Source: Mattias von Herrath, M.D., researcher at La Jolla Institute for Allergy and Immunology
ALS THERAPY: ALS, or Lou Gehrig's Disease, is a devastating, progressive disease in which a gene mutation attacks the nerve cells of the central nervous system, leaving patients unable to control voluntary muscle function. It is a fatal, hereditary disease with no known cure, affecting between 7,500 and 10,000 new patients every year. Typically, patients live only three to five years after initial diagnosis. This new therapy is designed to slow the progression of the disease by silencing the mutated gene, protecting neurons from premature death. Researchers hope a treatment of therapeutic molecules known as antisense oligonucleotides can be delivered to the brain and spinal cord through the spinal fluid to slow ALS progression. When tested in rats in a laboratory setting, the therapy was shown to be effective. To deliver the treatment to patients, surgeons would implant a small pump into a patient and a catheter into the area surrounding the spinal cord, allowing antisense oligonucleotide drugs to be continuously pumped directly into the nervous system. Doctors say the implantation surgery is routine, similar to pain management devices implanted everyday across the country. Researchers say if the gene-silencing therapy proves effective, a similar approach could be used to treat patients suffering from other neurodegenerative disorders, including Alzheimer's, Parkinson's and Huntington's diseases. Clinical trials in humans could begin as early as the start of 2008.
Source: Don Cleveland, Ph.D., Neuroscientist at the University of California, San Diego
Diabetes:Bonnie Ward
La Jolla Institute
(619) 303-3160
contact@liai.org
ALS:
alstrials.ucsd.edu