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UMMS and Lundbeck to explore potential targeted therapy for Huntington's disease
Date:1/28/2011

te at UCLA, an expert on HD animal models. The study is expected to be completed in 24 months.

"During my years helping patients who are living with HD, our understanding of the disease has increased dramatically and opened doors for new approaches to treating the condition," said Anne Young, MD, PhD, Chief of Neurology at

Massachusetts General Hospital. "In my opinion, this approach hits the disease right at its core. If this method can reduce the huntingtin protein throughout the brain, it carries the potential to change the course of the disease."

"The Hereditary Disease Foundation recognized the potential of RNA interference to cure Huntington's disease when it was just being discovered," says Nancy Wexler, Ph.D., President of the Hereditary Disease Foundation and Higgins Professor of Neuropsychology, Columbia University. "We organized the first Workshop in the world on this topic: 'RNA Modalities in Huntington's Disease Therapy,' in 2002. The Workshop was led by Phillip Sharp, who won the Nobel Prize in 1993. This was the same year that our research team discovered the HD gene, a breakthrough that made gene silencing possible. Neil Aronin was a critical Workshop participant. The Hereditary Disease Foundation funded the very first research projects proving that RNA inference cures HD in mice. Silencing the abnormal protein gets at the heart of the problem. It is a unique and revolutionary approach to finding treatments."

This collaboration is part of Lundbeck's Huntington's disease research initiative to identify and ultimately commercialize therapies that may slow or halt the progression of the disease. This research is driven by collaborations with academic institutions and companies with promising compounds in development.


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Contact: Alison Duffy
alison.duffy@umassmed.edu
508-856-2000
University of Massachusetts Medical School
Source:Eurekalert

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