CU Boulder researchers discover new direction for treating ALS

A University of Colorado Boulder study has discovered a new protein believed to play a role in causing ALS, giving researchers a new direction for potential treatments.

The research suggests that the protein, known as PEG10, changes cell behavior in ways that lead to ALS when it’s present at high levels in nerve tissue.

The protein is ancient and virus-like, known for its role in aiding the development of the placenta and preventing cancer progression. However, when it’s overly abundant in the wrong places, it may also cause disease.

“The fact that PEG10 is likely contributing to this disease means we may have a new target for treating ALS,” said Alexandra Whiteley, senior author of the study and assistant professor in the Department of Biochemistry, in a news release. “For a terrible disease in which there are no effective therapeutics that lengthen lifespan more than a couple of months, that could be huge.”

More than 5,000 people are diagnosed annually with ALS, also known as Amyotrophic Lateral Sclerosis and Lou Gehrig’s Disease. It is a neurological disease that attacks nerve cells in the brain and spinal cord that control movement.

Over time, the disease gradually inhibits people’s ability to speak, move, eat and breathe. Drugs exist to slow the progression of the disease, but there is no cure.

Whiteley’s research found that PEG10 is present in high levels in the spinal cord tissue of ALS patients, where it likely interferes with communication between the brain and nerve cells.

With funding from the ALS Association and the National Institutes of Health, CU Boulder researchers in Whiteley’s lab are continuing their work to find a way to inhibit the protein and find possible new treatments.

The research is still in its “early days,” Whiteley said in a statement, “But the hope is this could potentially lead to an entirely new class of potential therapeutics to get at the root cause of this disease.”

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