Amyotrophic Lateral Sclerosis (ALS) Fact Sheet
CIRM funds many projects seeking to better understand ALS and to translate those discoveries into new therapies.
About 6,400 people are diagnosed with ALS (also known as Lou Gehrig’s disease) each year in the U.S., and the average survival time is three years. The disease results when the cells in the brain or spinal cord that instruct muscles to move—called motor neurons—die off. People with the disease lose the ability to move their muscles and, over time, the muscles atrophy and people become paralyzed and eventually die. There is no effective therapy for the disease.
California’s stem cell agency has funded several research projects that could help people with ALS (the full list of CIRM awards in this disease is below). Some of those projects are very basic—researchers are trying to understand the origin of the disease and what causes the motor neurons to die. These are the kinds of questions researchers need to understand if they are going to develop the most effective therapies.
With CIRM funding, researchers have made progress understanding which cells are responsible for damaging the motor neurons. It turns out that the cells surrounding those neurons—called astrocytes—are secreting a chemical that damages the neurons. They’ve also learned how to take certain kinds of stem cells and turn them into motor neurons and astrocytes and this might help us better understand the relationship of these cells and even one day prove useful in developing new ways to treat people with ALS.
We also fund projects that are in the later stages of research leading up to and in some cases including clinical trials. These projects, known as our Disease Teams, involve teams of researchers who carry out the experiments that are required before the Food and Drug Administration will allow the potential therapy to be tested in people.
Disease Team Awards
University of California, San Diego
This team of researchers plans to protect surviving neurons in people diagnosed with ALS from further degeneration. They intend to take human embryonic stem cells and turn them into astrocytes—the cells believed responsible for damaging the motor neurons in people with ALS. The goal is to transplant those new cells directly into the spinal cord to see if they can prevent further damage to the neurons.
Cedars-Sinai Medical Center
This team plans to modify neural stem cells as a possible therapy for ALS. The genetically modified stem cells will produce a protein that they hope will protect those cells and also helps protect any remaining nerve cells in the brain that are not already damaged. The team intends to inject those modified stem cells into the brain where they will replace the type of cell—called astrocytes—that are damaged in the disease. They will then run a series of tests to make sure the cells are safe, and to determine if they really do have a protective effect.
CIRM Grants Targeting ALS
CIRM ALS Videos
News and Information
- The Stem Cellar's entries about ALS research
- Mending the Mind: Toward Stem Cell-Based Therapies for Neurological Disease (CIRM)
- CIRM Awards Samuel L. Pfaff $15.6 Million to Develop Stem Cell-Based ALS Therapy (Salk)
- Stem Cellar blogs on ALS
- CDC: ALS Information
- NIH: ALS Fact Sheet
- Find a clinical trial near you: NIH Clinical Trials database
- ALS Association
- ALS Foundation for LIfe
- ALS Hope Foundation
- Family Caregiver Alliance
- National Family Caregivers Association