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New clinical trial approved for ALS

OAKLAND, CA (US), June 2021 — A new clinical trial designed to determine the feasibility of using genetically engineered stem cells to treat amyotrophic lateral sclerosis (ALS) is being launched by Cedars-Sinai.

ALS, also known as Lou Gehrig’s disease, is a neurodegenerative disease that results in the death of nerve cells in the brain and spinal cord, causing the muscles in the body to gradually weaken, leading to loss of limb function, difficulty breathing, paralysis, and eventually death. There are medications that can slow down the progression of ALS, but unfortunately there is no cure for the disease.

Clive Svendsen, Ph.D., executive director of Cedars-Sinai’s Board of Governors Regenerative Medicine Institute, and his team will be conducting a trial that uses a combined cell and gene therapy approach as a treatment for ALS. The trial builds upon the stem cell agency’s first ALS trial, also conducted by Cedars-Sinai and Dr. Svendsen.

Genetically engineered stem cells will be transplanted into the motor cortex, an area of the brain responsible for voluntary movements. These transplanted cells then become astrocytes, a type of support cell that help keep nerve cells functioning. The astrocytes have been genetically altered to deliver high doses of a growth factor that has been shown to protect nerve cells. 

The goal of this approach is to protect the upper motor neurons controlling muscle function and meaningfully improve the quality of life for ALS patients.

The governing board of the California Institute for Regenerative Medicine (CIRM) awarded $11.99 million to Cedars-Sinai and Dr. Svendsen to conduct the trial.

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