Glioblastoma Multiforme (GBM) is the most commonly diagnosed brain cancer in adults. It is insidiously aggressive and is the disease that so quickly took the life of Arizona Senator John McCain. Reglagene’s approach to targeting the TERT gene offers new hope for a better therapy. The TERT gene is mutated in 80% of GBM patients. Patients presenting with the mutated gene have a median survival of 10 months, compared with 20 months for patients presenting with the normal gene. The National Institute of Neurological Disorders and Stroke (NINDS) notified Reglagene of a $400,000 STTR small-business award on April 5, 2019, to enable application of its novel TERT gene-targeting technology to discover and develop a new therapy for GBM. Reglagene’s partner in this effort is Dr. Michael Berens, Professor and Director, Cancer and Cell Biology Division at The Translational Genomics Research Institute (TGen). The Principal Investigator on the award is Reglagene’s CSO Dr. Laurence Hurley.
The envisioned therapeutic agent leverages the action of DNA quadruplex structures to selectively attenuate expression of the TERT gene. Distinguishing the GBM program from Reglagene’s effort to apply this same technology to prostate cancer is that the targeted therapeutic agent must access the brain. This difference is substantial and will require a therapeutic agent with unique characteristics. Successful product development will provide a new therapy for GBM, a disease that has seen limited progress in treatment paradigms over the last several years.