"Researchers have shown a drug engineered to combat the gene causing spinocerebellar ataxia type 2 (SCA2) might also be used to treat ALS. Two studies were published in the journal Nature with funding from the National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health (NIH).'
Our results provide hope we may one day be able to treat these devastating disorders,' said Stefan M. Pulst, M.D., Medical Director at the University of Utah. In 1996, Dr. Pulst and others discovered mutations in the ataxin 2 gene cause spinocerebellar ataxia type 2, causing problems with balance, coordination, walking, and eye movement.
In the first study, Dr. Pulst's team found they could reduce problems associated with SCA2 by injecting mouse brains with a drug programmed to silence the ataxin 2 gene. They then demonstrated injections of the same type of drug prevented neurological problems associated with ALS and prolonged life.
'The ataxin 2 gene may unlock treatments for ALS and other neurological disorders,' said Aaron Gitler, Ph.D., Associate Professor at Stanford University and senior author of the second study. In 2010, Dr. Gitler and colleagues discovered a link between ataxin 2 mutations and ALS.
The type of drug used acts like an incomplete row of teeth on a zipper; these drugs are short sequences of DNA designed to bind to a portion of a gene’s instructions carried by a molecule. This stops cells from manufacturing proteins, a process known as gene silencing. Researchers are conducting early phase clinical trials on the safety and effectiveness of gene silencing drugs to treat several neurological disorders including ALS.
Drs. Pulst and Gitler agreed that more research needs to be done before these types of drugs can be tested in patients. Both labs are currently taking next steps by conducting further preclinical experiments." Read this full news release as published by the National Institutes of Health
There are now as many as 100 genetic permutations known to affect ALS/MND and related diseases. Mutations in genes are involved in both familial and sporadic ALS/MND. They play a role in the functioning of neurons, or are involved in regulating the production of various proteins. Please know research in genetics is just beginning to expand.