Brain Neurotherapy Bio, Inc. (BNB) announced the treatment of the first patient in clinical trial testing a new approach to treat Parkinson’s using MRI-guided neurosurgery to inject gene therapy into damaged regions of the brain.
This CIRM‑funded trial, led by Dr. Krystof Bankiewicz, is one of 64 clinical trials supported by the California stem cell agency to date.
More on Parkinson’s disease
Parkinson’s disease, which affects one million people in the U.S., causes shaking, stiffness, and problems with walking, balance, and coordination.
At its core, the condition results from the breakdown and death of dopaminergic neurons. Those brain cells produce dopamine, a chemical messenger essential for normal movement.
Using gene therapy
Recently, the first patient received treatment at The Ohio State University Wexner Medical Center. The gene therapy is designed to increase production of GDNF. In turn, the protein GDNF protects dopaminergic neurons. By boosting GDNF, the treatment aims to raise dopamine levels in the brain, ease Parkinson’s symptoms, and potentially slow disease progression.
“We are pleased to support this multi‑institution California collaboration with Ohio State,” said Maria T. Millan, MD, President and CEO of CIRM. “This marks the culmination of years of research by the Bankiewicz team to deliver GDNF to damaged neurons. We join the Parkinson’s community in closely watching the outcome.”
Encouraging multiple approaches
CIRM Board Member and patient advocate David Higgins, Ph.D. is also excited. Advocating for Parkinson’s is a very personal Higgins said. His grandmother, and his uncle were diagnosed with the disease.
“Our best chance for developing better treatments for Parkinson’s is to test as many logical approaches as possible,” Higgins said. “CIRM encourages out-of-the-box thinking. The Parkinson’s community is a-buzz with excitement about the GDNF.”
This approach could lead to a one-time treatment for Parkinson’s, Dr. Sandra Kostyk, director of the Movement Disorders Division at Ohio State Wexner Medical Center.
“This is a one‑time treatment strategy that could have ongoing lifelong benefits,” Kostyk said. “Though we hope this treatment will slow disease progression, we don’t expect it to completely stop or cure it. We’re cautiously optimistic as this research moves forward.”
Meanwhile, other trial sites in California are actively recruiting patients. Currently those sites are at the University of California, Irvine (UCI) and the University of California, San Francisco (UCSF). Specifically, the UCI trial site is using the UCI Alpha Clinic, one of five leading medical centers in the CIRM Alpha Clinic in the state. This network specializes in delivering cell therapies and support to clinical research.
For more on clinical trials
- The Ohio State University: OSUgenetherapyresearch@osumc.edu
- University of California, San Francisco: GDNF@ucsf.edu
- University of California, Irvine: chewbc@hs.uci.edu