TGen to lead first-in-patient clinical trial studies to test novel drugs for glioblastoma

Glioblastoma pilot trial funded by Ivy Foundation

In 2012, The Ben & Catherine Ivy Foundation awarded $10 million in grants for two groundbreaking brain cancer research projects at the Translational Genomics Research Institute (TGen). One of those projects has officially received the final regulatory approval from University of California, San Francisco, which means patient enrollment for the trial can begin.

In the $5 million project, "Genomics Enabled Medicine in Glioblastoma Trial," TGen and its clinical partners will lead first-in-patient clinical trial studies that will test promising new drugs that might extend the survival of GBM patients. This multi-part study will take place in clinics across the country and TGen laboratories.

"GBM is one of the top three fastest-killing cancers out there and it affects people of all ages," said Catherine (Bracken) Ivy, founder and president of The Ben & Catherine Ivy Foundation. "It is critical that we fund research that will help patients live longer so we can study and treat brain cancer."

The project begins with a pilot study of 15 patients, using whole genome sequencing to study their tumor samples to help physicians determine what drugs might be most beneficial.

To support molecularly informed clinical decisions, TGen labs also will examine genomic data from at least 536 past cases of glioblastoma, as well as tumor samples from new cases, developing tools that will produce more insight into how glioblastoma tumors grow and survive. TGen also will conduct a series of pioneering lab tests to measure cell-by-cell responses to various drugs.

"GBM is a disease that needs answers now, and we strongly believe those answers will be found in the genome," said Dr. David Craig, TGen's Deputy Director of Bioinformatics, Director of TGen's Neurogenomics Division, and one of the projects principal investigators. "Identifying the genes that contribute to the survival of glioblastoma will provide valuable information on how to treat it, and may also lead to an improved understanding of what drives other cancers as well."

To get new treatments to patients as quickly as possible, this five-year study will include a feasibility study involving up to 30 patients, followed by Phase II clinical trials with as many as 70 patients. TGen is teaming with the Ivy Early Phase Clinical Trials Consortium that includes: University of California, San Francisco; University of California, Los Angeles; the MD Anderson Cancer Center; Memorial Sloan Kettering Cancer Center; University of Utah; and the Dana-Farber/Harvard Cancer Center.

The results of these clinical trials should not only help the patients who join them, but also provide the data needed for FDA approval and availability of new drugs that could benefit tens of thousands of brain cancer patients in the future.

"Working with physicians, the project will aim to understand treatment in the context of the tumor's molecular profile. We will have the opportunity to determine when combinations of drugs might be more effective than using a single drug, quickly identify which therapies don't work, and accelerate discovery of ones that might prove promising for future development," said Dr. John Carpten, TGen's Deputy Director of Basic Science, Director of TGen's Integrated Cancer Genomics Division, and another of the project's principal investigators.

In addition to helping patients as quickly as possible, the project should significantly expand Arizona's network of brain cancer experts.