CIRM Board Approves New Clinical Trial for ALS

This past Friday the governing Board of the California Institute for Regenerative Medicine (CIRM) awarded $11.99 million to Cedars-Sinai to fund a clinical trial for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. 

ALS 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 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 which 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.

“ALS is a devastating disease that attacks the spinal cord and brain and results in the progressive loss of the ability to move, to swallow and eventually to breathe. ” says Maria T. Millan, M.D., President and CEO of CIRM.  “This clinical trial builds on Dr. Svendsen’s work previously funded by CIRM. We are fortunate to be able to support this important work, which was made possible by California citizens who voted to reauthorize CIRM under Proposition 14 this past November.”

8 thoughts on “CIRM Board Approves New Clinical Trial for ALS

    • Hi Susan, we don’t even now if it will work for ALS. If it does then it’s possible it could also be tested for CP but that would be many years away.

  1. Can someone contact me ? My husband was just diagnosed with ALS and we would like to know more about these trials.

    • Hi Mona, I’m so sorry to hear about your husband. I can only imagine how distressing that must be for him and you and the whole family. If you go to the website http://www.clinicaltrials.gov and search there for ALS and stem cells you will find several clinical trials already underway. When the newly approved CIRM-funded trial opens up you’ll find details there about how you can apply to have your husband be part of it. Here is one that is underway already and might be of interest to you. https://clinicaltrials.gov/ct2/show/NCT03268603

  2. I was recently diagnosed with PLS and only the upper motor neurons are affected. Is this something I could be involved in?

    • Hi Michael, it hasn’t started yet but if you check out the http://www.clinicaltrials.gov website and search for ALS and Clive Svendsen you will find the trial including details of how you can determine if you are eligible and then how to apply. The trial isn’t listed there just yet but check in once a week and it should be up soon.

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