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Disease
Medulloblastoma
Late-stage therapeutic developmentEmerging research
2
Publications
19
Clinical trials
1
Related conditions
2024
Latest publication
Latest activity
betaRecent clinical, regulatory, research and industry developments relating to this disease.
Adolescents and Young Adults With Cancer: CNS Tumors.
Research2023-12-08Journal of clinical oncology : official journal of the American Society of Clinical Oncology
Molecular subgroups of medulloblastoma: the current consensus.
Research2011-12-02Acta neuropathologica
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
Executive briefingUpdating summary…Momentum: Moderate
Key developments
- 5 clinical trials expected to report results, the earliest in Q4 2027.
Upcoming Milestones
- Q4 2027Phase III Randomized Double-blind Placebo-controlled Trial of Metformin for Cognitive Recovery and White Matter Growth in Paediatric Patients With a Brain Tumour
- Q2 2028Phase 2 Trial of a Novel Peptide Vaccine (PEP-CMV) Targeting CMV Antigen for Newly Diagnosed Pediatric High-grade Glioma and Diffuse Intrinsic Pontine Glioma and Recurrent Medulloblastoma
- Q4 2029A 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
- Q4 2030HeadStart4: Newly Diagnosed Children (<10 y/o) With Medulloblastoma and Other CNS Embryonal Tumors Clinical and Molecular Risk-Tailored Intensive and Compressed Induction Chemotherapy Followed by Consolidation With Randomization to Either Single Cycle or to Three Tandem Cycles of Marrow-Ablative Chemotherapy With Autologous Hematopoietic Progenitor Cell Rescue
- Q3 2035SJiMB21: Phase 2 Study of Molecular and Clinical Risk-Directed Therapy for Infants and Young Children With Newly Diagnosed Medulloblastoma
Major developments
Major trial resultsHigh impact
Results posted2026-06-09
Major trial resultsHigh impact
Results posted2026-06-08
Clinical MilestonesView all 11Hide
Results posted
- 2026-06-09A Study Evaluating Limited Target Volume Boost Irradiation and Reduced Dose Craniospinal Radiotherapy (18.00 Gy) and Chemotherapy in Children With Newly Diagnosed Standard Risk Medulloblastoma: A Phase III Double Randomized TrialResults posted
- 2026-06-08Efficacy of Carboplatin Administered Concomitantly With Radiation and Isotretinoin as a Pro-Apoptotic Agent in Other Than Average Risk Medulloblastoma/PNET PatientsResults posted
- 2026-01-23A Phase III Randomized Trial for the Treatment of Newly Diagnosed Supratentorial PNET and High Risk Medulloblastoma in Children < 36 Months Old With Intensive Induction Chemotherapy With Methotrexate Followed by Consolidation With Stem Cell Rescue vs. the Same Therapy Without MethotrexateResults posted
Results expected
- 2026-06-03HeadStart4: Newly Diagnosed Children (<10 y/o) With Medulloblastoma and Other CNS Embryonal Tumors Clinical and Molecular Risk-Tailored Intensive and Compressed Induction Chemotherapy Followed by Consolidation With Randomization to Either Single Cycle or to Three Tandem Cycles of Marrow-Ablative Chemotherapy With Autologous Hematopoietic Progenitor Cell RescueResults expected Q4 2030
- 2026-05-11Phase 2 Trial of a Novel Peptide Vaccine (PEP-CMV) Targeting CMV Antigen for Newly Diagnosed Pediatric High-grade Glioma and Diffuse Intrinsic Pontine Glioma and Recurrent MedulloblastomaResults expected Q2 2028
- 2026-05-05A 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 FeaturesResults expected Q4 2029
- 2026-05-01Phase III Randomized Double-blind Placebo-controlled Trial of Metformin for Cognitive Recovery and White Matter Growth in Paediatric Patients With a Brain TumourResults expected Q4 2027
- 2026-02-12SJiMB21: Phase 2 Study of Molecular and Clinical Risk-Directed Therapy for Infants and Young Children With Newly Diagnosed MedulloblastomaResults expected Q3 2035
Recently completed
+3 more in the activity timeline below
Activity timeline11
- 2026-06-09ClinicalA Study Evaluating Limited Target Volume Boost Irradiation and Reduced Dose Craniospinal Radiotherapy (18.00 Gy) and Chemotherapy in Children With Newly Diagnosed Standard Risk Medulloblastoma: A Phase III Double Randomized TrialResults posted
- 2026-06-08ClinicalEfficacy of Carboplatin Administered Concomitantly With Radiation and Isotretinoin as a Pro-Apoptotic Agent in Other Than Average Risk Medulloblastoma/PNET PatientsResults posted
- 2026-06-03ClinicalHeadStart4: Newly Diagnosed Children (<10 y/o) With Medulloblastoma and Other CNS Embryonal Tumors Clinical and Molecular Risk-Tailored Intensive and Compressed Induction Chemotherapy Followed by Consolidation With Randomization to Either Single Cycle or to Three Tandem Cycles of Marrow-Ablative Chemotherapy With Autologous Hematopoietic Progenitor Cell RescueResults expected Q4 2030
- 2026-05-11ClinicalPhase 2 Trial of a Novel Peptide Vaccine (PEP-CMV) Targeting CMV Antigen for Newly Diagnosed Pediatric High-grade Glioma and Diffuse Intrinsic Pontine Glioma and Recurrent MedulloblastomaResults expected Q2 2028
- 2026-05-05ClinicalA 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 FeaturesResults expected Q4 2029
- 2026-05-01ClinicalPhase III Randomized Double-blind Placebo-controlled Trial of Metformin for Cognitive Recovery and White Matter Growth in Paediatric Patients With a Brain TumourResults expected Q4 2027
- 2026-03-31ClinicalPhase 2 Study of Intraventricular Omburtamab-based Radioimmunotherapy for Pediatric Patients With Recurrent Medulloblastoma and EpendymomaPrimary completion
- 2026-02-12ClinicalSJiMB21: Phase 2 Study of Molecular and Clinical Risk-Directed Therapy for Infants and Young Children With Newly Diagnosed MedulloblastomaResults expected Q3 2035
- 2026-01-23ClinicalA Phase III Randomized Trial for the Treatment of Newly Diagnosed Supratentorial PNET and High Risk Medulloblastoma in Children < 36 Months Old With Intensive Induction Chemotherapy With Methotrexate Followed by Consolidation With Stem Cell Rescue vs. the Same Therapy Without MethotrexateResults posted
- 2025-09-10ClinicalPersonalized Risk-Adapted Therapy in Post-Pubertal Patients With Newly-Diagnosed Medulloblastoma (PersoMed-I)Terminated
Therapeutic landscape
Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.
Approved & established therapies
Carmustineapproved
Approval — Carmustine is indicated n adults in the following malignant neoplasms as a single ag… (2018)
Clinical trials
The current development programme across all trial phases.
Clinical programme
19
6
7
6
Late-stage studies
Recruiting
Recently completed
Regulatory timeline
Drug regulatory events matched to this condition by indication — EMA.
First approvals
2018emaApproval — Carmustine· Carmustine is indicated n adults in the following malignant neoplasms as a single agent or in combination with other antineoplastic agents and/or other therapeutic measures (radiotherapy, surgery): Brain tumours (glioblastoma, brain-stem gliomas, medulloblastoma, astrocytoma and ependymoma), brain metastases Secondary therapy in non-Hodgkin’s lymphoma and Hodgkin’s disease as conditioning treatment prior to autologous haematopoietic progenitor cell transplantation (HPCT) in malignant haematological diseases (Hodgkin’s disease / Non-hodgkin’s lymphoma). source ↗
European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Publications over time
20122024
Most influential
Major themes7
- Cerebellar Neoplasms2
- Medulloblastoma2
- Central Nervous System Neoplasms1
- Consensus1
- Ependymoma1
- Glioma1
- Neoplasms1
Leading journals2
- Acta neuropathologica1
- Journal of clinical oncology : official journal of the American Society of Clinical Oncology1
Leading researchers8
- Gajjar A2
- Cho YJ1
- Clifford SC1
- Eberhart CG1
- Ellison DW1
- Gilbertson RJ1
- Heidelberg RE1
- Kool M1
Affiliations (unnormalised)3
- Center for Cancer Research1
- Hospital for Sick Children1
- St Jude Children's Research Hospital1
Related conditions
Diseases frequently studied alongside this one. Number shows shared papers.
Reference
Authoritative identity, definition & identifiers.
Defined in MeSH
A malignant neoplasm that may be classified either as a glioma or as a primitive neuroectodermal tumor of childhood (see NEUROECTODERMAL TUMOR, PRIMITIVE). The tumor occurs most frequently in the first decade of life with the most typical location being the cerebellar vermis. Histologic features include a high degree of cellularity, frequent mitotic figures, and a tendency for the cells to organize into sheets or form rosettes. Medulloblastoma have a high propensity to spread throughout the craniospinal intradural axis. (From DeVita et al., Cancer: Principles and Practice of Oncology, 5th ed, pp2060-1)
Identifiers
References & data sources
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.