Amyotrophic lateral sclerosis (ALS) Fact Sheet

Amyotrophic lateral sclerosis (ALS) Fact Sheet

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.

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

Stem cell research for ALS

About 5,600 people are diagnosed with ALS (also known as Lou Gehrig’s disease) each year in the U.S., and the majority of those people survive only about four 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

Researcher name Institution Grant Title Approved funds
Fred Gage The Salk Institute for Biological Studies Molecular and Cellular Transitions from ES Cells to Mature Functioning Human Neurons $2,749,293
Ying Liu University of California, San Diego Generation of disease models for neurodegenerative disorders in hESCs by gene targeting $709,829
Bin Chen University of California, Santa Cruz Molecular mechanisms of neural stem cell differentiation in the developing brain $2,147,592
Steven Finkbeiner The J. David Gladstone Institutes Development of Novel Autophagy Inducers to Block the Progression of and Treat Amyotrophic Lateral Sclerosis (ALS) and Other Neurodegenerative Diseases $2,278,080
Eric Ahrens University of California, San Diego Molecular Imaging for Stem Cell Science and Clinical Application $5,924,428
Binhai Zheng University of California, San Diego Genetic manipulation of human embryonic stem cells and its application in studying CNS development and repair $600,441
Bin Chen University of California, Santa Cruz In vitro differentiation of hESCs into corticospinal motor neurons $465,624
Samuel Pfaff The Salk Institute for Biological Studies Gene regulatory mechanisms that control spinal neuron differentiation from hES cells. $704,543
Jeremy Reiter University of California, San Francisco High throughput modeling of human neurodegenerative diseases in embryonic stem cells $2,259,092
Hans Keirstead University of California, Irvine hESC-Derived Motor Neurons For the Treatment of Cervical Spinal Cord Injury $2,158,445
Clive Svendsen Cedars-Sinai Medical Center Progenitor Cells Secreting GDNF for the Treatment of ALS $16,961,287
Zack Jerome University of California, Los Angeles Generation of clinical grade human iPS cells $1,341,000
Fred Gage The Salk Institute for Biological Studies Development of Induced Pluripotent Stem Cells for Modeling Human Disease $1,737,720
Clive Svendsen Cedars-Sinai Medical Center Stem Cells Secreting GDNF for the Treatment of ALS $63,487
Eugene Yeo University of California, San Diego Molecules to Correct Aberrant RNA Signature in Human Diseased Neurons $1,532,323
Eugene Yeo University of California, San Diego Stem cell models to analyze the role of mutated C9ORF72 in neurodegeneration $1,260,360
Bennett Novitch University of California, Los Angeles Molecular Characterization of hESC and hIPSC-Derived Spinal Motor Neurons $1,229,922
Lawrence Goldstein University of California, San Diego Stem Cell-Derived Astrocyte Precursor Transplants in Amyotrophic Lateral Sclerosis $4,139,754
Lawrence Goldstein University of California, San Diego Stem Cell-Derived Astrocyte Precursor Transplants in Amyotrophic Lateral Sclerosis $5,694,308
Total:
$53,957,528.00

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