Contact:
Carrie Munk
The ALS Association

cmunk@alsa-national.org

 

FOR IMMEDIATE RELEASE

ALS Cell Models May Need a Little Aging

Washington, D.C. (July 18, 2016) — Researchers funded by The ALS Association through the Neuro Collaborative have developed deeper insights into the fidelity of cell-based models of ALS, according to a new study published in the journal Nature Neuroscience. The results point toward aging as a potentially key feature to be incorporated into new cellular models. Data and statistical models generated in the study will be available to the ALS research community in order to speed the development of new treatments.

ALS (amyotrophic lateral sclerosis) 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.

Induced pluripotent stem cells (iPSCs) derived from ALS patients have become important sources of neurons for modeling the disease and screening for new treatments. However, Clive Svendsen, PhD, and Ritchie Ho, PhD, of Cedars-Sinai in Los Angeles, along with colleagues, have demonstrated that this technique reverts the patient’s tissue to a state more similar to embryonic neurons than to the aged neurons that are affected in ALS. They have also explored the implications of this similarity and found that critical age-related changes in molecular activity were exacerbated by ALS itself, indicating that the genes involved may be important therapeutic targets. Thus, it might be beneficial to induce some aspects of aging in iPSC-derived neurons so that they accumulate critical vulnerabilities similar to those of aged neurons that die from ALS. This would make them better models for understanding disease processes and screening for potential therapies, Svendsen said.

In addition to laying the foundation for improving iPSC-based cell models, Svendsen and colleagues identified a test panel of motor neuron maturation markers that will aid future work in manipulating the correct maturation and aging of cells in culture. “By knowing the gene expression patterns that define adult motor neurons in the spinal cord, we can push the iPSC-derived motor neurons in the right direction in the petri dish,” said Svendsen, director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute, the Kerry and Simone Vickar Family Foundation Distinguished Chair in Regenerative Medicine and professor of Medicine and Biomedical Sciences.

“This important study will help ALS researchers better model the disease in cells,” said Lucie Bruijn, Ph.D., M.B.A., Chief Scientist for The ALS Association. “iPSCs have become vital for understanding the disease and screening for new treatments, and by applying the results from this study, they can become even more valuable to the development of therapies for ALS. The discovery that age-related gene changes are also a consequence of the ALS disease process points directly toward possible targets for new treatments.

About the Neuro Collaborative
The Neuro Collaborative is a partnership between three leading laboratories in California: Clive Svendsen, Ph.D., at Cedars-Sinai in Los Angeles; Steven Finkbeiner, M.D., Ph.D. at Gladstone Institutes, affiliated with University of California San Francisco; and Don Cleveland, Ph.D. at University of California San Diego. In 2014, The ALS Association Golden West Chapter and patient advocate Jim Barber partnered to build the Neuro Collaborative concept. That year, following the amazing outpouring of support from the ALS Ice Bucket Challenge, The ALS Association committed $5 million dollars to the project. With additional help from The ALS Association Orange County and Wisconsin Chapters, the Neuro Collaborative has become an engine for ALS therapeutics.

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 www.alsa.org.

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