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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 Paediatric low-grade glioma, drawn from the whole corpus: 4 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.
How long to continue MAPK inhibitors and whether tumours regrow on stopping; intermittent dosing and stop rules are being studied in FIREFLY-2 and the COG selumetinib trials.
Long-term effects of RAF and MEK inhibition on growth plates, skin and heart in children who may take them for years; registries and trial follow-up are collecting these data.
Preserving vision in optic pathway glioma, where imaging response and visual outcome do not always agree; visual endpoints are now built into trials.
Access to molecular testing and to the new drugs outside high-income countries.
Nothing recorded yet.
Nothing recorded yet.
Also on OnCo: Financial help · Coverage by country · HTA decisions.
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 15 changes by month →When this page itself was last checked or edited.
Relapsed/refractory paediatric low-grade glioma (conditional)
Relapsed/refractory BRAF-altered paediatric low-grade glioma (accelerated)
Tovorafenib (FIREFLY-1; FDA accelerated approval April 2024 for patients aged six months and over), or a MEK inhibitor such as selumetinib in trials.
FIREFLY-1 (Nature Medicine 2024) shows durable responses across fusions and V600E; FDA accelerated approval April 2024.
Dabrafenib plus trametinib first line (TADPOLE: higher response rate and longer progression-free survival than carboplatin-vincristine; FDA approval March 2023 for patients aged one year and over).