Antisense drug shows promise in amyotrophic lateral sclerosis

Reported by Susan Aldridge, PhD, medical journalist

Experiments in rats suggest it is possible to slow down amyotrophic lateral sclerosis using an antisense approach to therapy.
Amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig's disease) is a progressive disease of the nervous system which causes paralysis and death within a few years of diagnosis. It affects around 30,000 Americans and there is no effective treatment. Now a team at the University of California, San Diego, report on results of a trial in animals involving antisense oligonucleotides. These are molecules which can turn off a gene for a mutant protein which otherwise kills neurons in ALS.

In this new study, the researchers delivered the antisense molecules to the brain and spinal cords of rats through the cerebrospinal fluid and found the course of ALS to be slowed down. They hope this means that ALS could be managed as a chronic disease in humans. Their plan is to insert a small pump into the patient with ALS using a procedure already approved for the management of pain. Then a small catheter would be implanted into the area around the spinal cord so that the antisense drug could be pumped through. They also believe that this kind of antisense approach could work for other neurodegenerative diseases like Alzheimer's, Parkinson's and Huntington's diseases.

Source
Journal of Clinical Investigation early-advance 27th July 2006 (print, August)

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