Stem cell therapy for ALS
The power of stem cells lies in their potential. Embryonic stem cells are pluripotent, meaning they can grow into any type of cell in the body – including bone, blood, muscle, skin, and neurons. In recent years, researchers have learned how to circumvent the need for controversial embryonic stem cells by producing human-induced pluripotent stem cells (iPSC) in the lab from adult skin or blood cells.
In ALS, one current research effort focuses on the use of stem cells to deliver nutrients that help repair damaged motor neurons. In late 2016, biotech company Brainstorm Cell Therapeutics announced plans to launch a phase III clinical trial of NeurOwn stem cells, which are derived from bone marrow.
Cedars-Sinai Medical Center is testing a combination stem cell-gene therapy treatment to stall the progress of ALS. The stem cells are engineered in a lab to produce glial cell line derived neutrophic factor (GDNF) – a protein that helps protect motor neurons.
“Motor neurons that die in ALS don’t exist in a vacuum – they have support cells called glia that enable the motor neurons to live and operate,” said Clive Svendsen, PhD, the Kerry and Simone Vickar Family Foundation Distinguished Chair in Regenerative Medicine. “What we and others have found in ALS is that the support cells become sick and lack certain proteins that keep motor neurons alive. When the support cells die, the motor neurons also die, causing paralysis that gets worse and worse until the patient can no longer move.”
By providing the supportive glial cells, Svendsen and his team hope to keep motor neurons alive, and keep patients’ moving. “This is a highly innovative approach to a disease that has mystified medicine for generations,” said Shlomo Melmed, MD, Cedars-Sinai Philip E. Hixon Distinguished Chair in Investigative Medicine.
March 16, 2020
Christopher Nystuen, MD, MBA