The first 60 days: Medulloblastoma

Staging tests establish exactly what and where the cancer is. Everything else follows from the answers.

These specialties appear in the standard of care for this cancer. In most centres they meet weekly as a tumour board to agree each plan; you can ask when yours was discussed and what was decided.

• SurgeonNamed in the standard of care for: Average risk (≥3 years, M0, <1.5 cm² residual, no MYC amp).
• Medical oncologistNamed in the standard of care for: Average risk (≥3 years, M0, <1.5 cm² residual, no MYC amp), High risk (metastatic, residual, anaplastic, MYC), Infants (<3 years), Relapsed.
• Clinical oncologist (radiotherapy)Named in the standard of care for: Average risk (≥3 years, M0, <1.5 cm² residual, no MYC amp), Infants (<3 years), Relapsed.
• Transplant and cell therapy teamNamed in the standard of care for: Average risk (≥3 years, M0, <1.5 cm² residual, no MYC amp), High risk (metastatic, residual, anaplastic, MYC), Infants (<3 years).

A second opinion from a centre that treats many similar cases is normal, not rude; the standard of care tells you what to compare it against.

Each row is a setting from the standard of care, in the order it usually arises. Not all will apply to you; your stage and biomarkers decide which do. The guideline grade, where recorded, says how strong the evidence is.

1. 1.Infants (<3 years)

   Radiation-avoiding intensive chemotherapy (Head Start, HIT-SKK with intraventricular methotrexate); desmoplastic/SHH infants do well, Group 3 infants poorly.

   MethotrexateCyclophosphamideAutologous stem cell transplant (high-dose therapy)
2. 2.High risk (metastatic, residual, anaplastic, MYC)COG ACNS0332

   CSI 36 Gy ± concurrent carboplatin (ACNS0332 for Group 3), then multi-agent chemotherapy; high-dose chemotherapy with stem-cell rescue in some protocols.

   CarboplatinCisplatinCyclophosphamideAutologous stem cell transplant (high-dose therapy)
3. 3.Average risk (≥3 years, M0, <1.5 cm² residual, no MYC amp)COG ACNS0331; SIOP PNET5

   Resection, CSI 23.4 Gy with boost to 54 Gy (proton where available), then cisplatin/vincristine/cyclophosphamide or lomustine-based chemotherapy (ACNS0331); WNT tumours receive reduced CSI in trials.

   CisplatinVincristineCyclophosphamideLomustine (CCNU)Proton therapyIMRT / IGRT (modern external beam)
4. 4.Relapsed

   Re-irradiation, temozolomide-irinotecan ± bevacizumab, SMO inhibitor (vismodegib/sonidegib) for SHH in post-pubertal patients, clinical trials; cure is rare.

   TemozolomideIrinotecan (and liposomal irinotecan)BevacizumabVismodegib

Generated from this cancer's standard of care, biomarkers and open problems. Take the group that matches where you are. To tick, add your own and print, open the one-page appointment sheet.

Trials open now for this cancer in OnCo, largest phase first. Joining a trial is a decision like any other: ask what the comparison arm is, whether a placebo is used, and what happens if you leave. The cancer page searches ClinicalTrials.gov live for more.

• A Study of Treatment for Medulloblastoma Using Sodium Thiosulfate to Reduce Hearing LossPhase 3 · recruiting · NCT05382338A Phase 3 Study of Sodium Thiosulfate for Reduction of Cisplatin-Induced Ototoxicity in Children With Average-Risk Medulloblastoma and Reduced Therapy in Children With Medulloblastoma With Low-Risk Features
• ACNS1422Phase 2 · active · NCT02724579Newly diagnosed average-risk WNT-driven medulloblastoma in children aged 3 to 21: reduced craniospinal irradiation of 18 Gy with a reduced tumour-bed boost and reduced maintenance chemotherapy (cisplatin, cyclophosphamide, lomustine, vincristine)
• SJMB12Phase 2 · active · NCT01878617Newly diagnosed medulloblastoma aged 3 to 39, stratified by molecular subgroup and clinical risk: reduced craniospinal irradiation (15 Gy) for low-risk WNT tumours, vismodegib added for skeletally mature SHH tumours, and pemetrexed and gemcitabine added for intermediate- and high-risk group 3 and 4 tumours, with exercise and cognitive remediation randomisations
• Study Of Palbociclib Combined With Chemotherapy In Pediatric Patients With Recurrent/Refractory Solid TumorsPhase 2 · active · NCT03709680PHASE 1/2 STUDY TO EVALUATE PALBOCICLIB (IBRANCE®) IN COMBINATION WITH IRINOTECAN AND TEMOZOLOMIDE OR IN COMBINATION WITH TOPOTECAN AND CYCLOPHOSPHAMIDE IN PEDIATRIC PATIENTS WITH RECURRENT OR REFRACTORY SOLID TUMORS
• Childhood Cancer Survivor Study (CCSS)Observational study · active · NCT01120353Retrospective cohort with prospective follow-up of five-year survivors of childhood cancer diagnosed 1970-1999 at 31 North American centres, with sibling controls

Much of what helps in the first weeks is free if you know to ask: testing, helplines, rides and lodging, second opinions, trial travel.

• Free in oncologyFree testing, helplines, rides and lodging, second opinions and trial travel help, with who is eligible.
• Financial helpManufacturer programmes, national schemes and charities by country and product.
• Second opinionExpert centres for this cancer in your country and how referral works.

• Medulloblastoma: the full pageMedulloblastoma is the most common malignant childhood brain tumour, arising in the cerebellum. Surgery, radiation to the whole brain and spine, and chemotherapy cure about 70%, at a heavy cost to thinking and growth; treatment is now being tailored to four molecular subgroups so that the low-risk children get less.
• One-page appointment sheetYour questions, the words you may hear, what to bring, and space for the answers. Print it.
• Treatment sequencingWhich treatment tends to follow which, line by line.
• NavigatorStandard of care for your stage, what you have tried, and trials near you.