Latest News

• A Phase 1 trial of iron metabolism-targeting oral gallium maltolate in recurrent and refractory glioblastoma        

  We’re excited to see these Phase 1 study results now published in Neuro-Oncology Advances.
  
  The drug appeared safe and generally well tolerated, and the median overall survival was about 16 months, which compares favorably to historical outcomes in recurrent glioblastoma. While this is a small, non-randomized trial of only 22 analyzed patients, thes results are encouraging for a Phase 1 study in this setting.

  Most patients still followed the expected course of progression over time, but five of the 22 included patients are still alive. Four of those are beyond 18 months, and two of them are at 31 and 39 months since starting treatment with gallium maltolate.

  We helped fund an expanded access program for oral gallium maltolate, and we’re encouraged to see it is now expected to open to about 20 additional patients, giving more families access while the research continues.

  
  
• Rethinking Proton Pump Inhibitors in Glioblastoma Care        

  This signal concerning proton pump inhibitors (PPIs) first emerged from analyses of real-world registry data from the xCures database, which suggested a potential link between PPI use and shorter survival in glioblastoma (GBM) patients. It is a good example of how patient registries and real-world evidence can help identify clinically relevant signals early, often years before they are evaluated or confirmed in larger randomized clinical trial datasets. Now, the association has been independently validated in a large analysis of nearly 3,000 patients from five randomized GBM clinical trials.

  The recent study found that patients taking certain PPIs, including omeprazole and pantoprazole, had shorter progression-free and overall survival, even after adjusting for key factors such as age, MGMT status, extent of surgery, and steroid use. Other stomach-protecting medications, such as famotidine (Pepcid), did not show the same association, suggesting the effect may not simply be related to acid suppression.

  This does not prove that PPIs cause worse outcomes, but the consistency of the signal across datasets adds to the concern. Since many GBM patients are placed on PPIs automatically when steroids are prescribed, it is reasonable to periodically reassess whether a PPI is truly needed or whether an alternative agent could be used instead.

  As always, patients should not stop prescribed medications without discussing it with their medical team.

  
  
• AI Predicts Brain Tumor Molecular Subtypes in Twelve Minutes        

  Researchers in Germany have developed a deep-learning AI system called “Hetairos” that can predict the molecular classification of brain and spinal cord tumors in minutes using standard histology slides. The system was trained on over 11,000 digitized tissue sections from 9,606 patients across 11 centers on four continents, with ground-truth diagnoses based on DNA methylation profiling, which is currently the gold standard for CNS tumor classification. It can distinguish 102 molecular tumor subtypes, covering nearly the full WHO classification spectrum for CNS tumors.

  In a head-to-head comparison with five expert neuropathologists across 210 challenging cases, Hetairos achieved significantly higher diagnostic accuracy (68% vs ~30% for humans), and 84% when considering top-three predictions. In prospective testing, it produced molecular subtype predictions in about 12 minutes, compared to roughly 12 days for standard methylation-based diagnostics.

  The AI also includes a confidence scoring system, with high-certainty predictions in 50-70% of cases reaching ~87-88% accuracy, and it can narrow broad differential diagnoses into a small number of likely subtypes. Importantly, it highlights the specific tissue regions driving its decisions, improving interpretability and clinical trust.

  While this system shows great potential, additional validation in independent cohorts and further regulatory evaluation will be required before it can be approved for routine diagnostic clinical use.

  
  
• TBI Linked to Higher Brain Cancer Mortality Risk        

  A large 37-year analysis of over 20,000 patients from the Traumatic Brain Injury Model Systems National Database found that a history of traumatic brain injury (TBI) is associated with a 1.75-fold higher risk of death from brain cancer compared to the general population. The risk was especially pronounced in certain subgroups, including patients with gunshot-related TBIs (over 14-fold higher risk) and those with “mild but complicated” injuries. Researchers believe chronic post-injury inflammation may contribute, and while these findings do not prove causation, they suggest TBI may have long-term oncologic as well as neurologic consequences that deserve further study.

  
  
• MAGMAS Inhibition Enhances Temozolomide Efficacy in Chemotherapy-Resistant Glioblastoma Models        

  A new preclinical study found that blocking a protein called MAGMAS, which helps tumor cells manage energy inside their mitochondria, may make glioblastoma cells more sensitive to temozolomide (TMZ). In laboratory and animal models, inhibiting MAGMAS weakened tumor cell survival and appeared to improve the effect of TMZ, radiation, and tumor treating fields, including in treatment-resistant cells.

  This is early-stage research, but it adds to a growing body of evidence suggesting that targeting how tumor cells produce and manage energy may help overcome resistance to standard GBM treatments.