Henry Ford Health System First in World to Test Treatments for Brain Cancer

Henry Ford Cancer Institute is the first on the planet to active two treatments for the deadliest form of brain cancer. // Photo courtesy of Henry Ford Health System

Detroit-based Henry Ford Cancer Institute has announced it is the first site in the world to activate two treatments for glioblastoma, the deadliest form of brain cancer, as part of a patient-centered adaptive platform trial called GBM AGILE.

Led by Global Coalition for Adaptive Research, GBM AGILE tests multiple therapies for patients with newly diagnosed and recurrent GBM. Henry Ford was the first in the world to enroll a patient when enrollment began in 2019.

“We are excited for this major step forward in the GBM AGILE trial, and especially for the hope it provides those battling glioblastoma brain cancer,” says Dr. Tom Mikkelsen, principal investigator for GBM AGILE at Henry Ford Cancer Institute and medical director of the Precision Medicine Program and Clinical Trials Office at Henry Ford Health System.

“Through global collaboration, GBM AGILE is making it possible for some of the world’s foremost experts in glioblastoma research and treatment to collaborate and advance the pace at which scientific and clinical breakthroughs can be achieved.”

The two new interventions – VAL-083 from Kintara Therapeutics and paxalisib from Kazia Therapeutics Ltd. – will open at more than 35 trial sites across the U.S., with additional sites in Canada, Europe, and China to follow.

VAL-083 is a small molecule that has been studied in more than 40 Phase I and Phase II clinical trials in multiple indications sponsored by the National Cancer Institute. It has been granted Orphan Drug Designation (drugs developed for diseases so rare it’s not profitable to make them without government assistance) for glioblastoma by the Food and Drug Administration and for glioma by the European Medicines Agency. The FDA also granted Fast Track Designation for VAL-083 in recurrent glioblastoma.

Paxalisib is a small molecule inhibitor of the PI3K/AKT/mTOR pathway, which appears to be disordered in more than 85 percent of cases of glioblastoma, making the pathway a high-potential target for new glioblastoma therapies. Paxalisib is a potent inhibitor of the PI3K pathway and has been shown to have an anti-tumor effect in animal models of glioblastoma. It was granted Orphan Drug Designation for glioblastoma by the FDA in February 2018 and Fast Track Designation for glioblastoma by the FDA in August 2020.

The trial will allow multiple therapies or combinations of therapies from different pharmaceutical partners to be evaluated simultaneously.

The Global Coalition for Adaptive Research is a nonprofit organization uniting physicians, clinical researchers, advocacy and philanthropic organizations, biopharma, health authorities, and other key stakeholders in health care.