CytRx Corporation has signed an exclusive, worldwide, royalty-bearing license agreement with the University of Massachusetts Medical School (UMMS) to use UMMS's RNAi gene silencing technology in the development of a treatment for amyotrophic lateral sclerosis (ALS), commonly known in the U.S. as Lou Gehrig's disease.
Research using RNAi technology in ALS is being conducted by Zuoshang Xu, Associate Professor of Biochemistry and Molecular Pharmacology at UMMS. According to Dr. Xu, RNAi will be used to determine the underlying cause of ALS at the genetic level by searching for and selectively silencing the mutant disease causing gene. In this way, researchers will be able to more easily determine the most advantageous therapeutic strategy.
"While several promising treatments are being developed for ALS, the specificity and power of RNAi technology could advance these efforts significantly," said Steven A. Kriegsman, the company's chief executive officer. "ALS is a hideous disease that can strike down a world renowned athlete as easily as your next door neighbor. It develops slowly and leaves those afflicted with virtually no treatment options. We are extremely pleased to extend our collaboration with UMMS, one of the world's leading pioneers in RNAi technology, to gain a foothold in the race for a cure for ALS." Financial details of the transaction were not disclosed by the parties for competitive reasons.
ALS was first described in scientific literature in 1869, and there is still no cure. The exact cause of this progressive neurodegenerative disease that results in motor neuron degeneration of the brain and spinal cord and eventual paralysis is unknown. In 50% of patients, death normally occurs 18 months after diagnosis. Only 20% of patients survive five years and 10% live longer than ten years. Noted British physicist Stephen Hawking has been living with ALS for more than 35 years. In the United States alone, approximately 30,000 people are living with ALS and almost 15 new cases are diagnosed daily.
The agreement is part of a broad-based strategic alliance with UMMS utilizing RNAi technology, which also includes exclusive licensing agreements for the use of RNAi in the development of therapeutic products to treat obesity and type II diabetes. Discovered in 1997 by UMMS faculty member Craig Mello, in collaboration with Andrew Fire, Ph.D., at the Carnegie Institution of Washington, RNAi is becoming an increasingly important tool in biomedical research. The technology essentially uses ribonucleic acid to selectively silence harmful genes within a living cell. Beyond providing researchers with a new way to understand gene function, the platform technology may also offer a comprehensive means of advancing effective new drug development.