For the past four years, Cedars-Sinai and Dr. J. Patrick Johnson, along with Robert Baloh, MD, Ph.D., and the ALS team, have been testing a combination stem cell-gene therapy developed to slow the progression of amyotrophic lateral sclerosis (ALS).
ALS, often referred to as Lou Gehrig’s disease, is a progressive disease that affects nerve cells in the brain and the spinal cord and leads to the loss of muscle control. Currently, there is no effective treatment for ALS. This new therapy involves engineering stem cells to produce the protein glial cell line-derived neurotrophic factor (GDNF), which can then protect the motor neurons from progressing to cell death and preserve neurologic function.
Any time you’re trying to treat an incurable disease, it is a long shot, but we believe the rationale behind our new approach is strong.”
~ Clive Svendsen, Ph.D., Director, Cedars-Sinai Board of Governors Regenerative Medicine Institute
The neurosurgical team for the study was led by J. Patrick Johnson, MD. As lead surgeon, Dr. Johnson performed the surgeries that delivered the cells to ALS patients that were entered into the program. The study is still ongoing, and patient outcomes will be followed for years to come, and findings are expected to be published in several major scientific journals soon.
Dr. J. Patrick Johnson talks with Dr. Phil McGraw on Phil In the Blanks