Research News for the Fourth Two Months of 2026
This is the thirty-eighth article of the project whose aim is to periodically collect (every two months) the research news on possible treatments for glioblastoma multiforme. Below I list the news items we considered most significant that emerged over the last two months. As with the previous articles in the series, each news item will be preceded by the original title with a link to the source and followed by a brief comment. The criterion by which the news is selected is always to include, in general, only news relating to research in the clinical phase, unless the research’s potential for the treatment of glioblastoma is truly remarkable.
NeOnc Technologies Reports Positive Topline Phase 2a Results for Intranasal NEO100 in Recurrent IDH1-Mutant High-Grade Glioma
One of the historical limits of brain-tumor therapy is getting the drug past the blood–brain barrier. NEO100 (the NEO100-01 study) is an ultra-purified formulation of perillyl alcohol that the patient self-administers at home through the nose, with a mask and nebulizer, four times a day: the intranasal route exploits the olfactory and trigeminal nerves to reach the brain directly, bypassing the barrier and avoiding systemic toxicity. In this phase 2a study of 24 patients with recurrent or progressive Grade III and IV IDH1-mutant glioma, the primary endpoint was met: six-month progression-free survival of 48.9% versus the 20% expected with standard of care (p=0.0047, RANO 2.0 criteria), with a median overall survival of about 26 months. The treatment proved well tolerated and has received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the FDA; the company has requested a meeting with the FDA to define a registrational path. It should be stated clearly that this concerns Grade III/IV IDH1-mutant gliomas, not the classic IDH-wildtype glioblastoma, and that the study is small and single-arm; but for a setting with no approved therapies at recurrence the results are encouraging.
UAB trial shows first-in-human immunotherapy that more than doubles progression-free survival in glioblastoma patients (INB-200)
Published in the Journal of Clinical Oncology, the phase 1 INB-200 study (NCT04165941, University of Alabama at Birmingham with IN8bio) is the first in the world to bring genetically modified gamma-delta T cells into the clinic. Gamma-delta T cells recognize the NKG2D ligands that appear on tumor cells after chemotherapy; here they were engineered to express the enzyme MGMT, which makes them resistant to temozolomide and allows them to be administered together with it during the maintenance phase of the Stupp protocol, in newly diagnosed glioblastoma. According to the reported data, 92% of evaluable patients exceeded the roughly 7-month median progression-free survival expected with standard treatment alone, with manageable toxicity in an outpatient setting — hence the summary that the therapy “more than doubles” progression-free survival. As this is a phase 1 trial the numbers are small and confirmation is needed, but the signal is enough to justify continuing: the phase 2 study INB-400 is already underway.
Anti-LAG-3 with or without anti-PD-1 in recurrent glioblastoma: a phase 1 trial
Published in Nature Medicine, the phase 1 ABTC 1501 study (NCT02658981) enrolled 46 patients with first-recurrence glioblastoma (23 per cohort) to test relatlimab, an antibody that blocks the immune checkpoint LAG-3, alone or in combination with nivolumab (anti-PD-1). LAG-3 is one of the “brakes” that keep exhausted T cells switched off: blocking it, alone or together with PD-1, can counter the immunosuppression typical of glioblastoma, against which nivolumab alone had already failed in the past. The primary endpoint of safety was met, with the maximum tolerated doses established; grade 3–4 adverse events occurred in 6 of 23 patients in the combination arm and in none with monotherapy. Neoadjuvant administration (before surgery) was associated with greater infiltration of CD8+ T cells into the tumor, and the preliminary one-year survival signals were more favorable with the combination. The study was not designed to prove efficacy, but the results motivated a randomized phase 2 trial, now underway, comparing the combination with lomustine.
BPGbio Reports Encouraging Preliminary Data from Ongoing Phase 2 Study of BPM31510 in Newly Diagnosed Glioblastoma
Preliminary phase 2 results (NCT04752813, presented at ASCO 2026) suggest that BPM31510, an intravenous mitochondrial therapy, may improve outcomes in newly diagnosed glioblastoma when added to standard radiation and temozolomide. The drug is a lipid nanodispersion of oxidized coenzyme Q10, given together with vitamin K1 to reduce effects on coagulation, and aims to target the altered metabolism of tumor cells by increasing oxidative stress. Across 39 evaluable patients, median overall survival was 19.3 months; the most interesting figure concerns the 24 patients with MGMT-unmethylated tumors — the subgroup with the worst prognosis — where median survival reached 29.3 months, roughly double the historical control. It is not yet clear why this subgroup in particular seems to benefit more; the drug proved well tolerated with no new related serious adverse events, and the results need confirmation: final data are expected by the end of the year.
Novel Radiation Strategy Redefines a New Treatment Paradigm for Glioblastoma (GammaTile)
After glioblastoma removal, several weeks are normally awaited — for the surgical wound to heal — before starting radiotherapy and chemotherapy; during that interval the residual tumor cells can regrow. GammaTile addresses precisely this problem: they are absorbable tiles containing radioactive cesium-131 seeds, placed directly into the surgical cavity at the time of the operation, so that radiation begins immediately and in a concentrated way, without interfering with healing. In the GESTALT feasibility and safety study (NCT05342883, 67 patients with newly diagnosed glioblastoma across 15 US centers, presented at ASCO 2026), only 6% of patients experienced rapid regrowth before the next phase of treatment, compared with the 50–70% historically reported. As this is not a randomized study, it cannot yet be said that GammaTile prolongs survival or should become the new standard: to answer that question the randomized phase 3 study BRIDGES has been launched. The concept — not granting residual cells weeks to reorganize — is convincing; now the randomized data are needed.
Ultra-hypofractionated versus conventional chemoradiation for newly diagnosed glioblastoma: survival and toxicity results of a multicenter randomized trial
A randomized phase 3 study from the Netherlands compared an ultra-short radiotherapy schedule (6 sessions of 6 Gy over 2 weeks) with the standard one (30 sessions of 2 Gy over 6 weeks), both with concurrent and adjuvant temozolomide, in newly diagnosed glioblastoma. The study enrolled only 135 of the planned 474 patients because of slow recruitment, but the results are nonetheless clear and worth knowing: the non-inferiority of the ultra-short schedule was not demonstrated, with a median overall survival of about 13 months versus about 21 for the standard schedule and a higher frequency of radionecrosis. The authors’ conclusion is clear: compressing radiotherapy into six high-dose sessions must not replace the standard in general newly diagnosed glioblastoma. It is important not to generalize — abbreviated schedules such as 40 Gy in 15 sessions remain an established option for elderly or frail patients who are not candidates for the full course — but it is one of those “negative” news items that are just as valuable, because they indicate precisely what to avoid.
DCVax-L-Associated Survival Extension Assessed Through Propensity Score Matching Analyses
DCVax-L is a vaccine made of autologous dendritic cells loaded with the patient’s own tumor lysate; the phase 3 results, which showed an extension of survival in both newly diagnosed and recurrent glioblastoma, had already been published in JAMA Oncology. At the British Neuro-Oncology Society (BNOS) congress, new and more rigorous statistical analyses were presented, based on propensity score matching, which according to the authors would confirm and strengthen the results, with a more than doubled 5-year survival rate in newly diagnosed glioblastoma. It should be framed for what it is: not a new trial, but a re-analysis of already existing data, presented at a congress and based on comparison with external controls — an approach that requires the usual interpretive caution relative to randomized studies. That said, for one of the longest-running and most debated vaccine paths in glioblastoma it is an update worth following.
Enrollment opens for Adaptin Bio’s Phase 1 trial of glioblastoma therapy (APTN-101)
We close with a news item that marks the passage from the laboratory to the clinic. Adaptin Bio has opened, at Duke University, enrollment in a first-in-human phase 1 study of APTN-101, a bispecific T-cell “engager” built on the BRiTE (Brain Bispecific T-cell Engager) platform. The idea addresses two problems of glioblastoma at once: using T cells not only to attack the tumor, but also to carry the therapeutic agent across the blood–brain barrier, directing it toward the cells that express EGFRvIII, a tumor-specific alteration present in a subset of glioblastomas (which, in theory, allows the tumor to be hit while sparing healthy cells). The study, open-label and with a dose-escalation design, will enroll up to 15 adults with EGFRvIII-positive grade IV malignant glioma and has as its primary objective safety and the definition of the maximum tolerated dose, not yet the demonstration of a survival benefit. In preclinical models the approach had shown promise; now it is a matter of finding out whether it works in the human brain. It is exactly the kind of trial we like to highlight.
We remind you that these updates have an informational purpose: many of the studies mentioned are in an early phase and often conducted abroad, and eligibility depends on very specific clinical criteria. The decision whether and in which study to take part must always be made with one’s own neuro-oncologist or referral center, the only one who can verify a given case’s eligibility and which trials are actually available.
That is all for this fourth two months of 2026. Heartfelt thanks to all the people who, with their support, allow us to keep the volunteer organization alive and to develop projects increasingly focused on concrete support for patients and their caregivers. If you are experiencing the glioblastoma journey directly or indirectly, we remind you that the Speranza e Coraggio project offers a specialized psychological support service, completely free of charge, designed to accompany patients and family members through the most difficult moments: asking for help is an act of strength.
To all those who are fighting against glioblastoma, and to their loved ones, we send our warm hug.
