In individuals with ALS muscle control gradually and rapidly degenerates, until all brain control on voluntary muscle movement is lost. Gene regulatory mechanisms that control spinal neuron differentiation from hES cells.Amyotrophic Lateral Sclerosis (ALS) is a rapidly progressive neurological disease that attacks motor neurons located in the brain and spinal cord, which are responsible for voluntary muscle control. Therapeutic Translational Research Projects Human Embryonic Stem Cell-Derived Neural Stem Cell Transplants in Amyotrophic Lateral Sclerosis In vitro differentiation of hESCs into corticospinal motor neurons Genetic manipulation of human embryonic stem cells and its application in studying CNS development and repairĭevelopment of a Relevant Pre-Clinical Animal Model as a Tool to Evaluate Human Stem Cell-Derived Replacement Therapies for Motor Neuron Injuries and Degenerative Diseases The CIRM funding will enable the company to test this therapy, called NurOwn, in a Phase 3 trial involving around 200 patients. 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. For more information about this clinical trial, click here.īrainStorm is using mesenchymal stem cells that are taken from the patient’s own bone marrow to treat patients with ALS.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. 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. 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. This team of researchers plans to protect surviving neurons in people diagnosed with ALS from further degeneration. Clinical Stage Programs Cedars-Sinai Medical Center You can read more about these trials below. Recently, CIRM has funded research into ALS that has advanced into clinical trials. Food and Drug Administration will allow the potential therapy to be tested in people. These projects involve teams of researchers who carry out the experiments that are required before the U.S. We also fund projects that are in the later stages of research leading up to and in some cases including clinical trials. 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. ![]() It turns out that the cells surrounding those neurons-called astrocytes-are secreting a chemical that damages the neurons. With CIRM funding, researchers have made progress understanding which cells are responsible for damaging the motor neurons. These are the kinds of questions researchers need to understand if they are going to develop the most effective therapies. 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. ![]() ![]() There is no effective therapy for the disease.Ĭalifornia’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). People with the disease lose the ability to move their muscles and, over time, the muscles atrophy and people become paralyzed and eventually die. The disease results when the cells in the brain or spinal cord that instruct muscles to move-called motor neurons-die off. DescriptionĪbout 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. CIRM funds many projects seeking to better understand ALS and to translate those discoveries into new therapies.
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