Cedars-Sinai Regenerative Medicine Institute today was awarded a $17.8 million grant from the California Institute for Regenerative Medicine to develop stem cell treatments for patients with ALS, also known as Lou Gehrig's Disease, a progressive, fatal and currently incurable neurodegenerative disease.
The grant - the largest to date from the stem cell agency to Cedars-Sinai -- will fund a project with combined stem cell and gene therapy to treat amyotrophic lateral sclerosis with stem cells found in early brain development and a protein called GDNF that promotes the survival of neurons. The stem cells alone have the potential to protect damaged motor neurons in ALS. This potential increases when combined with the additional known effects of GDNF. 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.
"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 and leader of the project,. "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.
"For the thousands living with ALS, there is no therapy now that can cure or slow their disease," said Shlomo Melmed, MD, senior vice president of Academic Affairs, dean of the Medical Faculty and Helene A. and Philip E. Hixon Chair in Investigative Medicine. "We're grateful that this grant will allow us to vigorously pursue novel therapies and benefit ALS patients."
The Cedars-Sinai 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.
Svendsen expressed his thanks to Sheryl Osborne, a San Diego-based consultant, as well as their collaborators and his research team at Cedars-Sinai.
"I am grateful for the dedication our team has shown to ALS," Svendsen said. "We have a hard road ahead, and everyone should be humbled by the aggressiveness of this devastating disease. This combined gene and cell therapy approach may be one hope to fight back. With this CIRM funding, we can now move the study forward."
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