Research News in the Last Two-Months of 2025
This is the thirty-fourth article of a project whose aim is to periodically collect (every two months) the latest research developments on possible treatments for glioblastoma multiforme. Below is a list of the news items we considered most significant over the past two months. As with previous articles in this series, each item is preceded by the original title with a link to the source and followed by a brief commentary. The selection criterion is, as always, to generally include only news related to research in the clinical phase, unless the potential impact of the research for the treatment of glioblastoma is particularly significant.
Servier presents longer-term INDIGO trial data showing durable effects of vorasidenib
Servier has presented new long-term follow-up data from the Phase 3 INDIGO study, confirming the sustained efficacy of vorasidenib in patients with grade 2 IDH-mutant glioma who have undergone surgery. The updated analysis shows a clinical benefit that is maintained over time, with a significant prolongation of time to disease progression compared with placebo and a delay in the initiation of more invasive treatments such as radiotherapy and chemotherapy. A particularly interesting finding is the reduction in seizure frequency observed in patients treated with vorasidenib, an important aspect for quality of life. The drug also demonstrated a favorable safety profile, with no significant negative effects on cognitive function. Overall, these results strengthen the role of IDH inhibitors as a targeted therapeutic approach in the early stages of IDH-mutant gliomas and confirm the potential of vorasidenib to modify the natural history of the disease.
Imvax announces positive top-line data from Phase 2b clinical trial of IGV-001 in newly diagnosed GBM
Imvax has released top-line results from a Phase 2b clinical trial of IGV-001, a personalized immunotherapy under investigation for patients with newly diagnosed glioblastoma (ndGBM). The data show that patients treated with IGV-001 lived, on average, 6.3 months longer in overall survival compared with the placebo group (20.3 vs 14.0 months), a clinically meaningful improvement over current standards and particularly relevant for a tumor known for its aggressiveness and limited therapeutic advances over the past two decades. The study was conducted as a randomized, double-blind, placebo-controlled trial in 99 patients following surgical resection, and although the primary endpoint of progression-free survival (PFS) was not met, the observed overall survival benefit and favorable safety profile represent an encouraging signal. These results, considered “potentially significant” by experts, could open new horizons in glioblastoma treatment strategies and will be discussed with the FDA for possible regulatory advancement of IGV-001.
Glioblastoma immunotherapy trial: a new breakthrough
A new glioblastoma immunotherapy trial described on KevinMD outlines an experimental approach that combines Natural Killer (NK) cell therapy with the use of the Optune Gio device, which generates electric fields to interfere with tumor cell division. In the first patient treated in this Phase 2 study, conducted at the Hoag Family Cancer Institute in California, the combination produced results that convinced researchers to expand the trial to evaluate its effectiveness in a larger group of patients. The innovative aspect of this study lies in the integration of cellular immunotherapy with a physical tumor-modulating device, combining an enhanced immune response with a technology already known in glioblastoma treatment. Researchers hope that this approach may not only provide additional benefit to patients but also guide the development of a possible new standard of care for GBM, a disease that has seen few significant therapeutic breakthroughs over decades. These early signals, although preliminary, represent an important step in exploring innovative immunological strategies in a context where immune response has traditionally been difficult to achieve, and they confirm how combination therapies may open new perspectives in the clinical management of glioblastoma.
Adaptive radiation therapy for glioblastoma: clinical efficacy and recurrence patterns
A Japanese study published in Radiation Oncology analyzed the clinical efficacy and recurrence patterns associated with adaptive radiation therapy (ART) in the treatment of glioblastoma. This approach involves adjusting the radiotherapy plan during treatment based on changes in tumor volume and the surgical cavity, with the aim of improving treatment precision while reducing exposure of healthy tissue. A retrospective evaluation of 59 patients showed encouraging results: relatively high overall survival rates at 1 and 2 years (93.9% and 54.6%) and a median overall survival (OS) of 26.6 months, with a predominantly central recurrence pattern and no increase in marginal recurrences. Median progression-free survival (PFS) was 10.5 months, and the therapy was associated with low toxicity and rare cases of clinically significant radiation necrosis. These findings suggest that ART may represent a promising option for optimizing radiotherapy in glioblastoma, allowing treatment to be adapted to tumor dynamics over the course of irradiation. Further studies will be needed to better define protocols and fully understand the clinical impact of this strategy.
Glioblastoma clinical trial focused ultrasound blood-brain barrier opening is safe, provides possible survival benefit
A recent multicenter clinical trial explored the use of focused ultrasound (MB-FUS) to temporarily open the blood–brain barrier prior to administration of standard chemotherapy (temozolomide) in patients with newly diagnosed glioblastoma. This Phase 1/2 study, conducted in the United States and Canada, demonstrated that the procedure is safe and feasible, with no serious treatment-related adverse events, and successfully allowed visualization of blood–brain barrier opening in all treatment sessions. The preliminary data are particularly encouraging: compared with a well-matched control group receiving standard therapy alone, patients treated with focused ultrasound showed an approximately 40% increase in overall survival and improved progression-free survival, suggesting that this technique may enhance drug delivery directly into tumor tissue. The researchers emphasize that these results could pave the way for new randomized, large-scale studies to confirm the potential clinical benefit of this strategy, with the goal of integrating controlled blood–brain barrier opening into the standard therapeutic pathway for glioblastoma.
A heartfelt thank you goes to all those who, through their support, allow us to keep this volunteer organization alive and to develop projects increasingly focused on providing concrete support to patients and their caregivers. The “Speranza e Coraggio” project is now an active resource used by those who have chosen not to face this journey alone: a psychological support service designed to accompany patients and families through the most difficult moments. If you are experiencing the glioblastoma journey directly or indirectly, we invite you to make use of this completely free service as well: asking for help is an act of strength. This concludes this issue dedicated to research updates. To all those fighting glioblastoma, and to their loved ones, we send our warmest embrace.
