Latest News

• NeOnc Reports Positive Phase II Results for Intranasal Glioma Therapy        

  The investigational treatment NEO100, a highly purified form of perillyl alcohol, showed encouraging preliminary results in a Phase IIa trial for patients with recurrent or progressive IDH1-mutant grade 3 or 4 gliomas. The NEO100 treatment is self-administered by patients through the nose four times a day, using a nasal mask and nebulizer, with the intranasal delivery intended to help overcome the blood-brain barrier. In the single-arm study, 24 patients received treatment in 28-day cycles; researchers compared the results with historical results for patients receiving treatment for recurrent high-grade glioma.
  
  The study met its primary endpoint, with 49% of patients progression-free at six months (compared with an expected 20% based on the study's historical benchmark). Median overall survival was reported as 26 months, with 87% of patients alive at six months, 61% at one year, and 54% at two years. Five patients were still receiving treatment when the preliminary results were reported, including one who had remained progression-free for 19 months, and the company reported several radiographic responses, including one patient whose enhancing tumor largely resolved after prolonged treatment. NEO100 was reportedly well tolerated, with the company describing no significant toxicity even with prolonged treatment, although the full safety and efficacy data have not yet been published in a peer-reviewed journal.
  
  It is important to note that this was a small, non-randomized trial, and our understanding of the prognosis of recurrent IDH1-mutant tumors continues to evolve (for example, recent research suggests that acquired RAS-MAPK mutations may be associated with shorter survival). Despite these caveats, the results are encouraging for a disease with no approved targeted therapies and very few salvage options in the recurrent setting. The company plans to meet with the FDA to discuss the next steps and a potential registrational trial. 

  
  
• Neurocognitive functioning in long-term survivors of glioblastoma, IDH-wildtype and astrocytoma, IDH-mutant, CNS WHO grade 4: A report from EORTC 1419 (ETERNITY)        

  The ETERNITY study looked at 185 people who had survived at least 5 years after a diagnosis of glioblastoma, IDH-wildtype, or grade 4 IDH-mutant astrocytoma, with patients surviving an average of about nine years. The researchers found that 78% had some measurable impairment in at least one area of thinking or memory. The most common difficulties involved processing speed, mental flexibility, and finding words, while recognition memory was less often affected. Importantly, however, cognitive function was mostly stable over time; the only consistent decline seen across the group was a small worsening in the ability to recall verbal information after a delay. Tumor location mattered; tumors involving the left side of the brain were associated with poorer verbal memory and word fluency, while temporal lobe involvement was particularly associated with memory difficulties. Patients who had experienced multiple tumor recurrences also tended to have worse cognitive flexibility. For patients and families, the findings are encouraging in that surviving many years does not necessarily mean that cognitive abilities will continue to decline, although some lasting difficulties may remain after the tumor and its treatment have affected the brain. The study did not evaluate potential treatments for the cognitive deficits observed, but the authors emphasized that regular neurocognitive monitoring and greater attention to preserving cognitive function should be an important focus of long-term care.

  
  
• This Deadly Brain Cancer Hijacks Brain Activity to Fuel Its Growth, Study Reveals        

  A new preclinical study from Stanford has added to the growing understanding of how gliomas leverage normal brain activity for growth. Researchers found that NLGN3, a protein made by normal neurons, interacts with the CSPG4 protein found on glioma cells, activating a mechanosensing pathway involving PIEZO1 that ultimately promotes tumor growth. This discovery broadens potential treatment targets beyond NLGN3 itself, possibly to CSPG4; however, the major challenge is that this pathway also appears to help maintain normal oligodendrocyte precursor cells, so blocking it could potentially interfere with healthy brain function. Nevertheless, this work provides important new insights into how gliomas exploit neuronal activity and could help researchers find ways to disrupt this process without harming normal neuron-glial signaling the brain needs.