Stem Cell Can Help
Amyotrophic Lateral Sclerosis
CIRM funds many projects seeking to better understand ALS and to translate those discoveries into new therapies.
About 6,000 people are diagnosed with ALS (also known as Lou Gehrig’s disease) each year in the U.S., and the average survival time is two to five 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 involve teams of researchers who carry out the experiments that are required before the U.S. Food and Drug Administration will allow the potential therapy to be tested in people. Recently, CIRM has funded research into ALS that has advanced into clinical trials. You can read more about these trials below.
Clinical Stage Programs
Cedars-Sinai Medical Center
This team of researchers plans to protect surviving neurons in people diagnosed with ALS from further degeneration. They will implant middle-man “progenitor” cells made by maturing stem cells from fetal tissue down a path destined to become astrocytes, the brain cells that protect nerves and that become defective in ALS. Those cells will be boosted with genes for a growth factor that when the cells release it after transplantation, will have an added protective effect on nerves. This approach recently received approval to treat ALS patients in a CIRM-funded clinical trial (read here). A feature story on this work appeared in The Stem Cellar blog in 2017.
Brainstorm Cell Therapeutics
BrainStorm is using mesenchymal stem cells that are taken from the patient’s own bone marrow to treat patients with ALS. These stem cells are then modified to boost their production of neurotrophic factors, which are known to help support and protect neurons, the cells destroyed by the disease. The CIRM funding will enable the company to test this therapy, called NurOwn, in a Phase 3 trial involving around 200 patients.