Understanding the basic biology of how a cell functions can be crucial to better understanding a disease and unlocking a potential treatment approach. Stem cells are unique because they let scientists create a controlled environment of cells that might otherwise be difficult to study.
Dr. Eva Hedlund and a team of researchers at the Karolinska Institute in Sweden utilize a stem cell model approach to uncover findings related to Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s Disease.
What is ALS?
ALS is a progressive neurodegenerative disease that destroys motor neurons, nerve cells essential for voluntary muscle movement. When motor neurons can no longer send signals to muscles, the muscles begin to deteriorate, a process called atrophy. P
rogressive atrophy leads to muscle paralysis, including in the legs and feet, arms and hands, and the muscles that control swallowing and breathing. It affects about 30,000 people in the United States alone, with 5,000 new cases diagnosed each year.
There is currently no cure.
ALS-resilient neurons
In a previous study, researchers at the Karolinska Institute successfully created oculomotor neurons from embryonic stem cells. For reasons not yet fully understood, oculomotor neurons are “ALS resilient” and can survive all stages of the disease.
In the current study, published in Stem Cell Reports, Dr. Hedlund and her team found that the oculomotor neurons they generated appeared more resilient to ALS-like degeneration than spinal cord motor neurons, a pattern commonly observed in humans. They also found that their “ALS-resilient” neurons, generated from stem cells, activate a survival-enhancing signal known as Akt, which is common in human oculomotor neurons and could explain their resilience. These results could help identify genetic targets for treatments that protect sensitive neurons from the disease.
In a press release, Dr. Hedlund is quoted as saying,
“This cell culture system can help identify new genes contributing to the resilience in oculomotor neurons that could be used in gene therapy to strengthen sensitive motor neurons.”
CIRM is currently funding two clinical trials for ALS: one at Cedars-Sinai Medical Center and another at Brainstorm Cell Therapeutics. The latter trial is currently recruiting patients, and information on how to enroll is available here.