Completed project

Ranjit Bindra, MD, PhD

Yale University School of Medicine

Exploiting Mutant IDH1/2-induced DNA Repair Defects in Pediatric Glioma

CureSearch Catapult Award

High-grade glioma

Project Overview About the Researcher Research Updates

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.

In the News

Biopharma Makes Progress against a Childhood Killer: Pediatric Glioma BioSpace

Project at a glance

Program: Catapult Award
Institution: Yale University School of Medicine
Focus: High-grade glioma

Ranjit Bindra, MD, PhD

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.

Follow the project’s progress.

Final Research Update

Dr. Bindra’s Catapult Award supported the translation of a precision medicine strategy designed to treat a specific subtype of pediatric and adolescent glioma that has a known weakness in how it repairs damaged DNA.

Building on foundational discoveries from his laboratory, Dr. Bindra led the launch and execution of a multi-institutional Phase I clinical trial through PNOC to test whether combining an FDA-approved DNA repair inhibitor with standard chemotherapy could make treatment more effective while reducing long-term toxicity.

During the funding period, the trial opened at leading children’s hospitals nationwide and enrolled 20 pediatric and adolescent/young adult patients with newly diagnosed or recurrent tumors. The team successfully identified a recommended Phase II dose for the drug combination, observed encouraging early signals of disease control in several patients, and conducted extensive molecular and drug-level analyses to better understand how patients respond to therapy. Together, these advances represent a critical bench-to-bedside step toward more targeted, less toxic treatment options for this underserved group of brain tumor patients.