Treating the Untreatable
Glioblastoma Multiforme (GBM)
About GBM
GBM is the most common and most aggressive form of primary brain cancer. It typically results in death within 15 months of diagnosis. It has a 1 Year Survival Rate of 37.2% and a 5-Year Survival Rate of 5.1%. There are over 15,000 US GBM deaths annually and over 250,000 annual deaths globally. The current standard of care for GBM costs over $130,000 for six months of treatment making it one of the most expensive cancers to treat. Annual treatment costs globally exceed $6B. There have been no advances in treatment for over fifteen years, and the current standards of care (Temozolomide/TMZ and radiation) have limited efficacy and are highly toxic.
Current Obstacles
The systemic impediments to developing innovative GBM therapies are indisputable. Big Pharma has continuously failed to develop new GBM therapies, and there have been no therapeutic advances since 2005. The 2019 failure of a major pharmaceutical company’s highly touted immunotherapy drug is the most recent set-back. The lack of any progress has chilled conventional investment interest in novel approaches, and GBM has been referred to as “investor graveyard” in the venture capital community. Foundation support is limited and large scale federal funding has been directed elsewhere.
Our Answer
TTX’s lead compound, 2P-Im, is an anti-inflammatory agent and inhibitor of oxidative stress that targets the GBM tumor microenvironment. 2P-Im penetrates the blood-brain barrier and effectively kills GBM tumor cells. It is non-toxic to Astrocytes and non-tumor cells. 2P-Im inhibits inflammasome activity and blocks essential mitochondrial Stress Response Proteins (SRPs) that fuel GBM tumors. It targets tumor-induced immune suppression and helps overcome resistance. Its ability to synergize with the current GBM standard of care provides an opportunity for “fast track” FDA approval. 2P-Im is administered orally and has strong bioavailability.