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ALS: A New Therapy May Be In Sight

ALS: A New Therapy May Be In Sight
January 29
16:26 2019

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative condition that affects a person’s motor neurons.

According to the National Institute of Neurological Disorders and Stroke (NINDS), people with ALS experience gradual paralysis, which often results in death from respiratory failure within 3–5 years. Approximately 10 percent of people who have the condition, however, go on to live for 10 years.

The NINDS also quote the Centers for Disease Control and Prevention’s (CDC) 2016 estimate that 14,000–15,000 people in the United States have the condition. ALS currently has no known cure.

The U.S. Food and Drug Administration (FDA) has only approved two drugs that slow down the disease, albeit modestly: riluzole and edaravone. Clinical trials have shown that riluzole extends survival by a few months, while edaravone improves the daily functioning of people with ALS.

Generally, however, individuals living with ALS mainly benefit from supportive or palliative care.

New research may help change these limited treatment options, as scientists have uncovered a gene which could serve as a new drug target.

Joseph Klim, a postdoctoral fellow in the Harvard Department of Stem Cell and Regenerative Biology in Cambridge, MA, is the first author of the new paper, which appears in the journal Nature Neuroscience.

‘Experiments provide great hope for patients’

Previous research has found that the protein TDP-43 aggregates in the neurons of people with ALS. Instead of remaining in the nucleus of these cells — as it would in a healthy neuron — in ALS, the protein leaves the nucleus and accumulates in the cell’s cytoplasm.

This discovery led researchers to believe that the neurons’ “trash-disposal” system was genetically faulty in a way that affected TDP-43, but they did not know which genes were responsible.

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