New WVU Drugs gene remedy trial to discover Parkinson’s illness therapy choice

First affected person handled in medical examine designed to create new mind circuits, enhance signs

Posted on 12/7/2022

MORGANTOWN, W.Va. – The West Virginia College Rockefeller Neuroscience Institute as we speak introduced the initiation of a medical trial evaluating the protection and efficacy of a brand new revolutionary gene remedy within the therapy of Parkinson’s illness. Whereas surgical implants utilized in deep mind stimulation can deal with Parkinson’s illness, gene remedy may be of explicit profit with out the necessity for implants within the mind. 

Ali Rezai, M.D., at left, and his surgical team treated the first patient in the gene therapy trial at WVU Medicine J.W. Ruby Memorial Hospital in Morgantown, West Virginia.
Ali Rezai, M.D., at left, and his surgical staff handled the primary affected person within the gene remedy trial at WVU Drugs J.W. Ruby Memorial Hospital in Morgantown, West Virginia.

“This new examine is the subsequent step from a earlier sham-controlled examine that confirmed promise in Parkinson’s illness,” Ali Rezai, M.D., govt chair of the WVU Rockefeller Neuroscience Institute, mentioned. “On this examine, AAV-GAD gene remedy is delivered to the subthalamic nucleus, a key space with dysfunction in a mind with Parkinson’s illness. Over time, the gene remedy is meant to assist Parkinson’s illness by making new mind connections and bettering signs.”

The randomized, double-blind, sham-controlled medical trial is sponsored by MeiraGTx, a gene remedy innovation chief.    

“AAV-GAD is an experimental gene remedy to guage security and therapy routine utilizing materials manufactured with our proprietary manufacturing course of that would help future commercialization,” Alexandria Forbes, Ph.D., president and chief govt officer of MeiraGTx, mentioned. “We’re excited to be working with the WVU Rockefeller Neuroscience Institute to maneuver ahead this revolutionary therapy for Parkinson’s illness, and we look ahead to reporting on the progress of the trial.” 

The primary participant is a grandparent of 14 from West Virginia who was pressured to retire due to the development and severity of Parkinson’s illness. Drugs had turn into much less efficient with worsening unintended effects. They hope their participation on this examine not solely helps them discover aid, but in addition results in advances in therapy for others with Parkinson’s. 

“I hope this gene remedy will assist my Parkinson’s and likewise assist different individuals,” the participant mentioned. 

For extra info on the examine, seek advice from identifier NCT05603312.

Concerning the Rockefeller Neuroscience Institute

The WVU Rockefeller Neuroscience Institute improves lives by pioneering advances in mind well being and therapeutics. With the most recent applied sciences, revolutionary analysis, an ecosystem of companions, and a multidisciplinary built-in method to affected person care, the RNI is making tangible progress in its purpose to fight public well being challenges starting from dependancy to Alzheimer’s. For extra details about the WVU Rockefeller Neuroscience Institute, go to 

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