Research News in the Second Two-months of 2025

This is the thirty-first article of the project that aims to periodically (every two months) collect updates on research into possible treatments for glioblastoma multiforme. Below is a list of the most significant news that has emerged in the last two months. As with the previous articles in the series, each item will be preceded by the original title with a link to the source and followed by a short comment. The criterion for selecting the news is always to generally include only news related to clinical-stage research, unless the research’s potential for glioblastoma treatment is truly remarkable.

Neoadjuvant triplet immune checkpoint blockade in newly diagnosed glioblastoma
This study describes the trial of an innovative combination of immunotherapies in the treatment of glioblastoma multiforme (GBM). This therapeutic strategy involves administering three immunotherapy drugs (nivolumab, ipilimumab, and relatlimab) before surgical resection of the tumor (neoadjuvant phase), aiming to maximize immune activation against tumor cells. Preliminary results, obtained in newly diagnosed patients, indicate a strong immune response, with a significant increase in immune cells infiltrating the tumor. The therapy was well tolerated, without serious side effects. Notably, one of the treated patients showed no recurrence seventeen months after treatment, exceeding expectations compared to standard therapies. A larger clinical trial is already being prepared to further confirm these promising results and verify the effectiveness of this innovative strategy in a broader patient population.

LAM561 as add-on may delay glioblastoma disease progression
LAM561, a synthetic derivative of oleic acid developed by Laminar Pharma, shows promising results in the treatment of newly diagnosed glioblastoma multiforme (GBM). Administered in addition to standard therapy (surgery followed by chemoradiotherapy with temozolomide), LAM561 demonstrated potential in delaying disease progression in patients with MGMT promoter methylation, a genetic modification present in 30–50% of GBM cases. Preliminary data from the phase 2/3 CLINGLIO clinical trial, which involved 144 patients, indicate that in subjects with MGMT methylation and RTOG scores of 4, the median progression-free survival (PFS) was 86.4 weeks with LAM561, compared to 54.7 weeks with standard therapy alone. However, no significant benefits were observed in patients without MGMT methylation. LAM561 works by altering the composition of tumor cell membranes, reducing the activity of signaling proteins that promote tumor growth. The therapy was well tolerated, with no additional serious side effects. Final results, including overall survival data, are expected by the end of 2026.

UT Health San Antonio-led research discovers a way to slow or block recurrence of glioblastoma
A team of researchers at UT Health San Antonio has identified a new strategy to delay or block recurrence of glioblastoma multiforme (GBM) after radiotherapy. The discovery is based on the use of senolytic drugs, capable of eliminating senescent cells induced by radiotherapy, which would otherwise promote tumor regrowth. Radiotherapy, the standard treatment for GBM, can induce senescence in some tumor cells, which, although not proliferating, release growth factors that stimulate residual tumor cells to resume proliferation. Researchers discovered that these senescent cells depend on the anti-apoptotic protein cIAP2 for survival. By administering the senolytic drug birinapant after radiotherapy, they successfully eliminated the senescent cells, significantly reducing tumor recurrence in mouse models. These preclinical results suggest that the approach combining radiotherapy with targeted elimination of senescent cells could represent a new therapeutic strategy to improve survival in GBM patients. This is preclinical research but is linked to topics published in previous issues of this column and responds to readers’ requests for more information on treatments with senolytic drugs.

Promising preliminary results of phase 1 study
IQ-AI has announced encouraging results from a phase 1 clinical study of orally administered Gallium Maltolate (GaM), tested in patients with recurrent glioblastoma multiforme. Among the 23 evaluable patients treated daily with GaM, the average overall survival was about 14 months from the start of therapy, a positive result compared to current therapies. The treatment was safe and well tolerated. Although progression-free survival (PFS) remained similar to standards (about 2 months), the overall survival data suggests a potential benefit in disease control. Further studies will be needed to confirm these promising results. This news also responds to readers’ requests for more information on treatments with gallium maltolate.

VXM01 plus avelumab is safe and shows preliminary clinical activity in recurrent glioblastoma
A new immunotherapy combination has shown promising results in the treatment of recurrent glioblastoma multiforme (GBM). The oral vaccine VXM01, which stimulates a T cell response against VEGFR-2, was administered alongside the anti-PD-L1 antibody avelumab in a phase 1 study conducted on pre-treated patients. The combination was well tolerated, with adverse events mainly of grade 1 or 2. Among the 27 treated patients, the 12-month overall survival rate was 52%, with a median survival of 10.6 months. Median progression-free survival (PFS) was 3.6 months, and about 37% of patients achieved disease stabilization for at least 6 months. These preliminary results suggest that VXM01, in combination with avelumab, could offer clinical benefits in patients with recurrent GBM, particularly regarding disease stabilization. Further studies are underway to confirm and deepen these data.

TLX101 shows promising efficacy in recurrent glioblastoma
Telix Pharmaceuticals has reported encouraging preliminary results from the phase 2 IPAX-Linz study, which evaluates TLX101, a targeted radiotherapy administered intravenously, in combination with external beam radiotherapy (EBRT) in patients with recurrent glioblastoma multiforme (GBM). TLX101 is designed to target the LAT1 transporter, overexpressed in GBM tumor cells, allowing targeted delivery of radiation. The study involved 8 patients with first or second recurrence GBM, of whom 5 had MGMT unmethylated tumors, known for their poor prognosis. The treatment was well tolerated, with no serious adverse events reported. The median overall survival (OS) was 12.4 months from the start of TLX101 treatment and 32.2 months from initial diagnosis, exceeding the historically observed 9.9-month median OS with EBRT alone in recurrent GBM patients. These results support previous data from the IPAX-1 study, which reported a median OS of 13 months from the start of TLX101 and 23 months from initial diagnosis. The ongoing IPAX-2 study is evaluating TLX101 in combination with standard therapy in newly diagnosed GBM patients.

Smart T-Cells Built to Last: Ultrasound-Activated Cancer Killers Target Solid Tumors
A research team at the University of Southern California has developed a new generation of engineered T cells, called EchoBack-CAR T, designed to fight solid tumors. These T cells are activated by focused ultrasound, allowing precise control of their antitumor activity. In preclinical models, EchoBack-CAR T cells showed five times longer action than conventional CAR T cells, maintaining their effectiveness over extended periods. Activation by ultrasound allows T cell activity to be directed precisely at the tumor, reducing the risk of systemic side effects. This innovation represents a significant step toward applying immunotherapy to solid tumors, which are traditionally more resistant to this type of treatment. Further studies are ongoing to assess the effectiveness and safety of this technology in clinical settings.

Thanks to all who have helped and continue to help keep the volunteer organization alive and to develop our projects always centered on supporting patients and their caregivers. That’s all for this issue on research news. Before we close, we remind you that we have experimentally launched an Instagram profile where we will publish short informational videos about glioblastoma. Our heartfelt best wishes to all those who are fighting glioblastoma and to their loved ones!