The mind remains acute, alive with intellect and curiosity. Meanwhile, the body deteriorates, atrophies, but still the mind and soul speak. This is what it is like to live with ALS -- commonly known as Lou Gehrig's disease.
Sixty-three years after the death on June 2, 1941, of baseball legend Lou Gehrig of the progressively degenerative, invariably fatal disease, researchers at the University of Massachusetts Medical School UMMS and CytRx Corporation are investigating RNAi (RNA interference) gene silencing. RNA can be described as the on/off switch and RNAi may be seen as a way to turn off killer genes to stop physical degeneration.
"Tiny snippets of RNA with two strands, instead of the usual one strand, can be used to 'shut off' specific genes. We hope that in gene silencing CytRx can find treatments-and even cures-for such fatal diseases as ALS," says Dr. Jack Barber, Scientific Director of CytRx Corporation.
CytRx Corporation is one of the first companies in the world to successfully eliminate the familial version of ALS in mice.
According to the ALS Association, in the U.S. alone, approximately 30,000 people are living with ALS and almost 6,000 new cases are diagnosed each year. The International Alliance of ALS/MND Associations estimates that more than 120,000 cases of ALS are diagnosed worldwide annually.
ALS acquired Gehrig's name from sports fans shocked to learn of Gehrig's illness and later death. He had been the Yankees first baseman during the late 20's through 1939. An American baseball legend, Gehrig holds the record for most career grand slams (23) and won a Triple Crown in 1934.
cytrx
Pursuant to a product development and commercialization agreement between Exelixis and GlaxoSmithKline (GSK), GSK has the option, after completion of Phase 2a clinical trials, to elect to develop a certain number of the cancer compounds in Exelixis' product pipeline (other than the company's cancer compound XL119), which may include XL999, thus potentially triggering milestone payments and royalties from GSK and co-promotion rights by Exelixis.
Exelixis, Inc. is a leading genomics-based drug discovery company dedicated to the discovery and development of novel therapeutics. The company is leveraging its fully integrated gene-to-drug platform to fuel the growth of its proprietary drug pipeline. Exelixis' development pipeline includes:
XL119 (becatecarin), for which a Phase 3 clinical trial has been initiated in patients with bile duct tumors; XL784, which has completed a Phase 1 clinical trial; XL647, which is currently in a Phase 1 clinical trial; XL999 for which an IND has been filed; XL844, XL820 and XL880, anticancer compounds that are potential IND candidates; and multiple compounds in preclinical development. Exelixis has established broad corporate alliances with major pharmaceutical and biotechnology companies, including GSK and Bristol-Myers Squibb Company. The company has also established agricultural research collaborations with Bayer CropScience, Dow AgroSciences and Renessen LLC. Other partners include Merck & Co., Inc., Schering-Plough Research Institute, Inc., Cytokinetics, Inc., Elan Pharmaceuticals, Inc. and Scios Inc.
exelixis.