Targeting Specific Inhibitors Shows Effectiveness in Treatment of ALS

SAINT PAUL, Minn., Jan. 7 /PRNewswire/ -- Scientists at the Hughes
Institute in St. Paul, Minn., report the effectiveness of a new agent with a
specific inhibitor of a cellular protein known as Janus Kinase 3 (JAK3) in the
treatment of Amyotrophic lateral sclerosis (ALS). The results of this research
are published this month in Biochemical and Biophysical Research
Communications.
ALS, more commonly known as Lou Gehrig's Disease, is a progressive, fatal,
neurodegenerative disorder involving the motor neurons of the cortex, brain
stem, and spinal cord. The onset of disease is usually in the fourth or fifth
decade of life and affected individuals succumb within 2 to 5 years of disease
onset. ALS occurs in both sporadic and familial forms, with approximately
10 percent of all ALS patients in familial cases (FALS).
A drug developed by Hughes Institute scientists, WHI-P131, effectively
inhibits the activity of JAK3, which is a member of the Janus family protein
tyrosine kinases. FALS mice treated with WHI-P131 experienced a significant
increase in survival, suggesting that specific inhibitors of JAK3 may be
useful in the treatment of human ALS. The results uniquely establish JAK3 as a
novel molecular target for the treatment of FALS. "We are very excited about
the findings from this research and the potential it has to help people
suffering from ALS," stated Vuong Trieu, Ph.D., director, Cardiovascular
Biology, Hughes Institute and lead author on the study.
Reference: Trieu VN, Liu R, Liu X-P, Uckun FM. A specific inhibitor of
janus kinase-3 increases survival in transgenic mouse model of amyotrophic
lateral sclerosis. Biochemical and Biophysical Research Communications,
267(1): 22-25, 1999.
The Hughes Institute ( http://www.hughesinstitute.org ), located in
Roseville, Minnesota, is a non-profit research organization dedicated to
combating cancer, AIDS, and diseases of the immune system.

SOURCE Hughes Institute
Web Site: http://www.hughesinstitute.org

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