RXi Pharmaceuticals, medical school to collaborate for ALS cure

WORCESTER, Mass. Three philanthropic organizations and a drug company will work together to fight a debilitating disease that leaves patients paralyzed for life.

The Angel Fund, the ALS Therapy Alliance and Project ALS announced they would provide funding for a research collaboration between RXi Pharmaceuticals Corp. and the University of Massachusetts Medical School to find treatments for amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease.

Lou Gehrig’s disease is a progressive, neurodegenerative disorder that causes motor neurons in the central nervous system to die, diminishing the brain’s ability to send signals to the muscles, leading to paralysis and death from respiratory failure. The cause of ALS remains unclear, though University of Massachusetts neurology professor Robert Brown and a team of researchers discovered a gene linked to the disease in 1993, a protein antioxidant called superoxide dismutase, or SOD1.

Under the collaboration, RXi will provide its RNA interference technology and materials to Brown and his research team, which will test gene-silencing treatments on mice that have been given the SOD1 gene.

“We have been searching for an RNAi treatment for ALS for many years, and while this approach is very promising, the limiting factor critical to an effective therapeutic has been delivery,” Brown said in a statement. “I am impressed with RXi’s RNAi platform and believe that RXi’s sd-rxRNAs are the unique solution that could be the key to treatment neurological disorders such as ALS.”