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Gene Therapy Clinical Trial For Rare Neurodegenerative Disorder Commences


Pediatric Clinical Trial

The clinical team at Nationwide Children’s Hospital has already enrolled one pediatric patient for the study.

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April 29, 2016 | by Sarah Massey, M.Sc.

Nationwide Children’s Hospital has just started a first-in-human clinical trial testing the effectiveness of gene therapy at treating the rare neurodegenerative disorder, Batten disease CLN6. The study – which is led by Drs. Brian Kaspar, Jerry Mendell and Emily De Los Reyes – received FDA approval in March.

The clinical team at Nationwide Children’s Hospital has already enrolled one pediatric patient for the study. The trial investigators are actively recruiting more participants for study, with the first patients to receive the gene therapy before June.

“The Center for Gene Therapy at Nationwide Children's Hospital has worked hard to bring to clinical trial an approach through replacing the missing gene for a disease that has no treatment,” said Dr. Jerry R. Mendell, of Nationwide Children’s Hospital. “The most direct way to correct a genetic disease is to restore what is missing and that is precisely what we have done in the work with the first patient with this devastating disease enrolled in the trial.”

The gene therapy will utilize the non-pathogenic AAV9 virus to introduce a functional copy of the CLN6 gene – the faulty gene in patients with Batten disease – into the brain. Previous research has shown that introducing a replacement copy of the CLN6 gene into mutated cells, can help restore the malfunctioning gene’s functionality, thereby allowing cells to remove built-up waste products from the brain.

The study is funded by The Charlotte and Gwenyth Gray Foundation to Cure Batten Disease. Since its launch 10 months ago, the foundation has raised approximately $3.5 million or its $10 million goal.

Nearly $2 million has already been donated to the medical researchers conducting the gene therapy trial. The Gray Foundation is still hoping to raise another $1 million through crowdfunding, in order to cover the expenses associated with seeing the clinical trial through to completion.

“We are working side-by-side with the Gray Foundation to make this approach available to other children with Batten Disease CLN6,” said Mendell. “While this is the first step in making treatment a reality for this condition, we want potential partners in this campaign to know that we will do everything we can to make a difference for this disease, including refining these initial efforts if any symptom of the disease remains unresolved.”

Keywords: Rare Disease, Pediatric Clinical Trial, Gene Therapy


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