July 27, 2006

Promising Therapy for ALS Delivers
Antisense Drug Directly to Nervous System
Delivering agents across the blood-brain barrier likely
to be effective treatment for other neurodegenerative diseases

By Debra Kain

Researchers from the UCSD School of Medicine, the Center for Neurologic Study and Isis Pharmaceutical Corporation have designed and tested a molecular therapy in animals that they hope will be a major development in the fight to treat amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease.

The study conducted in the laboratory of Don Cleveland, Ph.D., UCSD Professor of Medicine, Neurosciences and Cellular and Molecular Medicine and member of the Ludwig Institute for Cancer Research, shows that therapeutic molecules known as antisense oligonucleotides can be delivered to the brain and spinal cord through the cerebrospinal fluid (CSF) at doses shown to slow the progression of ALS in rats. The study will be published July 27 in advance of publication in the August issue of Journal of Clinical Investigation.

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Link: http://ucsdnews.ucsd.edu/newsrel/health/als06.asp