Although numerous agents have been tested for their efficacy against
amyotrophic lateral sclerosis (ALS), only one to date—riluzole—has
been shown to slow this progressive neurodegenerative disorder. But while
modestly effective, riluzole cannot ultimately stem the relentless loss of
motor neurons that occurs in patients with ALS.
Nevertheless, clinical trials are yielding lessons that are helping
researchers design more effective strategies against ALS. Such data, as well
as novel insights into the processes that give rise to the disease, are pointing
to new treatments involving methods such as gene therapy and RNA silencing,
as discussed at the 2004 Annual Meeting of the Society for Neuroscience.
Friedrich MJ. Experimental Therapies Hold Promise for Treating Amyotrophic Lateral Sclerosis. JAMA. 2005;293(9):1048-1049. doi:10.1001/jama.293.9.1048