Gene Therapy Is Halting Cancer. Can It Work Against Brain Tumors?

Grant of up to $11 million will fund a clinical trial at UCSF that uses a smarter new CAR-T guided by precision technology.

By Suzanne Leigh | | April 18, 2024

Engineered T-cells designed to attack glioblastoma. Image by Payal Watchmaker. Share on Facebook Share on Twitter Share on Reddit Email this article Print this article

Engineered T-cells designed to attack glioblastoma. Image by Payal Watchmaker.

A type of gene therapy called CAR-T that has extended survival for thousands of patients with leukemia and other blood cancers is being adapted at UC San Francisco to treat people with glioblastoma, the most common and deadly adult brain tumor. 

This new more powerful version of CAR-T employs a novel technology developed at UCSF called synthetic notch (synNotch) that both protects healthy tissue from damage and enables the treatment to work more effectively.  

UCSF opened enrollment this week for a clinical trial that is using the technology for the first time in people. A second trial, also at UCSF, is slated for 2025.

Approximately 12,000 Americans are diagnosed each year with glioblastoma. Patients survive on average for just 15 months after their diagnosis, and new treatments are urgently needed.

“This project is a prime example of bench-to-bed translation within UCSF, representing the strengths in basic and clinical science, said Hideho Okada, MD, PhD, a physician-scientist and director of the UCSF Brain Tumor Immunotherapy Center. “We have a truly home-grown project here.”

Okada has received up to $11 million for the first trial from the California Institute for Regenerative Medicine (CIRM), which funds stem cell and gene therapy research for incurable diseases and disorders through all stages of clinical trial development.

Initial funding for the second trial is provided by the National Cancer Institute Specialized Programs of Research Excellence (NCI SPORE). 

“We hope that the treatment will prolong lives for patients with glioblastoma,” said Okada, who is a professor of neurosurgery at UCSF and a member of the Weill Institute for Neurosciences. “However, the primary goal of the current phase 1 study is to ensure safety and characterize any toxicities.”