Contact:
Carrie Munk
The ALS Association
(571) 319-3047
cmunk@alsa-national.org

 

FOR IMMEDIATE RELEASE

Leaders of iPSC Technology Encourage Cohesive Approach to Speed ALS Therapy Development

Washington, D.C. (March 31, 2016)— In a state-of-the-art review, leaders in the field of induced pluripotent stem cell (iPSC) technology to derive motor neurons from people living with ALS, identify advances and challenges in the ability to model ALS with these cells and call for the development and adoption of stringent criteria to enable comparison of results among independent laboratories. The review titled, “Modeling ALS with motor neurons derived from human induced pluripotent stem cells” was published this week in the top-tier journal Nature Neuroscience by a team headed by Clive Svendsen, Ph.D. from Cedars-Sinai Medical Center in Los Angeles, CA.

ALS is a progressive neurodegenerative disease that affects 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. For unknown reasons, veterans are twice as likely to develop ALS as the general population. There is no cure, and only one drug approved by the U.S. Food and Drug Administration (FDA) modestly extends survival.

“This review was generated as a result of a round table discussion hosted by The ALS Association bringing together leaders in the field to discuss and compare protocols. This resource will provide invaluable guidance to researchers using iPSCs in ALS research and therapy development,” said co-author Lucie Bruijn, Ph.D., M.B.A., ALS Association Chief Scientist. “These cells have emerged as an important tool for modeling the disease—motor neurons in culture that are derived from individual ALS patients. They have the potential to identify new disease mechanisms and individual susceptibilities to disease that cannot be revealed with other models. This may allow iPSCs to serve as exceptionally valuable tools to find new treatments based on a person’s unique genetic make-up.”

To reach that potential, the authors point out, will require a deeper understanding of how to better tailor motor neurons derived from iPSCs to match motor neurons in living organisms, work that is currently underway. In addition, the authors urge the field to agree on standards for evaluation of motor neurons in order to allow different labs to compare results, a key step in accelerating therapy development.

Dr.’s Clive Svendsen and Dhruv Sareen are funded through the Neuro Collaborative. Cell lines are now available to investigators worldwide through this collaboration. For a listing of the currently available ALS iPSC lines click here.

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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