The Top Brain Cancer News of 2025

2025 proved to be a pivotal year in neuro-oncology, marked by several clinical breakthroughs and regulatory actions that promise to advance novel therapies for some of the most challenging and aggressive malignancies. In particular, several major developments emerged for glioblastoma (GBM), one of the most lethal and treatment-resistant brain tumors.

With these breakthroughs actively reshaping the landscape and improving the prognostic outlook for patients, here are the top 5 articles capturing some of the most significant brain cancer advancements of the year.

FDA Signals Potential for Standard Approval of Paxalisib in Glioblastoma

Following a Type C meeting, the FDA indicated that overall survival (OS) data for paxalisib in GBM could support standard approval, though it was deemed unlikely to meet the requirements for accelerated approval.

The decision is supported by secondary OS data from the phase 2/3 GBM-AGILE trial, which showed a 3.8-month improvement in OS for patients with newly diagnosed GBM and unmethylated MGMT promoter status. The drug was also well-tolerated with no new safety signals. Kazia Therapeutics and the FDA have aligned on the design of a pivotal phase 3 study to further evaluate paxalisib in this population.

FDA Grants Orphan Drug Designation to Novel Glioblastoma Therapy

In September, the FDA granted orphan drug designation to BA-101, an investigational small molecule therapy for GBM. The designation provides incentives such as tax credits, fee waivers, and 7 years of market exclusivity upon approval, encouraging the development of treatments for this high-unmet-need disease.

BA-101 targets dysregulated nitric oxide signaling, an important modulator of tumor proliferation and therapy resistance in GBM. Preclinical data suggest that BA-101 can reduce cancer cell growth and enhance the efficacy of temozolomide (Temodar), the current standard of care.

Sonodynamic Therapy Shows Promise for Glioblastoma Treatment

A study published earlier this year in the Journal of Neuro-Oncology indicates that sonodynamic therapy (SDT) safely destroys GBM cells while preserving healthy brain tissue. Using a noninvasive method of combining low-intensity ultrasound with oral 5-ALA, patients experienced no adverse events after a single dose.

In a separate phase 1/2 trial for recurrent high-grade gliomas, SDT significantly extended median OS beyond the historical 6- to 8-month benchmark to 15.7 months. This therapy's ability to target invasive zones across the brain hemisphere represents a potential breakthrough, and a randomized controlled trial with SDT in newly diagnosed patients with GBM is imminent.

Behind the FDA Approval of Dordaviprone, a New Hope for Glioma

The FDA’s August 2025 accelerated approval of dordaviprone (Modeyso) for treatment of adult and pediatric patients with K27M-mutant diffuse midline glioma marked a significant therapeutic development as the first FDA-approved systemic therapy for this aggressive and rare brain tumor. The supporting trials for dordaviprone showed a 22% overall response rate and favorable tolerability, with once-weekly oral dosing.

In this featured interview, Dr Patrick Wen of Dana-Farber Cancer Institute dove into the significance of the approval and highlighted the general methodological challenges and complexities of evaluating treatment candidates. While the approval offers a new option for a subset of patients, the ongoing phase 3 ACTION study remains crucial to confirming these clinical benefits.

Novel Immunotherapy Regime Shows Promise in Recurrent Glioblastoma

A novel chemotherapy-free regimen combining nogapendekin alfa inbakicept-pmln (Anktiva), natural killer cell therapy, and tumor treating fields has shown significant promise for recurrent GBM. In a pilot study of 5 patients, the regimen achieved a 100% disease control rate and 3 objective responses, including 2 near-complete responses.

The approach uses an interleukin-15 agonist to activate immune cells and overcome tumor-induced suppression. Initial data also showed reversed lymphopenia in all participants. A phase 2 trial is currently recruiting to further evaluate the safety and efficacy of this immune-stimulating combination in GBM.