Amyotrophic lateral sclerosis (ALS) Fact Sheet

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Amyotrophic lateral sclerosis (ALS)

Neurospheres derived from human embryonic stem cells forming connections (Fred H. Gage at the Salk Institute for Biological Studies)

CIRM funds several research projects investigating the causes of ALS, also known as Lou Gehrig’s Disease, and developing novel stem cell-based therapies for the disease.

If you want to learn more about CIRM funding decisions or make a comment directly to our board, join us at a public meeting. You can find agendas for upcoming public meetings on our meetings page.

Learn more about stem cell research:
Stem Cell Basics Primer | Stem Cell Videos | What We Fund

Find clinical trials:
CIRM does not track stem cell clinical trials. If you or a family member is interested in participating in a clinical trial, please see the national trial database to find a trial near you: clinicaltrials.gov

Description

ALS is a progressive disease that involves the death of motor neurons. The disease affects upper motor neurons—cells in the brain that carry information about muscle motion—and lower motor neurons—cells in the spinal cord that connect directly to muscle. As these cells die, signals from the brain to the muscle are cut off and patients lose motor control. Over time the muscle begins to atrophy and patients experience paralysis and, since ALS is progressive, death.

Symptoms vary from patient to patient. They include muscle weakness in the hands, arms, legs and muscles in the mouth and throat, twitching or cramping of muscles, thick speech and difficulty breathing and swallowing. ALS is often diagnosed in late stages of the disease, after significant cell loss has occurred. Survival times vary.

In people with the inherited form of ALS, mutations in the gene that produces the enzyme SOD1 are associated with neurodegeneration. SOD1 prevents DNA damage to cells caused by free radicals. People with spontaneous ALS have higher levels of the neurotransmitter glutamate in the spinal fluid. Since cells exposed to high levels of glutamate begin to die in lab experiments, scientists suspect that glutamate may be involved in ALS, but it isn’t yet clear what causes glutamate to build up.

Larry Goldstein of UC San Diego discusses the CIRM ALS Disease Team's effort to develop an ALS therapy

CIRM Grants Targeting ALS


CIRM ALS Videos

Disease Team Award

One of the research projects targeting ALS is a multidisciplinary team led by researchers at the University of California, San Diego. All Disease Team Award recipients are expected to meet milestones along the path to reaching FDA clinical trial submission within four years. 

The team plans to protect surviving neurons in people diagnosed with ALS from further degeneration. The strategy involves targeting glial cells, which are neuroprotective cells that surround and support neurons. A type of glial cell called an astrocyte is found in both the brain and spinal cord and acts as a regulator of glutamate surrounding motor neurons. The team intends to grow human embryonic stem cell-derived astrocyte precursors that will be transplanted directly into the spinal cord environment to prevent further neurodegeneration caused by ALS. The work, which is based on mouse experiments, should be effective in both familial and sporadic ALS.

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