Stanford team wins $13M grant to find genetic causes of ALS

A team at Stanford University has received a $13 million grant to uncover previously unknown genetic causes of amyotrophic lateral sclerosis (ALS), with the goal of identifying targets for future treatments.

The grant, awarded by the California Institute for Regenerative Medicine (CIRM) to a team led by Stanford genetics professor Michael Snyder, PhD, will support a project that combines artificial intelligence (AI) with advanced genetic analysis to search for genetic factors involved in ALS.

“I’m excited because we’re putting together a wonderful combination of new AI-based approaches that just weren’t possible a year ago — and a great team, with experts in various fields — to take on a tough problem,” Snyder said in a university news story.

ALS is a progressive, neurodegenerative disease marked by the loss of motor neurons, the nerve cells that control muscle movement. As these cells die, muscles become progressively weaker, and people with ALS gradually lose the ability to walk, speak, swallow, and eventually breathe independently.

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Researchers to study genes, DNA

Although researchers have identified several genetic mutations that increase the risk of developing ALS, these mutations are only present in a minority of patients. The vast majority of cases have no known cause.

According to Snyder, these cases are likely driven by a complex combination of multiple genetic changes and environmental factors rather than a single disease-causing mutation.

Snyder and his team will use newly developed AI-based methods to compare the genetic makeup of people with and without ALS to identify previously unrecognized genetic contributors. They will also examine regulatory regions of DNA that help control which genes are switched on or off in cells.

The team’s analyses will focus on several cell types. In addition to motor neurons, the nerve cells that are lost in ALS, the scientists will study other types of brain cells, as different genes may contribute to the disease in different cell types.

The researchers will test promising genetic targets in a cell model of ALS to see whether altering these genes changes disease-related features, helping determine whether they play a direct role in the disease.

Snyder said the approach has already identified one gene not previously linked to ALS, and the team hopes to uncover many more.

“You don’t want to put all your eggs in one basket; you want to find more genes like this,” Snyder said.

By identifying new genes and regulatory DNA regions linked to ALS, the researchers hope to provide scientists with a broader range of potential therapeutic targets. The team plans to make its findings publicly available so other researchers can build on the work and help accelerate the development of new ALS treatments.