Glioblastoma and Brain Tumours: The Latest Therapeutic Advances from the 2025 IBTA World Summit

We told you about our participation in the Sixth Biennial World Summit of Brain Tumour Patient Advocates organised by the International Brain Tumour Alliance (IBTA) in Rome last November. The final report of the event has now been published — a 58-page document capturing everything discussed over those four intense days. It is the right moment to revisit some of the most important topics addressed, those that directly concern anyone facing glioblastoma or a high-grade brain tumour on a daily basis.

When 108 representatives from patient organisations in 34 countries gather in the same place for three and a half days, a significant portion of the time is devoted to science. Not science for researchers, but science communicated in a way that can be useful to those who truly live with the disease. This is the unique value of the IBTA Summit: world-renowned clinicians explaining the latest advances to patient advocates, who then bring them back to their own communities. Below we report what emerges from the Summit report on the therapeutic fronts.

Professor Manfred Westphal from the University of Hamburg outlined how the surgical approach to brain tumours is evolving. The goal remains the same — to remove as much of the tumour as possible without compromising the patient’s neurological function — but the tools available are becoming increasingly refined. Today in the operating theatre, MRI tractography and transcranial magnetic stimulation are used to map functional pathways before surgery, while during the procedure surgeons can use fluorescent molecules such as 5-ALA to better distinguish tumour tissue from healthy tissue, alongside ultrasound, intraoperative MRI, and Raman spectroscopy, a technique that uses light to biochemically characterise tissue in real time. Nanopore sequencing is also under development, promising molecular identification of the tumour during the operation itself. For meningiomas, endoscopy and advanced imaging techniques have significantly improved rates of complete resection.

Dr Silvia Chiesa from the Fondazione Policlinico Universitario A. Gemelli in Rome — a centre of excellence in the field, right here in Italy — presented the state of the art in radiotherapy for brain tumours. The standard treatment for adult glioblastoma remains 60 Gy in 30 fractions (reduced to 40 Gy in 15 fractions for more fragile patients). Recent advances, however, are moving in several directions. Adaptive radiotherapy adjusts the treatment plan in real time based on changes in tumour anatomy. Proton therapy spares healthy tissue, including the hippocampus, thereby preserving cognitive function. Hypofractionation (higher doses over fewer sessions) reduces the number of hospital visits required. The most advanced and still experimental frontier is FLASH radiotherapy: ultra-high doses delivered in milliseconds, with pauses between pulses. The aim is to strike the tumour with equal efficacy while reducing toxicity to healthy tissue. It is still under investigation, but it is one of the most promising developments in recent years.

Professor Riccardo Soffietti — an internationally renowned neurologist and president of CancerSucks APS in Turin — provided an updated overview of systemic treatments for primary gliomas. Some concrete developments: Vorasidenib has recently been approved by the EMA (the European Medicines Agency) for the treatment of grade 2 oligodendrogliomas and astrocytomas harbouring IDH mutations. It is an oral inhibitor of IDH1 and IDH2 mutations, and represents the first targeted treatment approved specifically for low-grade gliomas in Europe. Dordaviprone has shown efficacy in recurrent tumours with the H3K27-M mutation (such as certain diffuse midline gliomas) and is now under investigation for newly diagnosed tumours with this molecular characteristic. Tovorafenib, a type II BRAF inhibitor, is under study for tumours with BRAF mutations and is showing promising results. Interest is also growing in liquid biopsy: analysing blood or cerebrospinal fluid for tumour molecular markers (such as IDH or H3K27-M mutations) to monitor the disease without the need for tissue sampling. Currently this is technically feasible for some mutations, while remaining challenging for others. Soffietti’s central message: the molecular classification of a tumour is not a technical detail — it is the foundation on which any treatment strategy must be built. Every tumour has its own genetic signature.

Professor Gaetano Finocchiaro from IRCCS Ospedale San Raffaele in Milan explained why immunotherapy for brain tumours is so difficult to develop — and why the field is not giving up. Brain tumours evade the immune system through a process called “immunoediting”: they develop cellular variants that resist immune attack. The strategies under investigation seek to overcome this problem in four ways: immune checkpoint inhibitors (which “release the brake” on T cells so they can attack the tumour), adoptive T cell therapies (the patient’s own cells modified in the laboratory to better recognise the tumour), cancer vaccines (which use tumour antigens to activate the immune system), and oncolytic virotherapies (engineered viruses that selectively infect and destroy tumour cells). None of these strategies has yet been approved for gliomas, but combinations of multiple approaches represent the most promising direction.

A crosscutting theme, explained by Dr Mark Kieran from the United States: even when a drug works in the laboratory, reaching the tumour through the blood-brain barrier (BBB) is an enormous challenge. The BBB is a protective mechanism of the brain that regulates what can enter — and often excludes precisely the drugs that would be most needed. Solutions under investigation include direct injection into the cerebrospinal fluid (Ommaya reservoir), focused ultrasound to temporarily open the barrier at the site of the tumour, convection-enhanced delivery (catheters inserted directly into the tumour to administer the drug under pressure), lipid nanoparticles (small enough to cross the BBB and release the drug within the tumour tissue), and intranasal administration, which exploits the neural pathways of the nasal cavity to reach the brain.

Reading the IBTA Summit report is a worthwhile and, in some respects, encouraging exercise. Not because the problems have been solved — glioblastoma remains an extremely difficult disease — but because research is advancing on many fronts simultaneously, with Italian researchers at the forefront of several of them.