Scientists may have found a way to slow down the debilitating and deadly disease amyotrophic lateral sclerosis.

ALS, which famously struck the late baseball player Lou Gehrig and long-surviving physicist Stephen Hawking, is a condition that degrades neurological function and takes away muscle control. It also acts quickly — according to the ALS Association, half of those diagnosed die within three years. But researchers at University of California, Riverside, and Sanford-Burnham-Prebys Medical Discovery Institute in La Jolla have designed molecules that act on a gene closely linked to the progression of ALS, which they hope will slow down the disease.

Read: Who is the Oldest ALS Survivor?

According to their study in Cell Chemical Biology, the gene, EphA4 “is critical for the progression of motor neuron degeneration.” The molecules bind to the gene and delay disease progression in mice, although the researchers admit they do not fully understand how the mechanism works. Still, “the described agent represents both an invaluable pharmacological tool … in ALS and potentially other human diseases, and a significant stepping stone for the development of novel treatments.”

Physicist Stephen Hawking, who has ALS, aboard a Boeing 727 aircraft in 2007. NASA, public domain

The information about how to inhibit the the EphA4 gene could prove useful to diseases other than ALS because it has also been implicated in Alzheimer’s disease, certain cancers, spinal cord and brain injuries and blood clotting issues, UC Riverside said in a statement. The researchers are pushing forward, looking to develop additional molecules that work similarly to their original design.

Source: Pellecchia M, Wu B, DE SK, et al. Potent and Selective EphA4 Agonists for the Treatment of ALS. Cell Chemical Biology. 2017.

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