Personalized Medicine for ALS

By Stephanie Watson @WatsonWriter
December 01, 2017

The future of amyotrophic lateral sclerosis treatments could be unique therapies matched precisely to patients, called personalized medicine.

For a condition with as dire a prognosis as amyotrophic lateral sclerosis (ALS), the need for treatments that can stop or reverse the disease course is especially urgent. As of today, only two drugs — riluzole (Rilutek) and adaravone (Radicava) — are approved to treat ALS, and neither can dramatically improve survival.


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One promising new direction in amyotrophic lateral sclerosis treatments is personalized — or precision — medicine. Unlike standard treatments, which apply the same approach to all ALS patients, personalized medicine evaluates a person’s genetic makeup, environment, and lifestyle to deliver more targeted treatments that are more likely to work for them.

Doctors have already started using personalized medicine to treat cancer — like the PARP inhibitor olaparib (Lynparza), which treats advanced-stage ovarian cancer in women who carry the BRCA gene mutation. Now, they’re beginning to apply the same principles to ALS.

Personalized medicine could potentially lead to much more effective treatments for ALS. And, it could save a significant amount of money, as doctors would no longer need to attempt therapies that aren’t likely to work.

Written in your genes

About 10 percent of people with ALS have an inherited form of the disease. Researchers have identified mutations to nearly two-dozen genes — including SOD1, TARDBP, C9ORF72, and FUS — that are responsible for these familial ALS cases. SOD1 was the first ALS gene mutation to be discovered, in 1992. When it’s normal, this gene produces an enzyme that keeps the brain healthy. When it’s mutated, that protective enzyme is lacking.


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March 16, 2020

Reviewed By:  

Christopher Nystuen, MD, MBA