Latest News

• What is MGMT?        
  One of the most frequently asked questions we get is: “What is MGMT?”
   
  MGMT is one of the most important biomarkers in glioblastoma, but it can be confusing to understand. We created this short animated video to explain what MGMT is, why it matters, and how it can affect treatment decisions.
   
  This is our first time using AI to help create an educational video. The script was carefully reviewed and approved by our team, including Dr. Steven Brem.
   
  We’d really like your feedback! What do you think of this format? Should we make more videos like this? Was it too technical, not technical enough, or just right? Most importantly, were you able to understand it?
   
  And please give us ideas for topics you’d like us to explain in future videos!
   
  
  
• New oral nanomedicine boosts effectiveness of cancer immunotherapies        

   Can a Gut-Derived Compound Make Immunotherapy Work Better?

   
  One of the biggest frustrations with cancer immunotherapy is that, while treatments such as checkpoint inhibitors can produce dramatic responses in some patients, they do very little for many others. Researchers at the University of Michigan may have found an interesting way to improve those odds.
   
  They developed an oral nanomedicine based on 3,4-dihydroxybenzoic acid (DHB), a natural compound produced by gut bacteria when dietary fiber is broken down. DHB appears to help T cells maintain a more durable, “memory-like” state instead of becoming exhausted and losing their ability to attack cancer.
   
  Because DHB itself is poorly absorbed and quickly eliminated, the researchers converted it into a prodrug and packaged it in a nanoemulsion designed to improve delivery. In mouse models of melanoma, colorectal cancer and breast cancer, combining the oral treatment with checkpoint inhibitors eradicated tumors and generated immune memory that protected against recurrence. The treatment also improved the activity of CAR T-cell therapy.
   
  For the brain tumor community, the concept is particularly interesting. Glioblastoma has historically responded poorly to checkpoint inhibitors, in part because its immune environment suppresses T-cell activity. Anything capable of keeping T cells functional longer could potentially help immunotherapies work better.
   
  However, there is an important caveat: this research has so far been demonstrated in mice, not patients, and glioblastoma was not one of the tumor models reported in this study. We have seen many promising immunotherapy approaches cure tumors in mice only to disappoint in human trials.
   
  Still, I think this is an intriguing avenue of research. Instead of inventing an entirely new immune therapy, researchers may be able to make existing treatments work better by changing the metabolic environment of the immune cells themselves. If this approach translates to humans, combinations with checkpoint inhibitors, vaccines, CAR T cells or other immune therapies could eventually be worth exploring in brain tumors.
   
  As always, the next important step is human clinical testing.
  
  
• NEO100-01 Meets Primary End Point, Supporting Further Development in IDH1-Mutant Glioma        

   We recently reported on NEO100, an unusual treatment that delivers highly purified perillyl alcohol directly through the nose, allowing patients to self-administer the drug at home. Earlier results were intriguing, particularly because some of the longest survivors had IDH1-mutant tumors.

   
  These new Phase 2a results strengthen that story. In 24 patients with recurrent or progressive IDH1-mutant grade 3 or 4 glioma, the 6-month progression-free survival rate was 48.9%—more than double the study's prespecified 20% benchmark—and median overall survival was an impressive 26.1 months. The treatment was also reported to be generally well tolerated, with no major toxicities.
   
  There are important limitations. This was a small, single-arm study, so the results need to be confirmed in a larger trial with an appropriate control group. The objective response rate was only 8.3%, reminding us that prolonged disease control does not necessarily show up as dramatic tumor shrinkage on MRI.
   
  Still, a median survival of more than two years in patients with recurrent high-grade IDH1-mutant glioma deserves attention. Perhaps just as important, NEO100 appears relatively easy for patients to use compared with many experimental brain tumor treatments. The company now plans to meet with the FDA to discuss a possible registrational pathway.
   
  We will be watching closely. If these results hold up in a larger study, NEO100 could become an important new option for this molecularly defined group of brain tumor patients.