COLUMBUS, Ohio – For the first time ever, a team of neurologists and neurosurgeons at The Ohio State University Wexner Medical Center and The Ohio State College of Medicine has performed a novel gene therapy brain infusion to treat a patient with Parkinson's disease.
This multicenter, Phase 1b clinical safety study is sponsored by Brain Neurotherapy Bio, Inc. and funded by the California Institute for Regenerative Medicine to test GDNF gene therapy in patients with early to moderate stages of Parkinson's disease. The one-time treatment involves infusion of a gene therapy solution into deep structures of the brain that are affected by the disease.
Parkinson's disease is a neurodegenerative movement disorder that affects one million people in the United States. Degeneration of neural pathways deep in the brain causes symptoms such as tremor, slow movement and behavioral abnormalities, said Ohio State neurosurgeon Dr. James "Brad" Elder who performed the gene therapy surgery on Aug. 25.
"The overall goal of this gene therapy treatment strategy is to slow the neurologic deterioration associated with Parkinson's disease by enhancing levels of a naturally occurring growth factor called GDNF. Targeting gene therapy delivery to the putamen, a deep brain structure affected by Parkinson's disease, will hopefully improve overall quality of life, " Elder said.
The patient, a 55-year-old Ohio man first diagnosed with Parkinson's disease in 2008, takes medicine to help control his progressively worsening disease. He said the gene therapy surgery gives him hope that his disease won't get any worse, and that he may even feel better without medications. But he added that it could take up to six months before he notices any improvements.
There has long been evidence in animal and cell culture models of Parkinson's disease suggesting that glia cell derived neurotrophic factor (GDNF) has promise as a therapy for the disease, said Dr. Sandra Kostyk, director of the Movement Disorders Division at Ohio State Wexner Medical Center. Patients with Parkinson's disease and related disorders are diagnosed and treated in the Movement Disorders clinics and neurosurgery programs at Ohio State.
One of the biggest clinical hurdles has been getting the molecule to the regions in the brain that would benefit these patients the most. GDNF is a relatively large molecule that can't be administered as a pill, nor intravenously, since it can't penetrate the blood brain barrier.
This new targeted gene delivery approach overcomes many of the obstacles that have slowed GDNF clinical trial research and is expected to facilitate the production of a continuous supply of GDNF to a critical region of the brain affected by Parkinson's disease.
"This is a onetime treatment strategy that could have ongoing lifelong benefits. Though it's hoped that this treatment will slow disease progression, we don't expect this strategy to completely stop or cure all aspects of the disease. We're cautiously optimistic as this research effort moves forward," Kostyk said.
Brain Neurotherapy Bio is a biotechnology startup company founded in 2018 by Dr. Krystof Bankiewicz to develop gene therapies for neurological disorders. Bankiewicz is also a member of Ohio State Wexner Medical Center's Neurological Institute.
"I've been investigating therapeutic gene therapy approaches for Parkinson's disease for nearly 30 years, and this marks a significant milestone that may lead to major therapeutic opportunities for those suffering with this devastating condition," said Bankiewicz, who is CEO and chairman of the board of Brain Neurotherapy Bio.
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