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Everything in development, the open problems and what is being done about them, the roadmaps, and what changed on this record.
What is in development for Diffuse midline glioma, H3 K27-altered (including DIPG), drawn from the whole corpus: 11 items. Drugs are grouped by the most advanced trial phase they have reached anywhere; approved treatments sit under standard of care. Technologies are the methods being tested for this cancer, trials are the studies recorded here, and ideas are proposals not yet in a trial.
Most tumours still progress; dordaviprone helps a minority and the phase 3 ACTION trial will show whether earlier use extends survival.
Drug delivery across the blood-brain barrier into infiltrating tumour: convection-enhanced delivery, focused ultrasound and intraventricular cell therapy are being tested.
Response assessment: swelling and pseudo-progression confound MRI in the brainstem; cerebrospinal-fluid tumour DNA is being validated as a marker.
Trial speed: a disease measured in months needs platform trials and regulatory paths built for rarity, which the RACE for Children Act and paediatric consortia are meant to provide.
Nothing recorded yet.
Also on OnCo: Find a trial · Expert centres.
Dated changes read from the records linked to this cancer: approvals, regulatory steps, reported trials, guideline versions and milestones. Newest first; no date is inferred.
On EdgeAll 10 changes by month →When this page itself was last checked or edited.
Dordaviprone (ONC201), FDA accelerated approval August 2025 for patients aged one year and older with progressive disease; re-irradiation is an alternative or addition.
FDA accelerated approval on 6 August 2025 for progressive H3 K27M-mutant diffuse midline glioma, the first systemic therapy for the disease.
Durable objective responses in recurrent H3 K27M DMG reported in JCO; ACTION phase 3 open.
Actionable alteration in 31.
Majzner and colleagues (Nature) report clinical and radiographic improvement with intravenous then intracerebroventricular dosing.