First Gene Therapy Trial for Batten Disease to be Conducted at Our Medical Center
The University of Rochester Medical Center is set to lead the way in the fight against a rare and devastating neurodegenerative disorder, with the commencement of a groundbreaking clinical trial for CLN5 Batten disease.
CLN5 Batten disease, a member of the neuronal ceroid lipofuscinoses (NCLs) family, is a rare and fatal condition that affects children, leading to vision loss, cognitive and motor impairment, seizures, and premature death. Currently, there are no approved treatments that can reverse the symptoms of this debilitating disease.
The clinical trial, named NGN-101, is possible due to the work started in 2001 by Jonathan W. Mink, M.D., Ph.D., and others, to understand the natural history of Batten diseases. Dr. Mink, the Frederick A. Horner MD Distinguished Professor in Pediatric Neurology and Chief of Child Neurology at the University of Rochester Medical Center, has contributed significantly to the research on neurodegenerative diseases, including CLN3 (a form of Batten disease) and synucleinopathies.
NGN-101 is an adeno-associated virus-based treatment that delivers a healthy CLN5 gene directly to the central nervous system. This innovative approach aims to halt the progression of CLN5 Batten disease, targeting key features such as vision, motor, cognitive, and behavioral declines.
The trial will move research forward in developing a potentially disease-modifying treatment for CLN5 disease. Due to the rarity of the disease, patients from across the U.S. are expected to travel to Rochester to participate in the study.
The University of Rochester Medical Center is home to the University of Rochester Batten Center, one of the nation's premier centers dedicated to the study and treatment of Batten disease. The Batten Center, under the leadership of Dr. Mink, has developed the Unified Batten Disease Rating Scale, a tool used by researchers globally to track and quantify the progression of different forms of Batten disease.
The NGN-101 trial will be conducted by a team of dedicated researchers, including Amy Vierhile, D.N.P., Jennifer Vermilion, M.D., Heather Adams, Ph.D., and Erika Augustine, M.D.
Animal studies suggest that NGN-101 has the potential to halt key features of disease progression in CLN5 Batten disease. The experimental treatment is being developed by Neurogene, a company that has received clearance from the Food and Drug Administration to begin clinical trials and Orphan Drug Designation by both U.S. and European regulatory agencies.
The University of Rochester Medical Center anticipates enrolling its first study participants for the NGN-101 trial in early 2022, offering hope to families affected by this devastating disease. The trial will use a single intraventricular dose delivered directly into cerebrospinal fluid in the brain, marking a significant step forward in the treatment of CLN5 Batten disease.
While the trial is a promising development, it is important to note that the current treatment options for CLN5 Batten disease are limited to managing symptoms, and standard treatments for symptoms like seizures are not consistently effective. The NGN-101 trial represents a beacon of hope for families affected by this rare and devastating disease, offering the potential for a disease-modifying treatment.
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