Dr. Ranjit Bindra, Catapult Award Project: Pediatric Glioma

Dr. Ranjit Bindra, MD, PhD

Yale University School of Medicine

Focus: High-grade glioma

Project Title: Exploiting mutant IDH1/2-induced DNA repair defects in pediatric glioma

Ranjit Bindra is a physician-scientist and biotech entrepreneur at the Yale School of Medicine. Clinically, he treats adult and pediatric primary CNS tumors. He also runs Phase I and II clinical trials testing experimental therapeutics at Yale, which are largely based on discoveries from his own laboratory. His group recently led a team of four major laboratories at Yale, which reported the stunning discovery that IDH1/2-mutant tumors harbor a profound DNA repair defect that renders them exquisitely sensitive to PARP inhibitors. This work was published in Science Translational Medicine, and has received international attention with major clinical implications.

As a biotech entrepreneur, Dr. Bindra has started three life sciences ventures over the last 10 years: Helix Therapeutics, Cybrexa Therapeutics, and Athena Therapeutics. Of note, Cybrexa Therapeutics is a series B-funded company which is developing new classes of tumor-targeted DNA repair inhibitors. Dr. Bindra received his undergraduate degree in Molecular Biophysics and Biochemistry from Yale University, and both his MD and PhD from the Yale School of Medicine. He completed his medical internship, radiation oncology residency, and post-doctoral research studies at the Memorial Sloan-Kettering Cancer Center.

Pediatric glioma is an aggressive type of brain cancer that has a very low 5-year survival rate of less than 25%. Dr. Bindra is testing a novel drug in pediatric patients with a specific subtype of glioma that makes it sensitive to chemotherapy by inhibiting the PARP gene that is involved in repairing DNA mutations. PARP inhibitors make the cancer more sensitive to standard therapies, lowering the dosage needed and associated long-term toxicity when used in combination with chemotherapy. Dr. Bindra’s cutting-edge study has the potential to improve long-term survival rates and reduce toxicity in pediatric glioma patients and lead to a potential shift in treatment standards.

Project Update: As of May 2021, Dr. Bindra has treated his first cohort of three patients. There were no dose-limiting toxicities or significant findings. As such, dose escalation was approved for the next cohort of three patients. The first patient reached their two-year point on this trial in February of 2021.

Dr. Bindra is partnering with the Pacific Pediatric Neuro-Oncology Consortium (PNOC) to administer the clinical trial at 18 pediatric cancer centers in the U.S. enrolling 45 patients. The study is proposed to take 3 years to complete. Dr. Bindra will also collaborate with Teen Cancer America, an advocacy group dedicated to AYA patient survivorship, to share the study’s results.

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