ALS (Lou Gehrig’s disease)
ALS affects the motor neurons in the spinal cord, leading to paralysis of motor functions such as breathing. The cause of ALS is not known but appears to be related to the dysfunction of critical neuronal and supporting elements in the spinal cord. The immune system may also play a role in disease progression.
There are approximately 400,000 people worldwide with ALS. The disease typically begins in adulthood and, in most cases, is fatal within three to four years of onset.
In 2012, Brigham and Women’s Hospital scientists discovered that changes in a type of white blood cell are a hallmark of ALS. Cutting-edge research like this at the Ann Romney Center for Neurologic Diseases paves the way for innovative treatments that attack the disease at its molecular core, bringing newfound hope to ALS patients and their loved ones.
“My hope is that the Ann Romney Center goes down in history
as the place where cures for MS, Alzheimer’s, ALS, and other devastating
neurologic diseases were first discovered.” —Ann Romney
For information about ALS and associated resources at BWH, visit: http://www.brighamandwomens.org/Departments_and_Services/neurology/services/neuromuscular/services/amyotrophic-lateral-sclerosis.aspx
MOVING TOWARD A NEW THERAPY FOR ALS
Working closely with collaborators at Massachusetts General Hospital, Oleg Butovsky, PhD, is studying microRNA to develop potential new treatments for ALS. Dr. Butovsky and his team have focused their attention on miR-155, a microRNA molecule that plays a major role in regulating inflammation, and may be involved in the progression of ALS and other neurodegenerative conditions. Building on their studies showing that inhibiting MiR-155 prolonged life in animal models and lowered levels in human cells, Dr. Butovsky and his team have partnered with miRagen Therapeutics—a biopharmaceutical company that specializes in developing microRNA-targeting therapies—to develop specific inhibitors to shut down MiR-155 in humans. In the past year, they have identified two promising blockers that will soon be ready for clinical trials.
A POTENTIAL NEW DRUG TARGET FOR ALS
Working toward potential treatment options for this devastating disease, Dr. Weiner and his team have identified a specific microRNA—a small RNA molecule that regulates gene expression—that is elevated in the blood and spinal fluid of patients with ALS and may play a key role in disease progression. Studies in the lab have shown that this particular microRNA—MiR-155—can be blocked by administering an anti-MiR-155 compound, prolonging life in animal models and showing promising results by lowering levels in human cells. Dr. Weiner and his team, which includes collaborators at Massachusetts General Hospital, Johns Hopkins University, and UMass Memorial Medical Center, are now working with a pharmaceutical company to develop a drug aimed at this unique and promising target.
BWH Office of Strategic Communications