Brain Cancer

The first pediatric cancer progress report published by AACR identified strengths and weaknesses of contemporary pediatric cancer research.

Silevertinib produced responses in NSCLC harboring non-classical EGFR mutations and will also be investigated in newly diagnosed glioblastoma.

IGV-001 produced an OS benefit vs placebo in newly diagnosed glioblastoma.

Study findings indicate potential unmet needs for early vorasidenib treatment with minimal toxicity for patients with slow-growing IDH-mutant gliomas.

Retrospective data showed vorasidenib displayed strong disease stability in addition to being well tolerated in grade 3 or 4 IDH-mutant glioma.,

B7-H3–directed CAR T cell therapy was well-tolerated and demonstrated an acceptable safety profile in recurrent glioblastoma.

INDIGO TGR analyses showed vorasidenib markedly slowed tumor growth and improved PFS and TTNI vs placebo in IDH1/2-mutant grade 2 glioma.

Sequential intracerebroventricular and intraventricular administration of CAR T-cell therapy was better tolerated in pediatric central nervous system tumors.

Vorasidenib is being integrated in the real-world setting for the treatment of patients with IDH-mutated glioma.

18F-Fluciclovine PET/MRI detected disease progression and ruled out disease in non-progressors among patients with solid tumor brain metastases.

Phase 2 study finds 177Lu-Dotatate safe in advanced intracranial meningioma, with a 6-month PFS rate surpassing historical benchmarks.

Erdafitinib had a safety profile that was deemed tolerable in patients with recurrent or progressive IDH wild-type glioma harboring F3T3 gene fusions.

Eflornithine plus lomustine produced superior OS and PFS outcomes vs lomustine in WHO grade 3 IDH-mutated astrocytoma.

Temozolomide plus radiotherapy significantly improved OS vs radiotherapy alone in IDH-mutant low-grade gliomas without codeletions of 1q and 19q.

Long-term ReNeu results showed deeper, more durable responses to mirdametinib in NF1-PN with extended treatment in both adults and children.

HER2-directed CAR T-cell therapy was safe in patients with brain/leptomeningeal metastases from HER2-positive breast cancer.

B7-H3–directed CAR T-cell therapy given intraventricularly was well tolerated and showed early efficacy signals in patients with recurrent glioblastoma.

Mirdametinib had clinical activity and was deemed well tolerated in MEK inhibitor–naive pediatric patients with recurrent/progressive low-grade glioma.

The RP2D of regorafenib when given with temozolomide and radiotherapy in patients with MGMT-methylated, IDH wild-type glioblastoma was 120 mg.

Vorasidenib plus temozolomide was safe in glioma harboring IDH1/2 mutations.

The FDA granted orphan drug designation to tinostamustine for malignant glioma.

Long-term follow-up results show vorasidenib prolongs PFS and reduces tumor progression vs placebo in IDH-mutant grade 2 glioma.

The FDA granted orphan drug designation to an exosome-based therapy for glioblastoma multiforme.

Selumetinib has received approval in the EU for the management of plexiform neurofibromas in adult patients with neurofibromatosis type 1.

FDA grants fast track status to MT-125, a first-in-class NMII inhibitor, aiming to bring a novel treatment option to patients with aggressive glioblastoma.