The ALS Association

ALS Ice Bucket Challenge Progress

Carrie Munk
The ALS Association


Gene Therapy to Modulate Immune System Shows Potential Therapy Route

Washington, D.C. (October 10, 2014) — In work supported by The ALS Association, researchers have demonstrated the feasibility and potential therapeutic effectiveness of a form of gene therapy in an ALS mouse model. The study was published in the journal Molecular Therapy

ALS (amyotrophic lateral sclerosis), also known as Lou Gehrig’s disease, is a progressive neurodegenerative disease that affects neurons (nerve cells) in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which often leads to total paralysis and death within two to five years of diagnosis. There is no cure and no life-prolonging treatments for the disease. 

One major contributor to the progression of the disease is thought to be neuroinflammation, an immune reaction that, over the long term, worsens the neurodegeneration of ALS. To attempt to alter the neuroinflammation process, the research team used an adeno-associated virus (AAV) carrying a gene for an immune signaling chemical called interleukin-10. They injected the virus into the spinal cord of a mouse model of ALS before symptoms began. They found that treatment increased survival and reduced neuroinflammation. Gene therapy with AAV has been used for experimental treatment in humans in other conditions. AAV is thought to be a very safe viral carrier of therapeutic genes, since it does not provoke an immune response in the recipient.

“These exciting findings establish the proof of principle that modulating immune signaling may help mitigate the disease,” said Lucie Bruijn, Ph.D., M.B.A., Chief Scientist for The Association. “Gene therapy remains an important treatment option, which, based on these results, should be explored further as we seek to find the best treatments for ALS.”

The research was performed by 2014 Milton Safenowitz Post-doctoral Fellow Jacob Ayers, Ph.D., under the direction of Paramita Chakrabarty, Ph.D., and Yona Levites, Ph.D., as well as David Borchelt, Ph.D., and Todd Golde, M.D., Ph.D., all of the University of Florida at Gainesville.

About The ALS Association

The ALS Association is the only national non-profit organization fighting Lou Gehrig’s disease on every front. By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, The Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure. For more information about The ALS Association, visit our website at

Powered by Blackbaud
nonprofit software