>>LOS ANGELES, July 15 /PRNewswire-FirstCall/ -- In a groundbreaking discovery which impacts the potential for developing a therapeutic treatment for ALS (amyotrophic lateral sclerosis), CytRx Corporation (Nasdaq: CYTR - News) announced that its principal collaborator, Zuoshang Xu, M.D., Ph.D., of the University of Massachusetts Medical School (UMMS) has used RNAi technology to successfully "knock down" or "silence" the mutant SOD1 gene that causes familial ALS, commonly referred to as Lou Gehrig's Disease, in a mouse model. CytRx has an exclusive license for this technology from UMMS.
"Our results demonstrate the high potential for applying RNAi to treat previously untreatable diseases, including ALS. RNAi will be a highly effective strategy because it attacks the root of the disease," said Dr. Xu.
RNAi is an innovative scientific process using RNA "interference" to shut down or silence disease-causing genes within a living cell. For ALS, Dr. Xu's research team has designed a strategy to selectively silence the mutant SOD1 gene, which causes a familial form of the disease and is toxic to the body.
In their experiments, Dr. Xu's research team used a mouse model with the mutant SOD1 gene to approximate a human afflicted with ALS. By administering RNAi into the tail vein of the mouse, Dr. Xu's team demonstrated that the disease gene can be silenced.
The results from Dr. Xu's work are detailed in Aging Cell in an article entitled, "Selective Silencing by RNAi of a dominant allele that causes amyotrophic lateral sclerosis."
Dr. Louis J. Ignarro, Nobel Laureate and CytRx's Chief Scientific Spokesman commented, "What Dr. Xu has achieved in his lab has never before been accomplished -- never in the history of ALS has anyone succeeded in finding a way to attack the cause and arrest the development of the disease instead of just treating the symptoms. I have had the privilege of working with Dr. Xu who is a unique and brilliant scientist, and I am convinced that his discovery has paved the way for an effective treatment for ALS in the not too distant future."
CytRx Corporation has established itself as a leader in developing an RNAi-based treatment for ALS. In addition to CytRx's involvement with Dr. Xu, CytRx is also advised by Robert H. Brown, Jr., M.D., D.Phil., the co-discoverer of the mutant SOD1 gene as a cause of the familial form of ALS. Dr. Brown is the Founder and Director the Cecil B. Day Laboratory for Neuromuscular Research at Massachusetts General Hospital, Harvard University's teaching hospital.
ALS, which is commonly known as Lou Gehrig's disease, is a neurogenerative disease that causes motor neuron degeneration, skeletal muscle atrophy and paralysis. The disease is progressive and invariably fatal. According to the ALS Association, in the United States alone, approximately 30,000 people are living with ALS and almost 6,000 new cases are diagnosed each year. According to the International Alliance of ALS/MND Associations, more than 120,000 cases of ALS are diagnosed worldwide each year. These U.S. and worldwide statistics include patients suffering from various forms of ALS, including the familial form.<<
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Cheers, Tuck |
