CIRM Provides $17.8 Million Grant To Develop Combined Stem Cell And Gene Lou Gehrig’s Disease (ALS) Treatment

Stem cells alone have the potential to protect damaged motor neurons in ALS. This potential increases when combined with the additional known effects of  the protein GDNF which promotes the survival of nuerons. Currently there is no therapy that will cure or slow the progress of amyotrophic lateral sclerosis (Lou Gehrig's disease).

In the past, delivering GDNF to the brain or spinal cord has been nearly impossible because it does not cross from the blood to the tissue of the spinal cord.  Cedars believes they have a way to overcome this.

“We’re looking at a novel and exciting way of using stem cells as ‘Trojan horses’ that arrive at the sick motor neurons and delivers the protein exactly where it’s needed,” Clive Svendsen, PhD, director of the Regenerative Medicine Institute,. “Our early study indicates this approach has significant potential and we’re excited to bring this treatment a step closer to helping ALS patients.”

About 30,000 patients in the United States have ALS, which affects nerve cells in the brain and the spinal cord. Motor neurons connect the brain to the spinal cord and then to the muscles throughout the body. Degeneration of these motor neurons is eventually fatal to ALS patients. As the motor neurons die, the brain loses the ability to initiate and control muscle movement. Patients in later stages of the disease become paralyzed.

The Cedars-Sinai (previous post) proposal involves collaborations with Emory University in Atlanta and California Pacific Medical Center, bringing together teams of scientists, neurologists, neurosurgeons and nurses to take the stem cells from animal studies, through the regulatory process of FDA approval and into an 18-patient clinical trial for ALS in four years.

Adapted from the Cedars-Sinai announcement.