrolizumab to chemotherapy helped people with squamous lung cancer live longer whatever their PD-L1 level, and made chemo-immunotherapy the standard first treatment for this type.
FIGHT-302 set out to test whether the FGFR2 inhibitor pemigatinib should be used before chemotherapy in bile duct cancers driven by an FGFR2 fusion; the trial closed early after the standard of care changed, and its 2026 report showed pemigatinib delayed progression compared with chemotherapy, with similar overall survival.
A phase 2 trial of olaparib in gallbladder cancer and other biliary tract cancers, run by Academic and Community Cancer Research United, active and no longer recruiting.
Adding a third chemotherapy drug, nab-paclitaxel, to gemcitabine and cisplatin did not help people with advanced bile duct or gallbladder cancer live longer in this 452-patient US trial, although the gallbladder subgroup showed a hint of slower progression that needs its own test.
Adding a liposomal chemotherapy did not help in second-line bile duct cancer, contradicting an earlier Korean trial.
A phase 2 trial of Telotristat ethyl in Biliary tract cancer and Biliary tract cancer, run by TerSera Therapeutics LLC, recorded as terminated on the registry.
The trial behind the first HER2 drug approved for bile duct and gallbladder cancer: zanidatamab shrank tumours in 41 percent of 80 people with HER2-positive disease that had progressed on chemotherapy, and more than half of those enrolled had gallbladder cancer. It led to approvals in the United States, the European Union and the UK, where NICE recommended it in May 2026.
TreeTopp tested whether the HER-family blocker varlitinib added to capecitabine helps bile duct and gallbladder cancer after first-line chemotherapy. It did not: response, progression and survival were the same, and the planned phase 3 part was abandoned. A small gallbladder subgroup hinted at slower progression but the finding was not conclusive.
A Korean trial found that adding liposomal irinotecan to fluorouracil as second-line chemotherapy for bile duct and gallbladder cancer delayed progression, from about six weeks to four months on the re-read scans, at the cost of more neutropenia. A German trial of the same idea was negative, so it is an option rather than a standard, and it is not commissioned in the UK.
The basket trial that showed pembrolizumab alone rarely works in bile duct and gallbladder cancer: only 6 of 104 patients responded, though those who did stayed in response for years. It is also the trial behind the tumour-agnostic approvals of pembrolizumab for mismatch-repair-deficient and high mutation-burden cancers, which do apply to gallbladder cancer.
In the ROAR basket trial, the BRAF-blocking pair dabrafenib and trametinib shrank tumours in about half of 43 people with bile duct or gallbladder cancer carrying the BRAF V600E mutation, which is found in roughly 5 percent of biliary cancers. The pair is approved for any solid tumour with this mutation in the United States and is listed by guidelines for biliary cancer.
A phase 2 trial of Tucatinib, Trastuzumab and Fulvestrant in endometrial cancer, cervical cancer, biliary tract cancer and non-small-cell lung cancer, run by Seagen, a wholly owned subsidiary of Pfizer, active and no longer recruiting.
A second immunotherapy trial confirmed the modest survival benefit of adding PD-1 blockade to chemotherapy in bile duct cancer.
Futibatinib produced responses in over 40% of patients whose bile duct cancer carried an FGFR2 fusion.
TOPAZ-1 was the first immunotherapy success in bile duct cancer, with a small median gain but a growing tail of long survivors.
The first randomised trial of a targeted drug in bile duct cancer; it slowed the disease without shrinking it.
FIGHT-202 is the single-arm study that produced the first targeted approval in bile duct cancer.
The 60-patient study that made the chemotherapy triplet look promising in bile duct and gallbladder cancer: nearly half responded and median survival was 19 months, results that the larger S1815 trial later failed to confirm.
The only prospective trial of chemotherapy followed by chemoradiotherapy after surgery for bile duct and gallbladder cancer: 79 patients, a third with gallbladder cancer, had two-year survival of 65 percent and median survival of 35 months, similar whether or not the margin was clear. It is the basis on which some centres offer radiotherapy after an R1 resection.
Two years of hormone therapy alongside radiotherapy after prostate surgery kept the cancer from spreading in six more men in a hundred than six months did, at the cost of more side effects.
Showed that treating a fast-rising PSA after surgery or radiation with enzalutamide delays spread.
A phase 3 trial of Flutamide, Bicalutamide, Goserelin / leuprolide, Buserelin and Triptorelin in Prostate cancer, run by Radiation Therapy Oncology Group, with a status the registry has not verified recently.
Three trials asked the same question and none was big enough alone. Pooling them, planned in advance so nobody could cherry-pick, showed that waiting for the PSA to rise is as good as treating everyone.
The French version of the same question, and the same answer: treating everyone straight after surgery does not help, and it doubles the rate of lasting bladder and erection problems.
Treating one to three secondary tumours with precise radiotherapy cut the chance of the cancer progressing within six months from three in five to one in five, and worked best when a PSMA scan showed nothing had been missed.
Waiting to see whether the PSA rises after prostate surgery, and only then giving radiotherapy, works as well as treating everybody straight away, and spares two men in three the radiotherapy altogether.
Waiting for the PSA to rise before giving radiotherapy after prostate surgery gave the same cancer control and spared half the men pelvic radiation, though the trial closed too early to prove it formally.
Adding six months of hormone therapy to radiotherapy given for a rising PSA after prostate surgery raised the proportion free of progression at ten years from about half to about two thirds.
Zapping the handful of spots that show up when prostate cancer comes back put off the day hormone therapy was needed, by about eight months, in a small trial.
A phase 2 trial of Avelumab and Sacituzumab govitecan in bladder & urothelial cancer, run by EMD Serono Research & Development Institute, Inc., active and no longer recruiting.
For patients who cannot have cisplatin, the ADC-immunotherapy pair around surgery cut the risk of recurrence or death by 60% and the risk of death by half.
The trial that could replace chemotherapy before bladder removal in fit patients with an ADC plus immunotherapy.
The first trial to use a blood test for leftover cancer to decide who gets immunotherapy, and it worked.
Adding a year of durvalumab to standard BCG bladder instillations cut the risk of recurrence or progression by about a third, the first systemic immunotherapy approved in early bladder cancer.
A phase 3 trial of Nivolumab, Ipilimumab, Gemcitabine, Cisplatin and Carboplatin in bladder & urothelial cancer, run by Bristol-Myers Squibb, active and no longer recruiting.
A phase 3 trial of UGN-102 in bladder & urothelial cancer, run by UroGen Pharma Ltd., active and no longer recruiting.
A cancer-killing virus instilled into the bladder produced complete responses in three quarters of patients whose BCG had failed, with almost no serious side effects.
The first immunotherapy shown to improve survival when given around bladder-removal surgery.
A tiny pretzel-shaped device that slowly releases chemotherapy inside the bladder cleared carcinoma in situ in over 80% of patients whose BCG had failed.
A phase 1/2 trial of Pivekimab sunirine in blastic plasmacytoid dendritic cell neoplasm and myeloproliferative neoplasms, run by AbbVie, active and no longer recruiting.
PREOPANC-2 asked whether the strongest chemotherapy given entirely before surgery beats the Dutch standard of gemcitabine chemoradiotherapy before and gemcitabine after; survival was the same, about 22 months in both arms, so both remain reasonable ways to treat first, and the question of surgery first against treatment first moves to Alliance A021806 and PREOPANC-3.
ESPAC-5 randomised people whose pancreatic cancer sat on the border of being removable between going straight to surgery and having two months of chemotherapy or chemoradiotherapy first; those treated first were far more likely to be alive a year later, 78 to 84 percent with chemotherapy against 39 percent with immediate surgery, which supports treating before operating in borderline disease.
A021501 set out to test radiotherapy before surgery for borderline pancreatic cancer and closed that arm early because fewer patients reached a clear-margin operation; chemotherapy alone gave 18-month survival of 67 percent and a median of 29.8 months, and became the reference regimen for the setting.
The Dutch trial testing whether treating before surgery beats operating first. Chemoradiation first fell short at the primary analysis but won at five years, with one in five patients alive against one in fifteen.
Showed that giving the strong four-drug chemotherapy after pancreatic surgery adds years of life for fit patients.
IMspire150 tested adding the immunotherapy drug atezolizumab to the two targeted pills used for BRAF-mutant melanoma; the triple combination kept the cancer from growing for longer as judged by the treating doctors, which earned it approval, but it did not help people live longer.
A year of two targeted pills after surgery halves the risk of relapse in BRAF-mutant melanoma, and the benefit is still visible a decade later.
Doubled survival, from about 15 to about 30 months, in the worst-prognosis genetic subtype of bowel cancer by adding two targeted drugs to first-line chemotherapy.
Showed that a two-drug immunotherapy combination controls mismatch-repair-deficient bowel cancer for over four years on average, and beats immunotherapy alone.
The trial that explained why BRAF drugs alone fail in bowel cancer: blocking BRAF wakes up EGFR, so both must be blocked at once.
The trial that made immunotherapy alone, with no chemotherapy, the first treatment for the 5% of bowel cancers with a broken DNA spell-checker. Over half of patients were alive at five years.
BEACON was the first trial to show that blocking BRAF works in bowel cancer once the escape route through EGFR is blocked too: encorafenib with cetuximab lengthened life and shrank ten times as many tumours as chemotherapy.
PHAROS showed that the melanoma combination encorafenib and binimetinib shrinks three quarters of untreated BRAF V600E lung cancers and nearly half of previously treated ones, giving a second approved BRAF plus MEK pair for lung cancer.
Thirty-six patients showed that the melanoma combination of a BRAF and a MEK drug also works in the small slice of lung cancers with the same mutation.
MATRix/IELSG43 settled how to consolidate remission in lymphoma of the brain: after MATRix chemotherapy, a high-dose chemotherapy and stem cell transplant kept 78 percent of patients free of progression at three years against 51 percent with conventional consolidation chemotherapy, and also lengthened survival, so transplant is now the preferred consolidation for fit patients.
INTUITT-NF2 is a rolling trial for people with the inherited condition NF2, who grow many benign tumours of the nerves and brain lining; its first drug, brigatinib, shrank a share of tumours, slowed the growth of all types and improved hearing in a third of affected ears, which is the evidence behind offering it for these tumours.
NRG CC001 showed that shaping whole-brain radiotherapy to spare the hippocampus, the seat of memory, reduces cognitive decline without any loss of tumour control.
ACNS0121 showed that focused radiotherapy to the tumour bed straight after surgery cures most children with ependymoma, including those under three who used to be denied radiotherapy, and that children whose tumour could not be fully removed do far worse even with chemotherapy and a second operation.
ACNS1123 asked whether children with germ cell tumours of the brain who respond well to chemotherapy can safely have less radiotherapy; for non-germinomatous tumours survival stayed high but the few relapses were all in the spine, which was left out of the smaller field, and for germinoma the reduced doses held.
INTELLANCE-1 tested an antibody-drug conjugate that targets the EGFR amplification found in about half of glioblastomas, added to standard treatment; it did not help people live longer, and the drug was abandoned.
N0574 showed that adding whole-brain radiotherapy to radiosurgery for a few brain metastases damages memory and thinking without lengthening life, and radiosurgery alone became the standard.
IELSG32 built the MATRix regimen that is now the standard first treatment for lymphoma confined to the brain: adding rituximab and thiotepa to methotrexate and cytarabine doubled the complete remission rate, and its second part showed that a stem cell transplant works as well as whole-brain radiotherapy for consolidation, with less harm to thinking.
QUARTZ found that whole-brain radiotherapy added nothing measurable, in survival or quality of life, for lung cancer patients whose brain metastases could not be treated with surgery or radiosurgery.
ACNS0122 set the modern American standard for children with the more aggressive kind of germ cell tumour of the brain: chemotherapy first, surgery for anything left, then radiotherapy to the whole brain and spine, which cured about nine in ten and became the benchmark that later trials tried to match with less radiation.
A phase 3 trial of Fluciclovine F-18 in Brain metastases, run by Blue Earth Diagnostics, completed.
Enhertu shrank brain metastases in most patients, including those with untreated lesions, answering a question its earlier trials had excluded.
The first trial to prove a drug helps HER2-positive brain metastases, extending survival by four and a half months overall.
A phase 2 trial of Gamma Knife in Brain metastases, run by University of Alabama at Birmingham, completed.
Showed that adding whole-brain radiotherapy to focused radiosurgery for a few brain secondaries does not extend life, though it does halve the chance of new ones appearing.
The first biomarker-directed drug approval in pancreatic cancer, for the roughly 5-7% with inherited BRCA mutations, though it did not extend overall survival.
Showed that a year of a PARP inhibitor after standard treatment improves survival in women with inherited BRCA mutations.
EMBRACA showed that the PARP inhibitor talazoparib delays progression by about three months compared with chemotherapy in women with an inherited BRCA mutation and advanced breast cancer, with better quality of life but no gain in overall survival.
OlympiAD was the first trial to show a PARP inhibitor tablet beats chemotherapy in breast cancer, delaying progression by about three months in women with an inherited BRCA mutation and with fewer side effects, though it did not lengthen life overall.
A phase 2 trial of Poziotinib in Breast cancer, run by Spectrum Pharmaceuticals, Inc, recorded as terminated on the registry.
A phase 2 trial of FES PET in Breast cancer, run by University of Utah, recorded as terminated on the registry.
The largest trial of skipping the armpit operation, and the one with six years of follow-up. Women who had no lymph node surgery did as well as those who did, and had less arm swelling, better shoulder movement and less pain.
If chemotherapy given before surgery clears the lymph nodes completely, irradiating those nodes afterwards adds nothing. Fewer than one woman in ten had the cancer come back either way, and the difference between the two groups was within chance.
Putting the implant in front of the chest muscle rather than behind it was adopted across Britain on the argument that it hurts less and looks better. This study followed the first 347 women and found the implant was lost about as often as with the older technique, and that physical and sexual wellbeing were worse than before surgery at both three and eighteen months.
A phase 3 trial of Efbemalenograstim alfa and Filgrastim in Breast cancer, run by EVIVE Biotechnology, completed.
For women with one to three involved nodes or a larger node-negative tumour, radiotherapy to the chest wall after mastectomy did not help them live longer. It roughly halved the small chance of the cancer returning on the chest wall, from one in forty to one in ninety.
A phase 2 trial of Tusamitamab ravtansine and Gemcitabine in Breast cancer, run by Sanofi, recorded as terminated on the registry.
A phase 2 trial of Poziotinib in Breast cancer and Colorectal cancer, run by Spectrum Pharmaceuticals, Inc, recorded as terminated on the registry.
A phase 2 trial of Mifepristone and Paclitaxel / nab-paclitaxel in Breast cancer, run by University of Chicago, active and no longer recruiting.
Adding low-cost oral metronomic tablets to standard paclitaxel-carboplatin doubled median survival from 5 to 10 months in a randomised trial run in Varanasi.
NIVOPOSTOP showed that adding the immunotherapy nivolumab to chemoradiation after surgery for high-risk head and neck cancer kept about one in ten more patients free of relapse at three years, the first improvement in post-operative treatment in two decades.
A Mumbai trial that added about one-twentieth of the usual dose of the immunotherapy nivolumab to cheap oral chemotherapy and nearly tripled the share of patients alive at one year.
Two cheap tablets taken at home matched, and in fact beat, intravenous cisplatin for advanced head and neck cancer in a 422-patient Indian trial, with fewer side effects.
A Tata Memorial trial ended a 50-year debate by showing that removing the neck lymph nodes at the first operation for early mouth cancer raises three-year survival from about two-thirds to four-fifths.
Trained health workers looking inside the mouths of tobacco and alcohol users in Kerala cut oral cancer deaths by a third, and by four-fifths in those who came to every screening round.
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.
CUPISCO was the first randomised test of precision medicine in cancer of unknown primary, where doctors cannot see where the cancer started. After three rounds of chemotherapy, switching to a drug chosen from the tumour's genetic profile held the cancer back for longer than carrying on with chemotherapy.
A two-headed antibody that blocks two immune brakes at once extended survival in advanced cervical cancer regardless of PD-L1 status.
SHAPE showed that women with small, low-risk cervical cancers can safely have a simple hysterectomy rather than the more extensive radical operation, with the same low rate of recurrence and fewer bladder and sexual problems.
A second immunotherapy confirmed that adding a checkpoint inhibitor to chemotherapy plus bevacizumab extends life in advanced cervical cancer.
CALLA tested adding the PD-L1 antibody durvalumab to chemoradiotherapy for locally advanced cervical cancer and found no significant benefit, in contrast to the later positive KEYNOTE-A18 trial of pembrolizumab in a higher-risk population.
The first ADC to extend life in cervical cancer, for women whose disease has come back after chemotherapy.
Six weeks of cheap, generic chemotherapy before standard chemoradiation cut deaths by 40%, an advance usable anywhere in the world.
Adding immunotherapy to curative chemoradiation for locally advanced cervical cancer improved both control and survival, the first such advance in two decades.
One shot of HPV vaccine was 97.5% effective against the two most dangerous HPV types, making it realistic to vaccinate the whole world.
The first immunotherapy to extend life in recurrent cervical cancer, approved in Europe but declined by the FDA.
When an Indian vaccine trial was halted midway, thousands of girls had had only one shot; following them for ten years showed one dose protected as well as three, changing the world's vaccination policy.
A phase 1/2 trial of Durvalumab and Tremelimumab in childhood cancers, run by AstraZeneca, active and no longer recruiting.
ANBL17P1 tested whether the antibody dinutuximab, which works against relapsed neuroblastoma, can be given from the very start of treatment alongside induction chemotherapy for newly diagnosed high-risk disease; none of the 42 children had unacceptable toxicity, which opened the way to the randomised trial now under way.
ANBL0531 showed that children with intermediate-risk neuroblastoma can be cured almost every time with a treatment plan that gives less chemotherapy to those with favourable tumour biology and a good early response, keeping three-year survival at 95 percent while cutting treatment for many.
AREN0532 tested treating the lowest-risk Wilms tumours with surgery alone and no chemotherapy, and giving more treatment only to children whose tumours carried particular chromosome losses; both ideas worked and now guide how much therapy each child gets.
ANBL1221 found that adding the anti-GD2 antibody dinutuximab to irinotecan and temozolomide shrank relapsed neuroblastoma in about half of children, while adding temsirolimus almost never did, and chemo-immunotherapy became the standard salvage treatment.
SIOPEL-4 showed that giving cisplatin every week in a dense schedule, with doxorubicin, to children with high-risk hepatoblastoma got nearly all of them to respond and three quarters to complete resection, lifting three-year survival to 83 percent in a group that used to do poorly.
AEWS0031 showed that giving the same chemotherapy for Ewing sarcoma every two weeks instead of every three cured more patients without adding side effects, and the two-weekly schedule became the standard for localised disease in North America.
SIOPEL-3 showed that children with standard-risk hepatoblastoma can be cured with cisplatin alone before and after surgery, leaving out doxorubicin and its heart damage, because the cure rate was the same as with the two-drug combination.
REGOBONE showed that the tablet regorafenib delayed progression of osteosarcoma that had come back after chemotherapy, giving one of the few randomised results in relapsed bone cancer.
A phase 2 trial of Acalabrutinib and Umbralisib in Chronic lymphocytic leukaemia, run by Jennifer R. Brown, MD, PhD, active and no longer recruiting.
A phase 3 trial of Acalabrutinib, Rituximab and Chlorambucil in chronic lymphocytic leukaemia, run by AstraZeneca, active and no longer recruiting.
AMPLIFY is the trial behind the first all-oral, fixed-duration CLL regimen approved in the US (February 2026): 14 cycles of two pills, then stop.
The randomised trial that confirmed pirtobrutinib works after other BTK inhibitors fail, leading to full approval in December 2025.
CLL12 asked whether starting the pill ibrutinib early, before chronic lymphocytic leukaemia causes symptoms, is better than the usual practice of watching and waiting; early treatment delayed the disease but after nearly six years of follow-up people lived just as long either way, so watch and wait remains the standard.
A phase 2/3 trial of Umbralisib and Venetoclax in Chronic lymphocytic leukaemia, run by TG Therapeutics, Inc., recorded as terminated on the registry.
A phase 3 trial of Acalabrutinib, Rituximab, Idelalisib and Bendamustine in chronic lymphocytic leukaemia, run by Acerta Pharma BV, active and no longer recruiting.
In fit patients, one year of venetoclax plus obinutuzumab (with or without ibrutinib) clearly beat the old chemotherapy standard.
The study that brought CAR-T to CLL: one in five heavily pretreated patients achieved complete remission, most of them lasting.
A phase 2 trial of Acalabrutinib in chronic lymphocytic leukaemia, run by Acerta Pharma BV, active and no longer recruiting.
A phase 3 trial of Asciminib and Nilotinib in chronic myeloid leukaemia, run by Novartis Pharmaceuticals, active and no longer recruiting.
A phase 3 trial of Imatinib, Nilotinib, Bosutinib, Dasatinib and Asciminib in chronic myeloid leukaemia, run by Novartis Pharmaceuticals, active and no longer recruiting.
A phase 2 trial of Oprelvekin in Leukaemia and Chronic myeloid leukaemia, run by M.D. Anderson Cancer Center, completed.
DASISION showed that the second-generation drug dasatinib brought chronic myeloid leukaemia under control faster and more deeply than imatinib when used from diagnosis, though after five years patients lived equally long on either.
The Chinese randomised trial in which Claudin 18.2 CAR-T cells delayed progression compared with the doctor's choice of later-line drugs in stomach cancer, the first randomised trial of CAR-T cells in a solid tumour.
Two trials that made Claudin 18.2 the third biomarker in stomach cancer, adding about two to three months of survival with an antibody against it.
A phase 3 trial of Nivolumab in renal cell carcinoma, run by Bristol-Myers Squibb, active and no longer recruiting.
The first drug to help kidney cancer patients live longer after surgery, and the first adjuvant immunotherapy in any solid tumour to show an overall survival gain.
The first immunotherapy plus targeted-pill combination for kidney cancer, improving survival across all risk groups.
CARMENA showed that removing the kidney before starting sunitinib did not help people with kidney cancer that had already spread live longer, which ended the routine practice of upfront nephrectomy for intermediate- and poor-risk patients.
CheckMate 214 is the trial that brought dual immunotherapy to kidney cancer, with a survival advantage still visible eight years later and a fifth of patients in long-term remission.
CheckMate 025 was the trial that brought immunotherapy to kidney cancer: nivolumab helped people live longer than everolimus after anti-angiogenic drugs had failed, with fewer side effects.
A phase 3 trial of bevacizumab (glioblastoma use), fOLFOX (5-FU, leucovorin, oxaliplatin), leucovorin (folinic acid), fluorouracil (5-FU) and other drugs in colorectal cancer, run by Alliance for Clinical Trials in Oncology, active but no longer recruiting.
A Nordic trial showing that three years of low-dose aspirin roughly halves recurrence in bowel cancers carrying a particular set of mutations, a biomarker-directed use of a drug that costs pennies.
A phase 3 trial of regorafenib and fOLFIRI (5-FU, leucovorin, irinotecan) in colorectal cancer, run by Institut du Cancer de Montpellier - Val d'Aurelle, active but no longer recruiting.
Adding a year of immunotherapy to chemotherapy after surgery halved recurrences in stage III colon cancers with a broken DNA spell-checker.
The first randomised trial to show that a coached exercise programme after cancer treatment reduces recurrence and death, in colon cancer.
The sequel to DYNAMIC asked whether a blood test should also decide chemotherapy in stage III disease. It could not prove that less is safe, and more did not help.
The UK trial that showed giving some chemotherapy before the operation for colon cancer shrinks the tumour, leaves cleaner margins and cuts the chance of the cancer coming back.
Showed that a blood test can safely halve the number of colon cancer patients given chemotherapy after surgery.
In NICHE-2, four weeks of immunotherapy before surgery wiped out most mismatch-repair-deficient colon cancers, and nobody had relapsed three years later.
Proved that for RAS-normal tumours starting on the left side of the colon, an EGFR antibody beats the VEGF antibody as first partner for chemotherapy.
A phase 1/2 trial of Tislelizumab in colorectal cancer, run by BeiGene, active and no longer recruiting.
Treating the blood test result before the cancer shows up on a scan did not work. It delayed relapse by about four months and caused severe blood toxicity in three-quarters of patients.
AZUR-1 is the registrational trial of the 'no surgery, no radiation, no chemo' approach for mismatch-repair-deficient rectal cancer, built on the MSK study where every patient had a complete response.
A phase 2 trial of Pembrolizumab, Favezelimab in colorectal cancer, run by Merck Sharp & Dohme LLC, active and no longer recruiting.
A phase 1/2 trial of Daraxonrasib in non-small-cell lung cancer and small-cell lung cancer, run by Revolution Medicines, Inc., now recruiting.
The study behind the second approved blood test for bowel cancer screening: the test found about four in five cancers and kept false alarms low, but like Shield it missed most advanced polyps, which is why guidelines still rank blood tests below colonoscopy and stool tests.
The study behind Cologuard Plus: the new stool test caught 94% of bowel cancers and produced fewer false alarms than the original.
The study behind the first approved blood test for bowel cancer screening: it found 83% of cancers but only 13% of advanced polyps.
The first drug to hit the hormone behind cancer wasting: patients on the highest dose gained nearly 3 kg more than placebo in 12 weeks and reported better appetite and more activity.
The trial that chose the best chemotherapy for shrinking liver metastases enough to operate, using a panel of surgeons to judge resectability every two months.
This phase 3 trial showed that a daily injection which turns the brain's fullness signal back on cut body-mass index by about a sixth, and reduced hunger, in children and adults whose weight gain followed damage to the hypothalamus from a tumour or its treatment, while people on placebo gained weight; it supported the March 2026 US approval of Imcivree for that use.
Adding chemotherapy to the radiotherapy given after surgery for the most dangerous skin squamous cancers did not help. What the trial did show is how well surgery and radiotherapy already work: more than four in five of these high-risk cancers were still controlled five years later.
A person with a transplanted kidney who has had one skin squamous cancer will usually get more. Swapping one anti-rejection drug for another roughly halved the number who got another cancer, but it made people unwell often enough that a quarter stopped the new drug.
A phase 2 trial of Synthetic hypericin in Cutaneous T-cell lymphoma and Peripheral T-cell lymphomas, run by Soligenix, completed.
A phase 3 trial of Synthetic hypericin in Cutaneous T-cell lymphoma, run by Soligenix, completed.
MAVORIC showed that mogamulizumab, an antibody that clears the malignant T cells that carry the CCR4 protein, kept cutaneous T-cell lymphoma under control for longer than the standard tablet vorinostat, and did especially well in the blood-involved Sezary form, which led to its approval.
ALCANZA showed that brentuximab vedotin produced lasting improvement in the skin disease of far more people with CD30-positive cutaneous T-cell lymphoma than the standard tablets methotrexate or bexarotene.
Study 201 showed that a ready-made mechlorethamine skin gel worked at least as well as a pharmacy-compounded ointment for early mycosis fungoides, and it was the main evidence for the US approval of Valchlor.
A phase 2 trial of Romidepsin in Cutaneous T-cell lymphoma, run by Celgene, completed.
The first randomised trial to show a drug controls desmoid tumours, with better pain and function.
A phase 2 trial of Glofitamab, Tocilizumab, Doxorubicin, Vincristine, Prednisone and Rituximab in non-Hodgkin lymphoma, run by Hoffmann-La Roche, active and no longer recruiting.
Epcoritamab delayed progression but did not clearly extend life versus chemotherapy in relapsed lymphoma, missing its US primary endpoint in January 2026.
Adding an antibody-drug conjugate to a gentle outpatient chemotherapy pairing added about seven months of life for people with relapsed aggressive lymphoma who could not have a transplant.
The first frontline regimen to beat R-CHOP in high-risk large B-cell lymphoma since rituximab, by adding a CD19 antibody and lenalidomide.
A phase 1/2 trial of Tafasitamab, Lenalidomide in diffuse large b-cell lymphoma, run by Incyte Corporation, active and no longer recruiting.
A chemotherapy-free bispecific plus ADC doublet beat salvage chemotherapy in relapsed large B-cell lymphoma.
A bispecific plus chemotherapy improved survival in relapsed lymphoma, but a US advisory committee voted eight to one that the trial did not apply to American patients, and the second-line use is still not on the US label.
EPCORE NHL-1 was the pivotal single-arm study behind epcoritamab's approval; about half of complete responders are still in remission at three years.
The second trial to show a CAR-T beats transplant in early-relapsing large B-cell lymphoma.
The one second-line CAR-T trial that failed, a reminder that manufacturing time and trial design can erase a real effect.
A phase 3 trial of Trastuzumab and Trastuzumab biosimilars in Ductal carcinoma in situ, run by National Cancer Institute (NCI), completed.
KLASS-01 showed that removing an early stomach cancer through keyhole surgery is as safe for long-term survival as open surgery and causes fewer wound complications, which made laparoscopic gastrectomy the standard for early gastric cancer in Korea and beyond.
The first adjuvant immunotherapy trial in liver cancer looked positive early, then the benefit vanished with longer follow-up.
Enhertu cut recurrence or death by more than half compared with Kadcyla in women with cancer left after pre-surgery treatment, replacing the KATHERINE standard.
DESTINY-Breast11 brought Enhertu into pre-surgery treatment of HER2-positive breast cancer, replacing anthracyclines.
Using an early PET scan to spot responders let a third of women be cured of HER2-positive breast cancer without any chemotherapy.
Six months of trastuzumab was almost as good as twelve, with half the heart toxicity, though twelve remains the global standard.
Switching to an ADC after surgery when pre-surgery treatment left cancer behind halved the risk of recurrence and improved survival.
KRISTINE tested whether an antibody-drug conjugate could replace chemotherapy before surgery in HER2-positive breast cancer and found it could not: fewer tumours disappeared and more grew before the operation.
TRAIN-2 showed that HER2-positive breast cancer can be treated before surgery without an anthracycline: carboplatin and paclitaxel with the two HER2 antibodies made the tumour disappear as often as an anthracycline regimen did, with less harm to the heart.
Adding pertuzumab after surgery helps women whose cancer had reached the lymph nodes, by a few percentage points, and does not help those with node-negative disease.
For small HER2-positive tumours, a gentle regimen of weekly paclitaxel plus trastuzumab gives excellent cure rates, avoiding harsher chemotherapy.
NeoSphere showed that blocking HER2 with two antibodies, pertuzumab and trastuzumab, alongside chemotherapy before surgery made HER2-positive breast tumours disappear completely more often than one antibody alone, which led to the first approval of a cancer drug for use before surgery.
GeparDouze, the largest neoadjuvant immunotherapy trial in triple-negative breast cancer, found that adding atezolizumab to chemotherapy before and after surgery did not significantly reduce relapses (a 3.3-point gain at four years that missed statistical significance), unlike pembrolizumab in KEYNOTE-522.
ALEXANDRA asked whether a year of the immunotherapy atezolizumab added to chemotherapy after surgery stops triple-negative breast cancer coming back. It did not: relapses were no fewer, side effects were more, and the trial was stopped early for futility.
A phase 3 trial of Niraparib in triple-negative breast cancer, run by GlaxoSmithKline, active and no longer recruiting.
A-BRAVE tested a year of the immunotherapy avelumab after standard treatment for high-risk triple-negative breast cancer at Italian and eight British hospitals. Relapse-free survival, the main endpoint, was not significantly improved; a later analysis found the benefit concentrated in tumours rich in immune cells.
NeoPACT tested a shorter, anthracycline-free pre-operative combination of carboplatin, docetaxel and pembrolizumab in triple-negative breast cancer. Tumours disappeared completely in 58 percent of women and 86 percent were free of events at three years, which is the basis for the SCARLET phase 3 comparison with the KEYNOTE-522 regimen.
c-TRAK TN, run from the Royal Marsden and the Institute of Cancer Research, was the first trial to use blood tests for tumour DNA to trigger treatment in triple-negative breast cancer. A quarter of patients tested positive within a year, but most of them already had visible metastases on scans, so the test came too late to prevent relapse with pembrolizumab.
BRE12-158 sequenced the cancer left behind after pre-operative chemotherapy and matched a targeted drug to it. Matched therapy was no better than the doctor's usual choice, which increasingly meant capecitabine, and blood-borne tumour DNA after surgery predicted who relapsed.
NeoTRIP added the immunotherapy atezolizumab to platinum chemotherapy before surgery without anthracyclines and did not significantly increase the share of women whose tumour disappeared (49 versus 44 percent); its main endpoint, whether fewer women relapse, is still awaited.
EA1131 asked whether platinum chemotherapy after surgery beats capecitabine for triple-negative breast cancer that survived pre-operative chemotherapy. It was stopped early because platinum was not going to prove better and was more toxic, so capecitabine stayed the standard.
SYSUCC-001 gave a year of low-dose daily capecitabine after standard chemotherapy for early triple-negative breast cancer in China. Fewer women relapsed at five years (83 versus 73 percent) and at ten years (78 versus 67 percent), but the improvement in survival itself did not reach statistical significance.
Trying to spare people radiotherapy by using a clear scan after two rounds of chemotherapy cost them about seven in a hundred in disease control, so the radiotherapy stayed.
In early Hodgkin lymphoma, people whose scan was still positive after two rounds did much better on more intensive chemotherapy, and people whose scan was clear still needed their radiotherapy.
Halving the chemotherapy and cutting the radiation dose by a third worked just as well for early Hodgkin lymphoma with favourable features, and caused fewer side effects.
A phase 3 trial of Lazertinib in non-small-cell lung cancer and small-cell lung cancer, run by Janssen Research & Development, LLC, active and no longer recruiting.
A phase 2 trial of Osimertinib and Savolitinib in advanced solid tumours, run by AstraZeneca, active and no longer recruiting.
A phase 3 trial of Lazertinib, Amivantamab, Pemetrexed and Carboplatin in non-small-cell lung cancer, run by Janssen Research & Development, LLC, active and no longer recruiting.
A phase 1/2 trial of Sunvozertinib in non-small-cell lung cancer and small-cell lung cancer, run by Dizal Pharmaceuticals, active and no longer recruiting.
A HER3 ADC delayed progression by only a few days more than chemotherapy and did not extend survival, so its US application was withdrawn.
A phase 2 trial of Dexamethasone, Methotrexate, Amivantamab and Lazertinib in non-small-cell lung cancer, run by Janssen Research & Development, LLC, active and no longer recruiting.
A phase 3 trial of Amivantamab, Pemetrexed and Carboplatin in non-small-cell lung cancer, run by Janssen Research & Development, LLC, active and no longer recruiting.
A phase 2 trial of Patritumab deruxtecan in non-small-cell lung cancer and small-cell lung cancer, run by Daiichi Sankyo, active and no longer recruiting.
For stage III lung cancers with an EGFR mutation, a targeted pill after chemoradiation cut progression by more than 80%.
Adding chemotherapy to osimertinib extended both progression-free and, in 2025, overall survival.
GARNET showed that the PD-1 antibody dostarlimab produced lasting responses in about four in ten women with mismatch repair-deficient endometrial cancer that had returned after chemotherapy, which won the drug its first approvals.
Adding chemotherapy to radiation after surgery helped women with high-risk endometrial cancer, especially those whose tumours have a broken p53 gene.
Mapping and removing only the first draining lymph nodes finds spread as reliably as removing all of them, with far less lymphoedema.
PORTEC-2 showed that a short course of internal radiotherapy to the top of the vagina prevented local recurrence of endometrial cancer as well as several weeks of external pelvic radiotherapy, with far fewer bowel side effects, and it became the standard for intermediate-risk disease.
MAJIC-ET found that ruxolitinib was no better than the usual second-line drugs at controlling platelets in ET, although it eased itching and other symptoms.
PT-1 is the trial that made hydroxyurea the first-line drug in high-risk ET: anagrelide gave more arterial clots, more serious bleeding and more progression to myelofibrosis.
A phase 2 trial of Abemaciclib and Temozolomide in ewing sarcoma, run by Eli Lilly and Company, active and no longer recruiting.
CABONE showed that the tablet cabozantinib could shrink or hold back both Ewing sarcoma and osteosarcoma that had come back after standard chemotherapy, the first drug to show meaningful activity in both bone cancers.
SARC028 tested the immunotherapy pembrolizumab across the common sarcoma types and found that it works in some, undifferentiated pleomorphic sarcoma above all, and barely at all in bone sarcomas such as osteosarcoma and Ewing sarcoma.
Tests whether adding the DLL3 T-cell engager to first-line maintenance can push median survival in extensive-stage disease past two years, as the phase 1b hinted.
DeLLphi-304 was the first trial in which a T-cell engager improved survival in a common solid tumour.
IMforte produced the first maintenance treatment ever approved for extensive-stage small-cell lung cancer, adding lurbinectedin to the immunotherapy that continues after chemotherapy.
A phase 3 trial of Pembrolizumab, Etoposide, Cisplatin, Atezolizumab and Carboplatin in small-cell lung cancer, run by Merck Sharp & Dohme LLC, active and no longer recruiting.
A phase 3 trial of Tiragolumab, Atezolizumab, Carboplatin and Etoposide in small-cell lung cancer, run by Hoffmann-La Roche, now completed.
A phase 2 trial of Durvalumab, Carboplatin, Cisplatin in small-cell lung cancer, run by AstraZeneca, active and no longer recruiting.
A phase 2 trial of Vorolanib and Atezolizumab in Extensive-stage small-cell lung cancer, run by Washington University School of Medicine, recorded as terminated on the registry.
In ASTRUM-005, a Chinese PD-1 antibody produced the longest first-line survival of the chemo-immunotherapy trials; it is now approved in Europe and the UK but not yet in the US.
A phase 3 trial of Rovalpituzumab tesirine, Topotecan and Dexamethasone in Small-cell lung cancer, run by AbbVie, completed.
A phase 3 trial of Rovalpituzumab tesirine and Dexamethasone in Small-cell lung cancer, run by AbbVie, recorded as terminated on the registry.
A phase 3 trial of Sodium thiosulfate in Extragonadal germ cell tumour, Hepatocellular carcinoma, Neuroblastoma and Ovarian cancer, run by Children's Oncology Group, completed.
ZUMA-5 showed that a single infusion of CD19 CAR-T cells put most people with heavily pretreated follicular lymphoma into remission, and that many of those remissions have lasted for years.
AUGMENT showed that pairing rituximab with the immune-modulating tablet lenalidomide kept relapsed follicular lymphoma under control for much longer than rituximab alone, giving patients a chemotherapy-free option.
The first randomised trial of first-line drug treatment for MALT lymphoma found that combining an old tablet with an antibody delayed relapse better than either alone, without anyone living longer.
FoRT asked whether two very small doses of radiotherapy control follicular lymphoma as well as the standard twelve; they do not, with about three times the rate of regrowth in the treated area, so 24 Gy stays the standard when the aim is lasting control and 4 Gy is kept for palliation.
A phase 3 trial of Tafasitamab, Rituximab, Lenalidomide in follicular lymphoma, run by Incyte Corporation, active and no longer recruiting.
Adding a targeted tablet to an antibody roughly doubled the chance of the lymphoma shrinking and tripled the time before it grew again, in people whose follicular lymphoma had already returned twice.
A single infusion of a patient's own reprogrammed immune cells cleared follicular lymphoma completely in about seven out of ten people who had already been through several treatments.
Two years of an antibody given every two months after chemotherapy more than doubled the time before follicular lymphoma came back, but after nine years the two groups were equally likely to be alive.
RELEVANCE tested whether a chemotherapy-free combination of rituximab and the pill lenalidomide could beat standard chemo-immunotherapy for untreated follicular lymphoma; it was not better, but it was just as effective with a different set of side effects, so it became an accepted alternative.
Radiotherapy can cure early follicular lymphoma, but most relapses happen outside the treated area. Adding a short course of drug treatment afterwards halved that risk.
GALLIUM showed that the newer anti-CD20 antibody obinutuzumab kept follicular lymphoma at bay for longer than rituximab when given with chemotherapy and as maintenance, at the cost of more serious side effects, and it became a first-line option.
A phase 2 trial of Tucatinib, Trastuzumab, CAPOX in gallbladder cancer, run by Seagen, a wholly owned subsidiary of Pfizer, active and no longer recruiting.
The first ADC against Claudin 18.2 to prove it extends life, announced July 2026, giving stomach cancer its first ADC beyond HER2.
A two-armed HER2 antibody beat Herceptin head-to-head as first-line treatment for HER2-positive stomach cancer, the first such win since 2010.
The first randomised proof that a HER2 drug beats standard chemotherapy in second-line stomach cancer: Enhertu added about three months of life.
A new target, FGFR2b, showed a survival gain at first look that shrank with longer follow-up, and the trial did not include the immunotherapy patients now routinely get.
Adding immunotherapy before and after surgery cut deaths in early stomach cancer; nearly seven in ten patients were alive at three years.
A phase 1/2 trial of Olaparib and Bevacizumab in ovarian cancer, small-cell lung cancer and gastric & gastro-oesophageal junction cancer, run by AstraZeneca, active and no longer recruiting.
DESTINY-Gastric02 repeated the trastuzumab deruxtecan study in patients from Europe and the United States, after the first trial had been run only in Japan and Korea, and confirmed that the drug produces meaningful responses in HER2-positive stomach cancer after trastuzumab.
Confirmed that adding a PD-1 blocker to first-line chemotherapy helps in stomach cancer, with the biggest gain in tumours rich in PD-L1.
A double-blind trial at Tata Memorial in India randomised 124 patients starting chemotherapy to 2.5 mg of olanzapine, a decades-old tablet costing a few cents a day, or placebo. Six in ten gained more than 5% of their weight against one in ten on placebo, and ASCO listed it in its 2024 cachexia guideline; it was small and single-centre.
The Chinese stomach cancer trial that showed adding sintilimab to chemotherapy helps patients live longer, especially when the tumour expresses PD-L1.
A phase 3 trial of Ripretinib and Sunitinib in gastrointestinal stromal tumour, run by Deciphera Pharmaceuticals, LLC, active and no longer recruiting.
GRID showed that regorafenib held back GIST that had already escaped both imatinib and sunitinib, and gave patients a third line of targeted treatment.
TROPHIMMUN was the first immunotherapy trial in gestational trophoblastic disease, a rare tumour that arises from a pregnancy; the PD-L1 antibody avelumab cured about half of the women whose disease had stopped responding to single-drug chemotherapy, with mild side effects and a healthy pregnancy afterwards in one patient.
CheckMate 498 asked whether nivolumab could replace temozolomide in the glioblastoma patients who gain least from it. Patients given nivolumab lived a shorter time.
CheckMate 548 added nivolumab to standard radiotherapy and temozolomide in newly diagnosed glioblastoma and found no benefit. With CheckMate 143 and 498 it makes three large negative trials: the immunotherapy that transformed melanoma and lung cancer did nothing in glioblastoma.
A phase 1/2 trial of ACP-196 in glioma & glioblastoma, run by Acerta Pharma BV, active and no longer recruiting.
CheckMate 143 was the first large randomised test of an immune checkpoint drug in glioblastoma. When the tumour came back, nivolumab kept patients alive no longer than bevacizumab.
The only randomised trial of a ketogenic diet in brain tumours found no benefit. Patients could follow the diet and their ketone levels rose, but progression came just as quickly.
A phase 2 trial of Infigratinib in Glioma & glioblastoma, run by Novartis Pharmaceuticals, completed.
HERBY asked whether adding the anti-blood-vessel antibody bevacizumab to radiotherapy and temozolomide would help children with high-grade brain tumours; it did not delay the tumours returning, so the drug is not part of standard treatment for these children.
The trial that set treatment for older patients with glioblastoma: three weeks of radiotherapy instead of six, with temozolomide added, extended survival without worsening quality of life.
ACT IV was a double-blind phase 3 test of rindopepimut, a vaccine against the EGFRvIII mutant protein, in 745 patients with newly diagnosed glioblastoma. It showed no benefit over a control vaccine, and because both arms beat historical expectations it taught the field how misleading historical-control comparisons can be.
The trial that made a wearable electric-field device part of glioblastoma care, extending median survival by about five months.
The first head-to-head trial of a radioligand against a targeted pill in neuroendocrine tumours; the radioligand won on progression-free survival.
A phase 3 trial of Lutetium-177 dotatate in pancreatic ductal adenocarcinoma, run by Advanced Accelerator Applications, active and no longer recruiting.
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.
ACNS0332 found that giving carboplatin alongside radiotherapy improved survival for children with the most aggressive group 3 medulloblastomas, while isotretinoin, tested in the same trial, did nothing and was dropped.
A phase 2 trial of Retifanlimab in head and neck squamous cell carcinoma, run by Incyte Biosciences International Sàrl, active and no longer recruiting.
The first new treatment for curable head and neck cancer in six years: immunotherapy around surgery doubled the time patients stayed free of events.
Attempts to give HPV-positive throat cancer patients less radiation fell short: the standard dose remained better, so de-escalation is not yet routine.
ROMAN showed that an infusion of avasopasem before each dose of radiotherapy cut severe mouth ulcers during head and neck chemoradiation from about two thirds of patients to about half and shortened them from 18 days to 8, but the effect was smaller than an earlier trial had promised and the FDA asked for another study before approval.
TrilynX tested xevinapant, a drug meant to make tumours more sensitive to radiation, with cisplatin chemoradiation in 730 patients with locally advanced head and neck cancer. Despite a striking phase 2 result, the phase 3 was stopped for futility in 2024 with worse outcomes on xevinapant, a reminder that early wins in this disease often do not replicate.
A phase 3 trial testing Monalizumab in head and neck squamous cell carcinoma, active and no longer recruiting.
KEYNOTE-412 tested adding pembrolizumab to standard chemoradiotherapy for locally advanced head and neck cancer; the improvement in keeping patients free of disease fell short of statistical significance, so chemoradiotherapy alone remains the standard.
CAPTAIN-1st showed that adding the PD-1 antibody camrelizumab to gemcitabine and cisplatin kept advanced nasopharyngeal cancer under control for about three months longer, confirming with a second drug what JUPITER-02 found with toripalimab.
Brought immunotherapy to nasopharyngeal cancer, an Epstein-Barr-virus-driven cancer common in southern China, and won the first US approval for that disease.
TPExtreme compared a shorter, docetaxel-based version of the standard EXTREME chemotherapy for advanced head and neck cancer with the original; patients lived about as long on either, and the shorter regimen was easier to tolerate, so it became an accepted alternative.
In 2026 a dual-immunotherapy regimen plus lenvatinib added to TACE cut the risk of progression by about 30%; whether it extends life is not yet known.
The first trial to show that adding immunotherapy and an anti-VEGF drug to TACE delays progression in intermediate-stage liver cancer.
Adding lenvatinib and pembrolizumab to TACE delayed progression by four and a half months but did not help patients live longer.
A phase 3 trial of Ethiodized oil in Hepatocellular carcinoma, run by Stanford University, recorded as terminated on the registry.
A PD-1 antibody plus an oral anti-VEGF pill beat sorafenib on survival, one of the largest benefits seen in liver cancer.
LEAP-002 randomised 794 patients with untreated advanced liver cancer to lenvatinib with or without pembrolizumab. Adding the immunotherapy did not lengthen life beyond lenvatinib alone, partly because the control arm did unusually well, a reminder that not every immunotherapy plus anti-angiogenic pairing works.
A Chinese trial showing that a PD-1 antibody plus a bevacizumab copy extends life in liver cancer, mirroring the global IMbrave150 result in a hepatitis B population.
The first trial in which a new pill beat sorafenib on survival in liver cancer, with a Chinese deuterated redesign of sorafenib itself.
Two large trials found radioactive beads were gentler than sorafenib but did not help patients live longer.
The first trial to show an overall survival benefit for a first-line ADC in triple-negative breast cancer.
Moved Enhertu ahead of chemotherapy in hormone-positive breast cancer and extended it to 'ultralow' HER2.
Created a new category of breast cancer, HER2-low, by showing Enhertu works in tumours previously called HER2-negative.
The trial that proved a TROP2 ADC could nearly double survival in heavily pretreated triple-negative breast cancer.
Beamion LUNG-1 showed that zongertinib, a pill that blocks mutant HER2 while sparing EGFR, shrinks about seven in ten HER2-mutant lung cancers after chemotherapy, and it became the first oral HER2 drug approved for lung cancer.
SOHO-01 is the study behind the second oral HER2 pill for lung cancer, approved in November 2025.
A phase 2 trial of Trastuzumab deruxtecan in non-small-cell lung cancer and small-cell lung cancer, run by AstraZeneca, active and no longer recruiting.
DESTINY-Lung02 confirmed that the antibody-drug conjugate trastuzumab deruxtecan shrinks about half of HER2-mutant lung cancers after chemotherapy and that the lower dose works as well with less lung inflammation.
DESTINY-Lung01 showed that the antibody-drug conjugate trastuzumab deruxtecan shrank tumours in more than half of people whose lung cancer carried a HER2 mutation, which led to the first approved HER2-targeted treatment for lung cancer.
The two trials that made immunotherapy, alone or with chemotherapy, the first treatment for most advanced lung cancers.
BWEL is a trial of more than 3,000 women testing whether losing weight after breast cancer treatment reduces recurrence. The programme achieved weight loss; the effect on recurrence was not clearly shown at the first analysis.
Showed Enhertu plus pertuzumab beats the decade-old first-line standard for HER2-positive metastatic breast cancer.
In HER2CLIMB-05, adding the brain-penetrant pill tucatinib to maintenance antibodies after first-line chemotherapy delayed progression by more than eight months.
A Chinese HER2 ADC cut the risk of progression by nearly 80% versus a pill-plus-chemotherapy standard, matching the scale of Enhertu's results.
A site-specifically conjugated HER2 ADC beat lapatinib-capecitabine on progression-free survival in China.
Adding tucatinib to Kadcyla helped a little, mostly in patients with brain metastases, but did not extend survival.
In the study behind Lumisight's approval, scanning the lumpectomy cavity with a fluorescent dye found cancer left behind by standard surgery in about one patient in thirteen and spared some women a second operation, with few false signals but a modest hit rate on individual margins.
Injecting a common local anaesthetic around the tumour minutes before surgery cut the risk of recurrence and death in a 1,583-woman Indian trial, at almost no cost.
The head-to-head ADC trial where Enhertu beat Kadcyla by a wide margin, showing that payload and bystander effect matter.
Adding a second HER2 antibody, pertuzumab, extended survival by 16 months, the largest gain ever seen in metastatic breast cancer at the time.
A phase 2/3 trial of Margetuximab, Retifanlimab and Trastuzumab in HER2-positive gastric cancer, run by MacroGenics, completed.
SORAYA showed that mirvetuximab soravtansine, an antibody-drug conjugate aimed at the folate receptor, shrank about a third of platinum-resistant ovarian cancers that had already been treated with bevacizumab, and earned the first approval of a biomarker-directed drug for this setting.
Operating again at first relapse, in carefully selected women, added about seven months of life.
Adding olaparib to bevacizumab maintenance roughly doubled progression-free time in tumours with faulty DNA repair, and improved survival in that group.
Extended PARP maintenance to women without BRCA mutations, but the survival benefit did not materialise on longer follow-up.
Two years of an olaparib pill after chemotherapy made a large share of women with BRCA-mutated ovarian cancer long-term survivors.
The first oral SERD to reduce recurrence after surgery, by about 30%, compared with today's hormone pills.
Extended CDK4/6 inhibitors to a broad group of early hormone-positive breast cancer patients, including node-negative.
The first CDK4/6 inhibitor to reduce recurrence after surgery in high-risk hormone-positive breast cancer.
Two large trials found that adding palbociclib after surgery does not prevent recurrence, even though the same drug helps in metastatic disease.
Postmenopausal women with a few positive lymph nodes and a low gene-test score can skip chemotherapy; premenopausal women still benefit from it.
Showed that most women with the commonest breast cancer can safely skip chemotherapy if a gene test says their risk is low or intermediate.
MINDACT showed that women whose early breast cancer looked high-risk to doctors but low-risk on the 70-gene MammaPrint test could skip chemotherapy: those who did met the trial's prespecified safety threshold, and about 46% of clinically high-risk women could avoid chemotherapy. Women aged 50 or under may still gain from it.
For younger women, shutting down the ovaries and adding an aromatase inhibitor prevents more recurrences than tamoxifen alone, with the largest gains in the highest-risk women.
GD2-CART01 was the first CAR-T to produce durable complete remissions in a childhood solid tumour: two-thirds responded and a third achieved complete remission.
NMTRC003 was the single-arm study, compared against historical patients, that got eflornithine approved as maintenance; the design remains debated.
Study 201 was the single-arm phase 2 of 74 patients behind naxitamab's approval for relapsed or refractory high-risk neuroblastoma in bone or bone marrow: given with GM-CSF as an outpatient, the antibody produced responses in half of patients. Without a randomised comparison against dinutuximab, equal efficacy is unproven.
Europe's long-running high-risk neuroblastoma trial set busulfan-melphalan as the transplant regimen and showed that adding IL-2 to anti-GD2 therapy added toxicity but not benefit.
COG ANBL0532 showed that two transplants in a row beat one in high-risk neuroblastoma.
The trial that made anti-GD2 immunotherapy standard for children with high-risk neuroblastoma, improving survival by about 20 points.
A phase 3 trial of Magrolimab and Azacitidine in Myelodysplastic syndromes / neoplasms, run by Gilead Sciences, recorded as terminated on the registry.
BMT CTN 1102 settled whether older people with higher-risk myelodysplastic syndrome should be offered a stem-cell transplant: those who had a matched donor and went to transplant were far more likely to be alive three years later than those without a donor who had drug treatment instead, so transplant belongs in the plan for fit patients aged 50 to 75.
AZA-001 was the trial that showed a drug could help people with higher-risk myelodysplastic syndromes live longer: azacitidine beat the best conventional care, and it has been the backbone of treatment for this disease ever since.
BMT CTN 0803 showed that people living with HIV whose lymphoma had come back can have the same high-dose chemotherapy and stem cell transplant as anyone else, with 87 percent alive at one year and outcomes no different from matched HIV-negative patients, so HIV alone should not bar transplant.
A phase 1/2 trial of Acalabrutinib, Rituximab, Lenalidomide in hodgkin lymphoma and non-hodgkin lymphoma, run by Acerta Pharma BV, active and no longer recruiting.
A phase 2 trial of Panobinostat in Hodgkin lymphoma, run by Novartis Pharmaceuticals, completed.
In KEYNOTE-204, PD-1 blockade beat the CD30 ADC head to head in relapsed Hodgkin lymphoma.
Established PD-1 blockade in relapsed Hodgkin lymphoma with ~70% response rates and seeded the frontline nivolumab-AVD idea.
In AETHERA, a year of the CD30 ADC after transplant halved relapse risk in high-risk patients.
E3311 showed that after robotic surgery for HPV-positive throat cancer, patients at intermediate risk could safely have a lower radiotherapy dose, with about 95 in 100 free of progression at two years, so surgery-first de-escalation became a recognised option.
ORATOR was the first randomised comparison of surgery and radiotherapy for early throat cancer; swallowing scores a year later were slightly better after radiotherapy, but the difference was smaller than patients would notice, so both remain reasonable choices.
In FOURLIGHT-1, a CDK4-only inhibitor designed to avoid the low blood counts of current CDK4/6 drugs improved progression-free survival in second line.
An oral SERD did not beat the aromatase inhibitor in first-line treatment when both were combined with palbociclib. Oral SERDs earn their place after resistance, not before it.
In evERA, pairing an oral SERD with everolimus after CDK4/6 failure delayed progression by more than three months, and by four and a half months in ESR1-mutant tumours.
The first trial to change treatment because of a blood test rather than a scan: switching to camizestrant when an ESR1 mutation appeared in the blood delayed progression by seven months.
The trial that got the first PROTAC approved, with benefit only in tumours with ESR1 mutations.
An oral SERD that works alone in ESR1-mutant tumours and, combined with abemaciclib, doubles progression-free time in everyone regardless of mutation.
Continuing CDK4/6 blockade with a different drug after the first one fails gives a small but real benefit.
A phase 2 trial of Giredestrant and Fulvestrant in HR-positive / HER2-negative breast cancer, run by Hoffmann-La Roche, active and no longer recruiting.
A phase 2 trial of Camizestrant and Fulvestrant in HR-positive / HER2-negative breast cancer, run by AstraZeneca, active and no longer recruiting.
Ripretinib kept fourth-line GIST under control six times longer than placebo and improved survival.
Established immunotherapy plus chemotherapy as first-line treatment for metastatic triple-negative breast cancer with PD-L1 expression.
ACTG A5263 tested whether two cheaper, easier chemotherapies could replace paclitaxel for people with HIV and advanced Kaposi sarcoma in Africa; both were clearly worse, so paclitaxel with antiretroviral therapy is the treatment to aim for wherever it can be supplied.
The first head-to-head trial among KRAS drugs: Roche's divarasib beat the two approved pills on progression and survival.
A phase 2 trial of JDQ443 in non-small-cell lung cancer, run by Novartis Pharmaceuticals, active and no longer recruiting.
Confirmed that the KRAS pill adagrasib beats chemotherapy after first-line treatment, though the gain is modest.
The first randomised trial of a KRAS drug: better than chemotherapy on progression, not on survival.
The trial that nearly doubled survival in previously treated pancreatic cancer, presented in the ASCO 2026 plenary. The biggest result in the disease's history.
CodeBreaK 100 gave the first KRAS inhibitor to 38 people whose pancreatic cancer carried the rare G12C mutation and had progressed after chemotherapy; one in five tumours shrank and patients lived a median 6.9 months, proof that KRAS can be drugged in this disease even though the target is uncommon here.
A phase 2 trial of Zenocutuzumab in non-small-cell lung cancer and pancreatic ductal adenocarcinoma, run by Partner Therapeutics, Inc., active and no longer recruiting.
In the minority of pancreatic cancers without a KRAS mutation, adding the EGFR antibody nimotuzumab to gemcitabine helped people live longer, and China approved it in 2023.
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.
In leiomyosarcoma, adding trabectedin to doxorubicin roughly doubled the time the disease stayed controlled, the first randomised first-line gain in this sarcoma in decades.
The first randomised trial in retroperitoneal sarcoma found that radiotherapy before surgery did not reduce recurrence in the abdomen, ending a decades-long debate for most patients.
A phase 2 trial of Ziv-aflibercept in Ovarian cancer, Uterine sarcoma, Uterine carcinosarcoma and Leiomyosarcoma, run by National Cancer Institute (NCI), completed.
A phase 2 trial of Eltrombopag and Lenalidomide in Leukaemia, run by Albert Einstein College of Medicine, completed.
A phase 3 trial of Azacitidine and Pevonedistat in Leukaemia, run by Takeda, completed.
A phase 3 trial of Glasdegib, CPX-351, Azacitidine and Cytarabine in Leukaemia, run by Pfizer, completed.
A phase 2 trial of Rasburicase in Leukaemia, run by M.D. Anderson Cancer Center, completed.
A phase 3 trial of Cladribine, Cyclophosphamide and Rituximab in Leukaemia, run by Hoffmann-La Roche, completed.
A phase 3 trial of Moxetumomab pasudotox in Leukaemia, run by MedImmune LLC, completed.
A phase 2 trial of Rituximab, Filgrastim and Granisetron in Leukaemia and Mantle cell lymphoma, run by Stanford University, completed.
A phase 2 trial of Daunorubicin, Cytarabine and Eltrombopag in Leukaemia, run by Novartis Pharmaceuticals, completed.
A phase 3 trial of Palifermin, Cyclophosphamide and Etoposide in Leukaemia, run by Swedish Orphan Biovitrum, completed.
ADRIATIC brought the first improvement in curative-intent small-cell lung cancer treatment in 30 years: a year or two of immunotherapy after chemoradiation lengthens life.
Settled the radiotherapy schedule debate in limited-stage disease: neither schedule was superior, so twice-daily 45 Gy remains standard and once-daily is an acceptable alternative.
Showed that giving the same dose of radiotherapy twice a day for three weeks, rather than once a day for five, raises the five-year survival of limited-stage small-cell lung cancer from 16 to 26 percent.
A phase 2 trial of Afamitresgene autoleucel in sarcomas, run by USWM CT, LLC, active and no longer recruiting.
A phase 2 trial of Plitidepsin in Liposarcoma, run by Institut Bergonié, recorded as terminated on the registry.
Oestrogen patches suppress testosterone as well as the standard injections, protect the bones instead of thinning them, and, contrary to what killed the idea in the 1970s, do not cause more heart attacks and strokes.
The UCLA and UCSF scan study behind the first US approval of gallium PSMA PET: in men heading for prostate surgery it was very reliable when it called a pelvic lymph node positive, but it still missed about three in five of the nodes that pathology later found.
An androgen deprivation tablet that works faster than the standard injection, wears off faster when stopped, and halved the rate of heart attacks and strokes.
Proved PSMA PET is far more accurate than conventional scans for staging, with less radiation.
The trial that showed PSA screening does stop some men dying of prostate cancer, and showed at the same time how many men have to be tested, and how many treated for a cancer that was never going to harm them, to achieve it.
The Scandinavian HYPO-RT-PC trial showed that seven large radiotherapy doses control intermediate- to high-risk prostate cancer as well as 39 conventional ones, with similar late side effects.
Adding chemotherapy to hormone therapy and radiotherapy for the most aggressive localised prostate cancer improved four-year survival from 89 to 93 percent.
The trial that proved an operation for prostate cancer can save a life, in men whose cancer was found because of symptoms rather than a blood test, and that the average gain is about three years.
Adding radioactive seeds inside the prostate on top of external radiotherapy halves the chance that the PSA comes back, but it does not make men live longer and it costs more urinary side effects.
A phase 2 trial of Samarium-153 lexidronam in Prostate cancer, run by Radiation Therapy Oncology Group, completed.
PACE-B showed that five stereotactic treatments control low- and intermediate-risk prostate cancer as well as four to eight weeks of conventional radiotherapy, with similar side effects at five years.
A phase 2 trial of Padeliporfin in Prostate cancer, run by Steba Biotech S.A., completed.
A phase 3 trial of Bicalutamide, Flutamide, Goserelin / leuprolide, Buserelin, Triptorelin and Degarelix in Prostate cancer, run by Radiation Therapy Oncology Group, completed.
In men whose prostate cancer was found by a blood test, having the operation did not clearly help them live longer, and it left many more of them incontinent or unable to get an erection.
Confirmed that four weeks of radiotherapy works as well as eight for intermediate-risk prostate cancer, without more long-term side effects.
CHHiP showed that four weeks of prostate radiotherapy in 3 Gy fractions is as effective as seven and a half weeks of conventional treatment, and 60 Gy in 20 fractions became the standard of care.
Showed that men with screen-detected prostate cancer die of it rarely, whether monitored or treated, establishing active surveillance.
Four months of hormone therapy added to radiotherapy helped men with intermediate-risk prostate cancer live longer, and did nothing for men with low-risk disease.
PRECISION proved that an MRI first finds more dangerous cancers with fewer biopsies.
A phase 3 trial of Padeliporfin in Prostate cancer, run by Steba Biotech S.A., completed.
Showed that men with low-risk prostate cancer can have five and a half weeks of radiotherapy instead of eight without losing control of the cancer, at the price of slightly more bowel and bladder trouble later.
CONKO-007 is the largest test of radiotherapy after chemotherapy for pancreatic cancer that cannot be removed at diagnosis: chemoradiotherapy did not raise the share of all patients reaching a clear-margin operation (25 against 18 percent) or lengthen survival, but among those operated the margins were clear more often (69 against 50 percent).
PANOVA-3 is the trial behind the 2026 approval of a wearable electric-field device for pancreatic cancer, the first new approval in locally advanced disease in decades.
CROSSFIRE is the only randomised comparison of the two local treatments offered after chemotherapy for pancreatic cancer that cannot be removed, MR-guided stereotactic radiotherapy and irreversible electroporation; survival was 16.1 against 12.5 months with no significant difference, the trial stopped early for futility, and neither has been shown to beat chemotherapy alone.
NEOLAP asked whether switching to FOLFIRINOX after two months of gemcitabine and nab-paclitaxel converts more inoperable pancreatic cancers to operable ones; about a third to 44 percent were removed with either sequence and survival was similar, so both induction regimens are acceptable.
PANFIRE-2 treated 50 people with electric-pulse ablation of pancreatic tumours that could not be removed; they lived a median 17 months from diagnosis, longer than the trial's historical target, but more than half had complications and nearly half relapsed locally, so the treatment stays in specialist centres and trials.
LAP07 tested whether adding radiotherapy after four months of chemotherapy helps people whose pancreatic cancer has not spread but cannot be removed; it did not lengthen life, although it did keep the tumour in check locally for longer, and adding erlotinib to gemcitabine did not help either.
CONKO-003 was the first randomised trial to show a second-line chemotherapy helps in pancreatic cancer: adding oxaliplatin to fluorouracil after gemcitabine failed lengthened median survival from 3.3 to 5.9 months, which is why oxaliplatin-based second-line treatment is in the NICE guideline. A later Canadian trial with a different oxaliplatin schedule found the opposite.
SCALOP is the UK trial that decided which chemotherapy to give with radiotherapy for pancreatic cancer that cannot be removed: after three months of induction chemotherapy, capecitabine during radiotherapy gave longer survival and far less blood toxicity than gemcitabine, and it is the radiosensitiser NICE recommends.
CONTINUUM was the first trial to show that adding a PD-1 immunotherapy, sintilimab, to the chemotherapy and radiotherapy given for advanced but not yet metastatic nasopharyngeal cancer keeps more people free of relapse three years later, 86 against 76 percent, at the cost of more side effects.
COMMANDS showed that luspatercept freed more people with lower-risk myelodysplastic syndromes from blood transfusions than the erythropoietin injections that had been the first-line standard for decades.
IMerge showed that imetelstat, a drug that blocks telomerase in the abnormal blood-forming cells, freed many people with lower-risk myelodysplastic syndromes from transfusions for months or years after erythropoietin had failed.
MEDALIST showed that luspatercept let a substantial share of people with ring sideroblast myelodysplastic syndromes stop needing blood transfusions after erythropoietin had failed, which earned the drug its first approval.
TELESTO is the only placebo-controlled trial of removing excess iron in people with lower-risk myelodysplastic syndrome who depend on blood transfusions; the chelator deferasirox lengthened the time before heart or liver damage, progression to leukaemia or death by about a year.
A phase 1/2 trial of Selpercatinib in non-small-cell lung cancer and small-cell lung cancer, run by Eli Lilly and Company, active and no longer recruiting.
The global test of whether ivonescimab plus chemotherapy beats the Keytruda-chemotherapy standard. An interim look in May 2026 fell short on progression; final results are due later in 2026.
A phase 3 trial of Nivolumab, Carboplatin, Cisplatin, Pemetrexed and Docetaxel in non-small-cell lung cancer, run by Bristol-Myers Squibb, active and no longer recruiting.
The first head-to-head trial in which a new drug beat Keytruda, in China; a survival benefit followed in 2026.
The first phase 3 win for ivonescimab, in lung cancer that has stopped responding to EGFR pills, and the basis of its first approval in China.
The single-arm study behind the first c-Met-directed antibody-drug conjugate approval in lung cancer: tumours shrank in about a third of patients whose cancers carried high c-Met protein, with eye and nerve side effects to watch.
A Chinese trial showing that adding the PD-1 antibody toripalimab to chemotherapy before and after lung cancer surgery more than doubles the time before the cancer comes back.
PACIFIC-2 asked whether starting durvalumab during chemoradiotherapy, rather than after it, would help people with stage III lung cancer; it did not, so the sequence established by the original PACIFIC trial stands.
CALGB 140503 showed that removing only part of a lobe is as good as removing the whole lobe for small peripheral lung cancers, so screen-detected tumours can be cured with less lung taken out.
Showed that immunotherapy given both before and after lung cancer surgery lengthens survival.
Cabozantinib tripled the time without progression in neuroendocrine tumours that had outgrown other treatments, leading to a 2025 approval.
SPINET tried to prove that the somatostatin analogue lanreotide slows lung carcinoid tumours, as it does gut tumours, but recruited too few patients to give a definitive answer; the small comparison favoured the drug and it is used on that basis.
Lanreotide more than halved the risk of progression in gut and pancreatic neuroendocrine tumours.
The two trials that made everolimus a standard pill for pancreatic, lung and gut neuroendocrine tumours.
Adding ifosfamide to doxorubicin shrank more sarcomas and delayed progression but did not significantly lengthen life, so doxorubicin alone stayed the standard unless a tumour needs to shrink.
A phase 1/2 trial of Sonrotoclax in mantle cell lymphoma, run by BeiGene, active and no longer recruiting.
A British and Nordic trial took chemotherapy out of first-line treatment for older people with mantle cell lymphoma and found the two-drug, chemotherapy-free combination worked better.
A phase 1/2 trial of Acalabrutinib in mantle cell lymphoma, run by AstraZeneca, active and no longer recruiting.
SYMPATICO showed that adding the BCL2 blocker venetoclax to ibrutinib for people whose mantle cell lymphoma had returned kept the disease under control for about ten months longer than ibrutinib alone, and the combination also produced complete responses in most untreated patients with the hard-to-treat TP53 mutation.
TRIANGLE showed that adding the pill ibrutinib to the chemotherapy given to younger people with mantle cell lymphoma keeps the disease away for longer, and that when ibrutinib is used the stem cell transplant that used to be compulsory no longer adds a clear benefit while adding side effects.
Adding a targeted tablet to first-line chemotherapy gave older people with mantle cell lymphoma about two and a half more years before the disease returned, but they did not live longer.
ZUMA-2 showed that CD19 CAR-T cells could produce deep and lasting remissions in mantle cell lymphoma that had stopped responding to BTK inhibitors, a group that previously had few options.
Three years of an antibody every two months after a stem cell transplant kept younger people with mantle cell lymphoma in remission longer and helped them live longer.
A phase 2 trial of Rituximab, Bortezomib and Ibritumomab tiuxetan in Mantle cell lymphoma, run by Duke University, recorded as terminated on the registry.
A phase 2 trial of Selpercatinib in thyroid cancer, run by Eli Lilly and Company, active and no longer recruiting.
EXAM showed that cabozantinib, which blocks RET, MET and VEGF receptors, held back progressive medullary thyroid cancer for far longer than placebo, and gave the disease its second approved targeted drug.
ZETA showed that vandetanib, a pill blocking the RET protein that drives most medullary thyroid cancers along with the tumour blood supply, kept advanced disease from growing for much longer than placebo, and it became the first drug ever approved for this rare cancer.
A phase 2 trial of Pembrolizumab in non-small-cell lung cancer and small-cell lung cancer, run by Merck Sharp & Dohme LLC, active and no longer recruiting.
A phase 2 trial of Nivolumab, Relatlimab + nivolumab in melanoma, run by Bristol-Myers Squibb, active and no longer recruiting.
INTerpath-001 was the first positive phase 3 trial of a personalised cancer vaccine, announced 19 August 2026.
Adding relatlimab to adjuvant nivolumab did not reduce recurrence, so the LAG-3 combination stays a treatment for measurable disease.
A phase 2 trial of Trastuzumab deruxtecan in melanoma, run by AstraZeneca, active and no longer recruiting.
Twenty melanoma patients took stool capsules from healthy donors before starting immunotherapy. Thirteen responded, more than usually expected, and the transplant was safe.
CheckMate 915 asked whether adding a low dose of ipilimumab to nivolumab after melanoma surgery would prevent more recurrences; it did not, and added toxicity, so single-agent PD-1 blockade remains the adjuvant standard.
Simply moving three doses of the same drug to before surgery improved outcomes, a result that changed how the field thinks about timing.
Fifteen melanoma patients whose immunotherapy had failed received a stool transplant from someone whose immunotherapy had worked, then restarted the drug. Six of them benefited, including three with lasting responses.
The first drug ever to extend life in metastatic uveal melanoma, and the first T-cell receptor-based medicine to win a phase 3.
Alliance A071401 showed that a twice-daily tablet blocking the enzyme FAK kept more meningiomas with a faulty NF2 gene from growing at six months than history would predict, in both lower-grade and higher-grade tumours, the first mutation-matched treatment to show activity in this tumour.
The third PD-1 antibody tested in this rare and fast-growing skin cancer shrank it in more than half of the people who took it, and most of those responses lasted years. Six in ten were still alive three years later, in a disease that used to kill within months.
Merkel cell carcinoma comes back in a quarter of people even after the surgeon has removed it all. Giving immunotherapy afterwards for a year reduced that by about ten in every hundred, but the trial was small and cannot yet say whether anyone lived longer.
JAVELIN Merkel 200 showed that the PD-L1 antibody avelumab shrinks about a third of Merkel cell carcinomas that have already failed chemotherapy, most of them durably, and it produced the first approved drug for this cancer.
KEYNOTE-017 showed that pembrolizumab given as the first treatment shrinks more than half of advanced Merkel cell carcinomas, whether or not the tumour carries the Merkel cell polyomavirus.
The phase 3 test of durvalumab with chemotherapy was stopped early and did not meet its goal, despite an encouraging earlier study.
Adding atezolizumab to bevacizumab-chemotherapy slowed progression but did not lengthen survival overall; it did help patients with non-epithelioid tumours.
MARS 2 is the trial that overturned decades of surgical practice: removing the lining of the lung did not help patients live longer and left them worse off.
The one trial still testing an antibody-drug conjugate in mesothelioma, combining it with PD-1 blockade on the theory that payload-induced cell death primes an immune response.
Showed that adding a PD-1 blocker to standard chemotherapy helps in mesothelioma, giving a second immunotherapy-based first-line option.
The first randomised test of an antibody-drug conjugate in mesothelioma: no better than vinorelbine, which is why no ADC is approved for this cancer.
CheckMate 743 was the first immunotherapy trial to lengthen survival in mesothelioma, and gave the first new first-line option in 16 years.
LUME-Meso randomised 458 patients with epithelioid pleural mesothelioma to first-line cisplatin and pemetrexed with nintedanib or placebo. The phase 2 progression signal for the multi-kinase inhibitor vanished in phase 3, with no delay in progression and no gain in life, a lesson in the unreliability of small phase 2 signals in this disease.
STELLAR is the small single-arm study behind the device approval of tumour treating fields in mesothelioma.
In MAPS, adding the anti-VEGF antibody bevacizumab to chemotherapy lengthened survival by about three months, the first improvement after pemetrexed.
A phase 2 trial of Osimertinib, Savolitinib in non-small-cell lung cancer and small-cell lung cancer, run by AstraZeneca, active and no longer recruiting.
A phase 2 trial of Enfortumab vedotin and Pembrolizumab in advanced solid tumours, run by Astellas Pharma Global Development, Inc., active and no longer recruiting.
A phase 2 trial of Olaparib in advanced solid tumours, run by Merck Sharp & Dohme LLC, active and no longer recruiting.
A phase 2 trial of Sacituzumab govitecan in advanced solid tumours, run by Gilead Sciences, active and no longer recruiting.
A phase 2 trial of Durvalumab and Gemcitabine in advanced solid tumours, run by AstraZeneca, active and no longer recruiting.
A phase 2 trial of Tucatinib, Trastuzumab and Capecitabine in advanced solid tumours, run by Pfizer, active and no longer recruiting.
A phase 1/2 trial of Monalizumab, Durvalumab, Cetuximab, FOLFOX (5-FU, leucovorin, oxaliplatin) and Bevacizumab in advanced solid tumours, run by MedImmune LLC, active and no longer recruiting.
A phase 2 trial of Ramucirumab, Cisplatin, Gemcitabine in metastatic cancer, run by Eli Lilly and Company, active and no longer recruiting.
A phase 3 trial of Abemaciclib and Fulvestrant in advanced solid tumours, run by Eli Lilly and Company, active and no longer recruiting.
A phase 2 trial of Abemaciclib and Tamoxifen in advanced solid tumours, run by Eli Lilly and Company, active and no longer recruiting.
HORRAD found no overall survival benefit from irradiating the prostate in men presenting with bone metastases, though a possible gain in those with few metastases fed into STAMPEDE's later positive finding.
Combining the bone-seeking radioactive drug radium-223 with enzalutamide made men live about six months longer, and the fractures that sank the earlier version of this idea were prevented by giving bone-protecting drugs to everyone.
A phase 2/3 trial of Abemaciclib, Abiraterone acetate, Prednisone in prostate cancer, run by Eli Lilly and Company, active and no longer recruiting.
Curium's PSMA radioligand met its progression endpoint; survival data and an FDA filing are pending.
Adding pembrolizumab to enzalutamide did not help. The trial was stopped for futility, and three times as many men had a serious side effect.
Adding pembrolizumab to chemotherapy for advanced prostate cancer did not work, and it added side effects and lung inflammation.
A phase 3 trial of Cabozantinib, Atezolizumab, Abiraterone acetate in prostate cancer, run by Exelixis, active and no longer recruiting.
A phase 3 trial of Olaparib, Abiraterone acetate in prostate cancer, run by AstraZeneca, active and no longer recruiting.
In SPLASH, a second PSMA radioligand improved progression-free survival but not, so far, survival.
A phase 2 trial of Niraparib, Cetrelimab, Abiraterone acetate and Prednisone in prostate cancer, run by Janssen Research & Development, LLC, active and no longer recruiting.
PSMAfore moved Pluvicto before chemotherapy in prostate cancer.
A phase 3 trial of Abiraterone acetate, Niraparib and Prednisone in prostate cancer, run by Janssen Research & Development, LLC, active and no longer recruiting.
CAPItello-281 delivered the first AKT inhibitor success in prostate cancer, for the ~25% of men whose tumours have lost PTEN on the trial's own immunohistochemistry cut-off.
The last attempt to make immunotherapy work in prostate cancer, this time as early as possible. It was stopped for futility and made rashes and serious side effects much commoner.
PSMAddition moved the radioligand Pluvicto into the first treatment of metastatic hormone-sensitive prostate cancer, given alongside hormone therapy, and the FDA approved this use on 31 July 2026. It delayed progression on scans, but severe side effects were more common and whether it lengthens life is not yet known.
ARANOTE showed that darolutamide added to hormone therapy alone, without chemotherapy, delayed the progression of newly metastatic prostate cancer on scans, extending the drug's use to men who cannot or do not want to have docetaxel.
A phase 3 trial of Enzalutamide in prostate cancer, run by Astellas (Beijing) Pharmaceutical R&D Co., Ltd., active and no longer recruiting.
Proved 'triplet therapy': adding darolutamide to hormone therapy plus chemotherapy reduces death by a third.
PEACE-1 is the European triplet trial: abiraterone added to hormone therapy and docetaxel improves survival, especially in high-volume disease.
A phase 3 trial of Apalutamide in prostate cancer, run by Aragon Pharmaceuticals, Inc., active and no longer recruiting.
Brought enzalutamide into first-line metastatic treatment, with an overall survival benefit confirmed in 2021.
A randomised phase 2 found that adding the GSK-3 inhibitor elraglusib to standard chemotherapy lengthened survival in metastatic pancreatic cancer; a phase 3 is needed to confirm it.
Adding the metabolism drug devimistat to FOLFIRINOX did not help people with metastatic pancreatic cancer live longer.
The anti-stroma antibody pamrevlumab did not help people with locally advanced pancreatic cancer live longer.
CanStem111P is the largest first-line pancreatic cancer trial ever run, 1,134 patients, and it found that the stemness inhibitor napabucasin added nothing to nab-paclitaxel and gemcitabine; the trial was stopped for futility and its control arm is now the best description of what that chemotherapy achieves.
The first head-to-head trial of the two chemotherapy backbones, won narrowly by the four-drug regimen.
PANOPTIMOX showed that people whose pancreatic cancer is controlled after four months of FOLFIRINOX can drop to a gentler fluorouracil maintenance without losing survival, living longer before their quality of life fell; the alternating gemcitabine strategy did worse.
RESOLVE tested the leukaemia drug ibrutinib as a way to reprogramme the immune cells around pancreatic tumours; added to nab-paclitaxel and gemcitabine it shortened the time to progression and cut the chemotherapy patients could tolerate, with no survival gain.
Eryaspase, an enzyme carried inside red blood cells, did not lengthen survival when added to second-line chemotherapy for pancreatic cancer.
HALO-301 was the phase 3 test of breaking down the dense stroma of pancreatic tumours with a hyaluronidase enzyme so chemotherapy could reach them; response rates went up but survival did not move, and the stroma-busting idea in this form was abandoned.
ECLIPSE was the randomised test of the Johns Hopkins pancreatic cancer vaccine strategy, a whole-cell GVAX vaccine followed by a Listeria vector carrying mesothelin; after promising early results it did not beat chemotherapy, with survival of 3.7 months against 4.6, and the programme ended.
FIRSTMAPPP was the first randomised trial ever completed in metastatic phaeochromocytoma and paraganglioma, rare hormone-producing tumours; the kinase inhibitor sunitinib kept 36 percent of patients free of progression at a year against 19 percent on placebo, giving the disease its first drug with randomised evidence.
A phase 1/2 trial of Durvalumab, Capivasertib, Oleclumab, Trastuzumab deruxtecan and Datopotamab deruxtecan in triple-negative breast cancer, run by AstraZeneca, active and no longer recruiting.
The first phase 3 win for a bispecific ADC, in triple-negative breast cancer, announced February 2026.
A phase 3 trial of Capivasertib in triple-negative breast cancer, run by AstraZeneca, active and no longer recruiting.
A phase 1/2 trial of Datopotamab deruxtecan in non-small-cell lung cancer and triple-negative breast cancer, run by AstraZeneca, active and no longer recruiting.
Moved Trodelvy into first-line use for triple-negative patients who cannot receive immunotherapy.
Showed that pairing an ADC with immunotherapy beats chemotherapy plus immunotherapy in first-line PD-L1-positive TNBC.
IPATunity130 tried to confirm a promising early signal for the AKT-blocking tablet ipatasertib with paclitaxel in triple-negative breast cancers carrying PI3K-pathway mutations. It failed: progression and survival were the same with or without the drug, and the ipatasertib programme in breast cancer ended.
A phase 2 trial of Olaparib and Ceralasertib in triple-negative breast cancer, run by AstraZeneca, active and no longer recruiting.
KEYNOTE-119 tested pembrolizumab on its own against chemotherapy as second- or third-line treatment for metastatic triple-negative breast cancer. It did not lengthen life, even in tumours with high PD-L1, which is why immunotherapy in this disease is given with chemotherapy or an antibody-drug conjugate and only in the first line.
IMpassion131 was the sister trial to the first immunotherapy success in breast cancer. It failed, and the approval it was meant to confirm was withdrawn.
CAPP2 followed people with Lynch syndrome, an inherited condition that carries a very high risk of bowel cancer, for ten years after two to four years on aspirin or placebo: those who took aspirin developed about a third fewer bowel cancers, which is why daily aspirin is now offered to people with the syndrome.
Showed that a drug built specifically for the RET mutation beats the older multi-target pills in medullary thyroid cancer, with far fewer side effects.
A phase 1/2 trial of Iberdomide, Dexamethasone, Daratumumab in multiple myeloma, run by Celgene, active and no longer recruiting.
A phase 1/2 trial of Belantamab mafodotin, Nirogacestat, Pomalidomide in multiple myeloma, run by GlaxoSmithKline, active and no longer recruiting.
A phase 1/2 trial of Belantamab mafodotin in multiple myeloma, run by GlaxoSmithKline, active and no longer recruiting.
A phase 1/2 trial of Belantamab mafodotin, Isatuximab in multiple myeloma, run by GlaxoSmithKline, active and no longer recruiting.
A phase 1/2 trial of Belantamab mafodotin, Nirogacestat in multiple myeloma, run by GlaxoSmithKline, active and no longer recruiting.
A phase 1/2 trial of Belantamab mafodotin, Nirogacestat, Lenalidomide in multiple myeloma, run by GlaxoSmithKline, active and no longer recruiting.
A phase 3 trial of Belantamab mafodotin, Pomalidomide, Dexamethasone in multiple myeloma, run by GlaxoSmithKline, active and no longer recruiting.
A phase 3 trial of Belantamab mafodotin, Daratumumab, Bortezomib in multiple myeloma, run by GlaxoSmithKline, active and no longer recruiting.
iMMagine-1 is the pivotal study behind anito-cel's BLA, with responses in nearly every patient.
A phase 3 trial of Belantamab mafodotin, Daratumumab, Bortezomib in multiple myeloma, run by GlaxoSmithKline, active and no longer recruiting.
A phase 3 trial of Luspatercept in myeloproliferative neoplasms, run by Celgene, active and no longer recruiting.
VERIFY showed that weekly rusfertide, a hepcidin mimetic, freed most PV patients from phlebotomy and improved their symptoms, leading to the drug's approval in 2026.
MANIFEST-2 found that adding the BET inhibitor pelabresib to ruxolitinib shrank spleens in nearly twice as many people with myelofibrosis, but the improvement in symptoms did not reach statistical significance, leaving the drug's approval uncertain.
MOMENTUM showed that momelotinib, a JAK inhibitor that also eases anaemia, relieved symptoms and shrank spleens better than danazol in people with myelofibrosis who were anaemic after earlier JAK inhibitor treatment.
PROUD-PV and its extension showed that ropeginterferon matches hydroxyurea in the first year and then pulls ahead, with more complete responses and far more molecular responses by three years.
RESPONSE showed that ruxolitinib controls the haematocrit and shrinks the spleen in PV patients hydroxyurea has failed, and won the first drug approval specific to that setting.
CYTO-PV settled the most basic question in PV: keeping the haematocrit under 45 percent gives far fewer serious clots and cardiovascular deaths than a looser target.
COMFORT-I showed that the JAK-blocking pill ruxolitinib shrank the grossly enlarged spleens of people with myelofibrosis in about four in ten patients, against almost none on placebo, and eased their fatigue, night sweats and itching; it led to the first approved drug for the disease.
COMFORT-II was the European companion to COMFORT-I: ruxolitinib shrank the spleen by more than a third in 28 percent of people with myelofibrosis after nearly a year, while not one patient on the best treatment their doctor could otherwise offer achieved that, and symptoms and quality of life improved.
An alpha-emitting radioligand met all its primary endpoints, with responses in more than half of patients new to radioligand therapy.
In the SANET trials, a Chinese anti-angiogenic pill slowed both gut and pancreatic neuroendocrine tumours, but its US application was refused.
The trial that made radioligand therapy a standard: a radioactive drug homing to the somatostatin receptor held midgut neuroendocrine tumours in check far longer than high-dose octreotide, and it underpins Lutathera's approvals.
Showed for the first time that a hormone-suppressing injection also slows neuroendocrine tumour growth.
A phase 1/2 trial of Avelumab in penile cancer, run by Transgene, active and no longer recruiting.
A phase 2 trial of Brentuximab vedotin, Cyclophosphamide, Doxorubicin and Prednisone in non-Hodgkin lymphoma, run by Takeda, active and no longer recruiting.
A phase 2 trial of TAK-007 in non-Hodgkin lymphoma, run by Takeda, active and no longer recruiting.
A phase 2 trial of Valemetostat Tosylate in peripheral T-cell lymphomas and non-Hodgkin lymphoma, run by Daiichi Sankyo, active and no longer recruiting.
ASPEN compared two BTK inhibitors head to head in Waldenström macroglobulinaemia: zanubrutinib did not produce statistically more deep responses than ibrutinib, but it caused far fewer heart rhythm problems and other side effects, which is why many guidelines now prefer it.
A phase 2 trial of Ibritumomab tiuxetan and Rituximab in Non-Hodgkin lymphoma, run by University of Wisconsin, Madison, recorded as terminated on the registry.
PTLD-1 established how to treat lymphoma that arises after an organ transplant: start with the antibody rituximab alone, and use how well the patient responds to decide whether to continue rituximab or move to chemotherapy; seven in ten reached complete remission and patients lived a median of more than six years.
An asparaginase-based regimen gave a response in four out of five people with an aggressive nasal lymphoma that had always resisted standard chemotherapy, and nearly all of them had dangerously low white cell counts.
The only randomised trial ever run exclusively in the virus-driven T-cell leukaemia found an intensive Japanese regimen better than standard chemotherapy, at the cost of much more severe low blood counts.
ARAMIS showed that darolutamide, an androgen receptor blocker that barely enters the brain, delayed metastases and lengthened life in men whose prostate cancer was rising on hormone therapy but not yet visible on scans, with side effects close to placebo.
A phase 3 trial of Apalutamide in prostate cancer, run by Aragon Pharmaceuticals, Inc., active and no longer recruiting.
PROSPER showed that enzalutamide delayed the appearance of metastases by about two years, and later helped men live longer, when given at the point where prostate cancer is rising on hormone therapy but has not yet spread on scans.
GETUG 13 showed that men with the worst-risk testicular cancers whose tumour markers fall too slowly after the first cycle of chemotherapy do better if treatment is intensified: 59 percent were free of progression at three years against 48 percent with standard BEP, and fewer needed high-dose salvage chemotherapy.
A phase 3 trial of Osimertinib, Cisplatin, Carboplatin in non-small-cell lung cancer and small-cell lung cancer, run by AstraZeneca, active and no longer recruiting.
A phase 3 trial of Osimertinib in non-small-cell lung cancer and small-cell lung cancer, run by AstraZeneca, active and no longer recruiting.
A phase 2/3 trial testing Cobolimab in non-small-cell lung cancer, active and no longer recruiting.
A phase 3 trial of Durvalumab in non-small-cell lung cancer, run by AstraZeneca, active and no longer recruiting.
A phase 3 trial of Pemetrexed, Cisplatin, Carboplatin in non-small-cell lung cancer and small-cell lung cancer, run by Merck Sharp & Dohme LLC, active and no longer recruiting.
A phase 3 trial of Pemetrexed, Cisplatin, Carboplatin in non-small-cell lung cancer and small-cell lung cancer, run by Merck Sharp & Dohme LLC, active and no longer recruiting.
A phase 2 trial of Durvalumab, Ceralasertib, Olaparib in non-small-cell lung cancer and small-cell lung cancer, run by AstraZeneca, active and no longer recruiting.
A phase 3 trial of Sacituzumab govitecan, Docetaxel in non-small-cell lung cancer and small-cell lung cancer, run by Gilead Sciences, active and no longer recruiting.
A phase 2 trial of Tusamitamab ravtansine, Pembrolizumab, Cisplatin, Carboplatin and Pemetrexed in Non-small-cell lung cancer, run by Sanofi, recorded as terminated on the registry.
A lung cancer trial that planned to start durvalumab the moment a surveillance blood test found tumour DNA, before any scan showed relapse; the registry lists it as complete with 30 participants.
ESOPEC pitted the two standard ways of treating operable oesophageal adenocarcinoma against each other, chemotherapy before and after surgery or chemoradiotherapy before it, and found that the chemotherapy approach helped people live longer.
Added immunotherapy to first-line chemotherapy for all types of oesophageal cancer, with a survival benefit that held at five years.
The first bispecific ADC to extend both progression-free and overall survival in oesophageal cancer, for patients whose immunotherapy has stopped working.
For the third of patients whose tumour vanishes after chemoradiation, watching closely and operating only if it comes back gave the same survival as immediate surgery.
The trial that added toripalimab to chemotherapy for oesophageal cancer in China and showed a survival gain regardless of PD-L1 level.
The global trial in which tislelizumab beat chemotherapy for oesophageal cancer that had progressed after first treatment, which carried it to approval in the US, Europe and Japan.
RATIONALE-306 is the trial behind tislelizumab's US approval for squamous oesophageal cancer, with the biggest gains in PD-L1-positive tumours.
Showed two immunotherapy options for squamous oesophageal cancer, including one with no chemotherapy at all, both extending survival.
ESCORT-1st is China's first-line immunotherapy trial for squamous oesophageal cancer, one of five that together made chemo-immunotherapy the global standard.
A phase 2 trial of Mifamurtide and Ifosfamide in Osteosarcoma, run by University of Oxford, recorded as terminated on the registry.
A phase 2 trial of Samarium-153 lexidronam in Osteosarcoma, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, recorded as terminated on the registry.
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.
A phase 1/2 trial of Mirvetuximab soravtansine in ovarian cancer, run by Takeda, active and no longer recruiting.
A phase 3 trial of Niraparib and Dostarlimab in ovarian cancer, run by Tesaro, Inc., active and no longer recruiting.
After a decade of failures, the first immunotherapy trial to extend survival in ovarian cancer, leading to the first checkpoint-inhibitor approval in the disease.
Blocking cortisol signalling in tumour cells made chemotherapy work better and extended life in resistant ovarian cancer.
RAMP 201 produced the first approved treatment designed for low-grade serous ovarian cancer, a slow-growing type driven by the RAS pathway that shrugs off chemotherapy.
Adding immunotherapy and olaparib to bevacizumab slowed progression but has not improved survival, and the regimen is not approved.
The first ADC to extend life in ovarian cancer, for the roughly one-third of tumours with high folate receptor alpha.
A third PARP inhibitor showed the same pattern: longer disease control after first-line chemotherapy, biggest in tumours with faulty DNA repair.
The largest screening trial ever run for ovarian cancer found earlier detection but no reduction in deaths, so there is still no recommended screening.
A phase 3 trial of Olaparib in ovarian cancer, run by AstraZeneca, active and no longer recruiting.
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.
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.
The randomised test of an off-the-shelf KRAS vaccine after pancreatic surgery. It missed its primary goal in June 2026.
NORPACT-1 asked whether giving FOLFIRINOX chemotherapy before surgery helps people whose pancreatic cancer can be removed straight away; it did not, and patients who went straight to surgery were if anything more likely to be alive at 18 months, so upfront surgery remains the standard for clearly resectable disease.
APACT tested whether adding nab-paclitaxel to gemcitabine after pancreatic surgery delays relapse; by the independent radiologists' reading it did not (19.4 against 18.8 months), although patients on the combination lived about four months longer at five years, so the regimen is not a formal adjuvant standard.
This is the small New York trial behind the excitement about personalised mRNA cancer vaccines: half of the 16 patients vaccinated after pancreatic surgery made strong T cells against their own tumour mutations, and those responders had far fewer relapses after more than three years, with vaccine-induced T cells predicted to last for years.
NEONAX compared giving gemcitabine and nab-paclitaxel around surgery with giving it only afterwards; neither arm reached the trial's 55 percent target for being relapse-free at 18 months, but patients treated before surgery lived a median 25.5 months against 16.7, mostly because so few in the surgery-first arm were ever fit enough to start chemotherapy.
SWOG S1505 gave people with operable pancreatic cancer three months of chemotherapy before and after surgery with one of the two modern regimens; about half were alive at two years with either, no better than the 40 percent threshold the trial set from adjuvant-only history, and a third of the patients enrolled turned out not to have resectable tumours on central review.
CONKO-005 was the first adjuvant trial to add a targeted drug to chemotherapy after pancreatic surgery; six months of erlotinib with gemcitabine made no difference to the time before relapse (11.4 months in both arms) or to survival.
ESPAC-4 showed that adding the tablet capecitabine to gemcitabine after pancreatic cancer surgery lengthened median survival from 25.5 to 28 months, and the pair became the adjuvant standard for people who cannot tolerate the harsher FOLFIRINOX regimen.
JASPAC 01 found that six months of the oral fluoropyrimidine S-1 after pancreatic cancer surgery nearly doubled five-year survival compared with gemcitabine in Japanese patients, 44 against 24 percent, which made S-1 the adjuvant standard in Japan; the result has never been tested in Western patients, who metabolise the drug differently.
CONKO-001 was the trial that made chemotherapy after pancreatic cancer surgery routine: six months of gemcitabine doubled the time before the cancer came back and roughly doubled the share of patients alive at five years, from 10 to 21 percent.
PAPMET was the first randomised trial dedicated to papillary kidney cancer; cabozantinib held the disease back for longer than sunitinib, the previous default, while two more selective MET inhibitors did not.
A phase 3 trial of Savolitinib and Sunitinib in renal cell carcinoma, run by AstraZeneca, active and no longer recruiting.
A single-arm trial of albumin-bound sirolimus in the ultra-rare tumour PEComa produced lasting responses in about four in ten patients and won the first approval for the disease.
A phase 2 trial of Lutetium-177 dotatate in pheochromocytoma and paraganglioma, run by Advanced Accelerator Applications, active and no longer recruiting.
A phase 3 trial of Osilodrostat in Pituitary tumours, run by Novartis Pharmaceuticals, completed.
A phase 2 trial of Osilodrostat in Pituitary tumours, run by Novartis Pharmaceuticals, completed.
A phase 3 trial of Somatostatin analogues and Pegvisomant in Pituitary tumours, run by Ipsen, completed.
A phase 3 trial of Pasireotide in Pituitary tumours, run by Novartis Pharmaceuticals, completed.
A phase 3 trial of Pasireotide and Somatostatin analogues in Pituitary tumours, run by Novartis Pharmaceuticals, completed.
A phase 3 trial of Pasireotide in Pituitary tumours, run by Novartis Pharmaceuticals, completed.
A phase 2 trial of Busulfan, Gemcitabine, Melphalan and Panobinostat in Plasma cell leukaemia, run by M.D. Anderson Cancer Center, completed.
MAJIC-PV, a UK academic trial, found that ruxolitinib gave more complete responses than best available therapy and, for the first time, that a complete response went with fewer clots and progressions.
Low-PV asked whether even low-risk patients gain from interferon: more of them stayed at the haematocrit target without progression when ropeginterferon was added to phlebotomy.
RESPONSE-2 extended ruxolitinib's benefit to PV patients without an enlarged spleen: three times as many reached haematocrit control.
IELSG37 showed that people with primary mediastinal B-cell lymphoma whose scan is clear after chemotherapy do not need radiotherapy to the chest, sparing young patients decades of heart and breast cancer risk.
A phase 2 trial of Fostamatinib and Ruxolitinib in Primary myelofibrosis, run by Washington University School of Medicine, recorded as terminated on the registry.
The first multicentre randomised trial of protons versus IMRT for localised prostate cancer found no difference in bowel, urinary or sexual quality of life and the same cancer control at five years.
A phase 3 trial of Abiraterone acetate and Prednisone in Prostate cancer, run by Janssen Research & Development, LLC, completed.
A phase 3 trial of Degarelix in Prostate cancer, run by Ferring Pharmaceuticals, recorded as terminated on the registry.
In GÖTEBORG-2, 17,980 men were screened with PSA, then MRI; biopsying only what the MRI showed halved the detection of harmless prostate cancers (0.6% versus 1.2% of men) while the detection of dangerous cancers barely changed (0.9% versus 1.1%). It is the strongest evidence that MRI-first screening cuts overdiagnosis.
POP-RT, from Mumbai, showed that treating the pelvic lymph nodes as well as the prostate improves disease control in high-risk prostate cancer, reviving a question earlier trials had left unanswered.
A phase 3 trial of Choline C-11 in Prostate cancer, run by Global Isotopes, LLC d/b/a Zevacor Molecular, completed.
RTOG 9601 showed that adding two years of hormone therapy to salvage radiotherapy after prostate surgery lengthens survival, mainly in men with higher PSA levels at relapse.
A phase 3 trial of Docetaxel, Doxorubicin, Estramustine, Prednisone, Vinblastine and Dexamethasone in Prostate cancer, run by M.D. Anderson Cancer Center, recorded as terminated on the registry.
A phase 2 trial of Samarium-153 lexidronam and Sargramostim in Prostate cancer, run by National Cancer Institute (NCI), completed.
A phase 3 trial of Degarelix, Goserelin / leuprolide and Bicalutamide in Prostate cancer, run by Ferring Pharmaceuticals, completed.
In LITESPARK-011, belzutifan plus lenvatinib held advanced clear-cell kidney cancer in check for about four months longer than cabozantinib in people whose cancer had grown after immunotherapy, but at the interim look it had not been shown to help them live longer; a final survival analysis is still to come.
Adding belzutifan to first-line lenvatinib-pembrolizumab did not deliver, a reminder that later-line success does not guarantee first-line benefit.
Adding the HIF-2α pill to a year of adjuvant pembrolizumab further reduced recurrence, and became the first adjuvant combination approved in kidney cancer.
A phase 2 trial of Belzutifan in renal cell carcinoma, run by Merck Sharp & Dohme LLC, active and no longer recruiting.
A second trial confirming that restarting immunotherapy after it fails does not help kidney cancer patients.
Continuing immunotherapy after it has failed, alongside a targeted pill, added nothing but side effects. The first definitive test of 'IO rechallenge'.
The HIF-2α inhibitor beat the old standard everolimus after immunotherapy and targeted therapy had failed, with durable responses in a subset.
The first triplet trial in kidney cancer slowed progression but did not help patients live longer, and added toxicity.
The combination with the longest progression-free survival ever reported in first-line kidney cancer, at nearly two years.
Nivolumab with cabozantinib doubled progression-free survival versus sunitinib and lengthened life, with quality of life preserved.
A phase 2 trial of Apalutamide in salivary gland cancers, run by Janssen Pharmaceutical K.K., active and no longer recruiting.
A phase 1/2 trial of Selpercatinib in thyroid cancer and sarcomas, run by Eli Lilly and Company, active and no longer recruiting.
IGNYTE-ESO was the pivotal study for the second sarcoma TCR-T, with responses in 42% of patients with two rare sarcomas.
MOTION randomised 123 patients with symptomatic tenosynovial giant cell tumour not suitable for surgery to the CSF1R inhibitor vimseltinib or placebo. Tumours shrank in 40% on vimseltinib versus none on placebo, with better joint movement, pain and function, leading to approval in February 2025; whether responses last after stopping is unknown.
A phase 2 trial of Tazemetostat in Sarcomas, run by National Cancer Institute (NCI), completed.
The American Ewing sarcoma regimen beat the European one in a head-to-head trial, unifying practice.
Act.In.Sarc showed that injecting hafnium oxide nanoparticles into a soft-tissue sarcoma before preoperative radiotherapy roughly doubled the share of patients whose tumour, once removed, had almost no surviving cancer cells, and that result supported the product's European CE mark in 2019.
A PDGFRα antibody that had won accelerated approval on a small trial failed to improve survival in the confirmatory study and was withdrawn.
The CSF1R blocker pexidartinib shrank tenosynovial giant cell tumours in about four in ten patients against none on placebo, becoming the first approved drug for this joint tumour, with a liver-safety programme attached.
Standard anthracycline-ifosfamide before surgery beat subtype-tailored chemotherapy, and indirectly showed neoadjuvant chemotherapy helps high-risk sarcoma.
In SSGXVIII/AIO, three years of imatinib after surgery, rather than one, improved survival in high-risk GIST.
A phase 2 trial of Tarlatamab in small-cell lung cancer, run by Amgen, active and no longer recruiting.
The trial that added Hengrui's PD-L1 antibody adebrelimab to chemotherapy for small cell lung cancer, with one of the larger survival gains seen in that disease.
A phase 2 trial of Rovalpituzumab tesirine in Small-cell lung cancer, run by AbbVie, completed.
A phase 3 trial of Pimicotinib(ABSK021) in tenosynovial giant cell tumour, run by Abbisko Therapeutics Co, Ltd, active and no longer recruiting.
A phase 3 trial of Pexidartinib in tenosynovial giant cell tumour, run by Daiichi Sankyo Co., Ltd., active and no longer recruiting.
A phase 3 trial of Dabrafenib, Trametinib in thyroid cancer, run by Novartis Pharmaceuticals, active and no longer recruiting.
The UK trial confirming that low-risk thyroid cancer patients can safely avoid radioactive iodine, published in 2025.
A phase 3 trial of Cabozantinib in thyroid cancer, run by Exelixis, active and no longer recruiting.
Proved that most people with small, low-risk thyroid cancers can skip radioactive iodine after surgery without any increase in recurrence.
Adding a MEK inhibitor to boost iodine uptake before ablation did not improve complete remission rates, cooling the 'redifferentiation for everyone' idea.
Turned the thyroid cancer with the shortest survival, once measured in months, into a treatable disease for the third of patients whose tumours carry a BRAF mutation.
Turned lenvatinib into the main drug for thyroid cancers that no longer take up radioactive iodine, quadrupling the time before the disease grew.
The first drug approved for thyroid cancers that stopped responding to radioactive iodine.
Showed a third of the usual radioactive iodine dose ablates the thyroid remnant just as well, with fewer side effects and less time in isolation.
FOCUS showed that pumping high-dose melphalan through the liver's blood supply, then filtering it out before the blood returns to the body, shrank tumours in about one in three patients whose eye melanoma had spread to the liver, and it supported the treatment's US approval in 2023.
A phase 2 trial of Acalabrutinib in waldenström macroglobulinaemia, run by Acerta Pharma BV, active and no longer recruiting.
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.
PATHFINDER 2 is the US real-world study of the Galleri test that forms the core of its FDA submission.
ROCKstar showed that belumosudil, a daily pill, improved chronic graft-versus-host disease in about three in four people whose disease had not settled after at least two earlier treatments, which led to its US approval in 2021.
This open-label study showed that an antibody blocking the phosphate-wasting hormone FGF23 raised blood phosphate to normal and helped heal fractures in adults whose soft-bone disease was driven by a small tumour that could not be located or removed, which supported Crysvita for that cancer-care use.
MSB-GVHD001 showed that donor bone marrow stromal cells, given twice a week for a month, helped about seven in ten children whose acute graft-versus-host disease had not settled on steroids, which supported the first US approval of a mesenchymal stromal cell therapy in 2024.
NCI-MATCH was the largest trial to sequence tumours from thousands of patients across the United States and give each a drug matched to the mutation rather than to the organ. It proved the system could work at national scale, found real activity for a handful of matches, and showed that most single drugs given for a single mutation do little.
I-PREDICT flipped the basket idea: instead of one drug for one mutation, a tumour board built a personal combination for each patient covering as many of their tumour's changes as possible. People whose treatment matched more of their alterations lived longer without progression.
WINTHER was the first trial to pick cancer drugs using RNA, comparing what genes a tumour switched on against a biopsy of the patient's normal tissue, for patients whose DNA showed no obvious target. It missed its formal goal, but showed the approach was workable and that better-matched treatment went with better outcomes.
In healthy older people, daily low-dose aspirin did not extend disability-free life and, unexpectedly, was linked with more cancer deaths. It changed guidelines against routine aspirin in the elderly.
The definitive test of whether vitamin D or fish-oil pills prevent cancer or heart disease in healthy adults. They did not.
Vistogard Studies 1 and 2 gave an oral uridine antidote quickly after a fluorouracil or capecitabine overdose or early severe toxicity; almost everyone treated survived, while most people in an earlier group given supportive care alone had died, and the studies were the main evidence for the US approval of Vistogard.