December 3rd 2025
Treatment with IGV-001 demonstrated a 45% increase in overall survival compared with standard of care in patients with newly diagnosed glioblastoma.
December 1st 2025
Edward Chu, MD, a member of the gastrointestinal editorial advisory board, died of glioblastoma in November.
November 25th 2025
One patient with newly diagnosed glioblastoma multiforme reached 3 years of survival following treatment with temferon in a phase 1/2a trial.
November 13th 2025
Data from a phase 1 trial demonstrate the safety and feasibility of administering chlorotoxin-based cellular therapy to those with recurrent glioblastoma.
November 7th 2025
While more work must be done to minimize toxicity, antibody-drug conjugates have demonstrated benefits for patients with glioblastoma.
Prior Encouraging Data Support Further Study of Niraparib in Glioblastoma
Findings from a proof-of-concept study show a potential survival benefit with niraparib/radiotherapy in patients with newly diagnosed glioblastoma.
Sacituzumab Govitecan Shows Preliminary Activity in Patients With Brain Mets, GBM
Neoadjuvant/adjuvant sacituzumab govitecan showed encouraging intratumoral concentrations in patients with brain metastases as well as glioblastoma.
FDA Approves Vorasidenib in IDH+ Astrocytoma/Oligodendroglioma
Phase 3 data from the INDIGO trial support the FDA approval of vorasidenib for select patients with grade 2 astrocytoma or oligodendroglioma.
Paxalisib Improves Survival Vs SOC in Newly Diagnosed Glioblastoma
Developers plan to discuss findings from the phase 2/3 GBM AGILE study with the FDA to determine a potential approval pathway for paxalisib.
FDA Approves Tovorafenib for Relapsed/Refractory Low-Grade Glioma
The FDA approval of the single-agent, oral pan-RAF inhibitor tovorafenib is based on efficacy results from the phase 2 FIREFLY-1 trial.
Novel Radiotherapy Technology Shows “Great Advantage” in Brain Cancer
ZAP-X may provide submillimeter accuracy when administering radiation to patients with brain tumors.
PET Imaging Agent Earns FDA Fast Track Designation in Recurrent Glioma
TLX101 is currently under assessment as a treatment for glioma in the IPAX-2 and IPAX-Linz studies.
AZD1390/Radiation Yields Encouraging Efficacy, Safety in Glioblastoma
First-in-patient findings support AZD1390 as a potential radiosensitizer during the management of glioblastoma.
Bivalent CAR T-Cell Therapy Shows Tumor Shrinkage in Recurrent Glioblastoma
Findings highlighted tumor reductions in patients with recurrent glioblastoma who received CART-EGFR-IL13Rα2 cells at 2 dose levels.
CAN-3110 Earns FDA Fast Track Designation in Recurrent High-Grade Glioma
Findings from a phase 1b trial support the tolerability and potential survival benefit of CAN-3110 as a treatment for patients with recurrent high-grade glioma.
Eflornithine Is Approved by the FDA for High-Risk Neuroblastoma
Eflornithine, which was approved in adult and pediatric patients with high-risk neuroblastoma, was assessed in an externally controlled trial comparing results from Study 3b and Study ANBL0032.
SONALA-001/Transcranial Ultrasound Device Gets FTD in Pediatric Brain Cancer
The preliminary safety and efficacy of SONALA-001 is being assessed as part of the SDT-201 study in diffuse intrinsic pontine glioma.
FDA Gives Priority Review to Tovorafenib in Pediatric Low-Grade Glioma
The FDA has set a Prescription Drug User Fee Act date of April 30, 2024 for tovorafenib as a treatment for pediatric patients with relapsed or progressive low-grade glioma.
FDA Updates Temozolomide Prescribing Information in Brain Cancer
Prescribing information has been updated to include temozolomide as adjuvant treatment for adult patients with newly diagnosed anaplastic astrocytoma or refractory anaplastic astrocytoma.
Vorasidenib Significantly Improves PFS Vs Placebo in IDH+ Glioma
Investigators also report an improvement in time to next intervention with vorasidenib compared with placebo among patients with IDH-mutated glioma in the phase 3 INDIGO trial.
ABM-1310 Receives FDA Orphan Drug Designation in BRAF+ Glioblastoma
Findings from a phase 1 trial highlight the potential tolerability and efficacy of ABM-1310 as a treatment for patients with BRAF-mutated solid tumors.
Olaptesed Pegol Combo Yields ‘Meaningful’ 15-Month Survival in Glioblastoma
Overall survival is expected to continue improving among patients with glioblastoma being treated with frontline olaptesed pegol plus radiotherapy and bevacizumab who remain on the phase 1/2 GLORA study.
IL4R-Targeting Toxin MDNA55 Yields Promising OS in Recurrent Glioblastoma
MDNA55 appears tolerable and effective in a cohort of patients enrolled in a phase 2b trial, showing sustained efficacy in subgroup analyses.
Lenalidomide Demonstrates Promising Activity in Pediatric Gliomas/Astrocytomas
Data from a phase 2 trial assessing lenalidomide in patients under 22 years of age also highlight an optimal dose level for this population.
ERAS-801 Receives ODD From FDA for Malignant Glioma/Glioblastoma
ERAS-801 is under investigation as part of the phase 1 THUNDERBBOLT-1 trial in a population of patients with EGFR-mutant, recurrent glioblastoma.
PRT811 Yields Early Anti-Tumor Activity in Uveal Melanoma/Advanced Glioma
Common treatment-emergent adverse effects among patients receiving PRT811 for uveal melanoma or advanced glioma include nausea and vomiting in a phase 1 study.
Tovorafenib Produces Encouraging Responses in Pediatric Glioma
Treatment with tovorafenib appears well tolerated among pediatric patients with low-grade glioma, according to findings from the phase 2 FIREFLY trial.
Vorasidenib Reduces Risk of Progression/Death in Low-Grade Glioma
In the study, 331 patients with IDH-mutated low-grade glioma were randomized to receive oral vorasidenib at 40 mg once daily or matched placebo in 28-day cycles.
MGMT Promoter Methylation Status May Predict Survival in Glioma Subtypes
Data from a cohort study suggest MGMT promoter methylation could serve as a stratification factor for patients with low-grade and anaplastic gliomas harboring IDH–wild-type or IDH-mutant and co-deleted tumors.
Therapy Reduction May Not Affect Survival in Pediatric Neuroblastoma
Patients under the age of 3 with neuroblastoma experienced no significant negative impact on survival when their disease was reclassified from high-risk to intermediate-risk and their therapy was thusly reduced.
Proton Therapy May Improve Cognitive Outcomes in Craniopharyngioma
A phase 2 trial indicates that limited surgery and post-operative proton therapy result in a high rate of tumor control and less severe complications in young patients with craniopharyngioma.
Low-Dose Lenalidomide Demonstrates Activity in Adolescents With Glioma
Treatment with 20 mg/m2 of lenalidomide in patients under 22 years old who have pilocytic astrocytomas and optic pathway gliomas appears to be safe and effective.
Early Data Support GD2-Targeting CAR T in Pediatric High-Risk Neuroblastoma
Investigators of a phase 1/2 trial report that CAR T cells targeting disialoganglioside GD2 may result in long-lasting antitumor activity in young patients with high-risk neuroblastoma.
FDA Grants Orphan Drug Designation to INB-400/410 in Glioblastoma
Investigators will assess INB-400 as a treatment for patients with newly diagnosed glioblastoma in a phase 2 trial expected to initiate in the second half of 2023.
FDA Grants Fast Track Designation to Novel EGFR Inhibitor in Glioblastoma
ERAS-801, which now has FDA fast track designation for glioblastoma with EGFR alterations, is currently under investigation as a monotherapy in the phase 1 THUNDERBBOLT-1 trial.