New Technique Generates ALS-Derived Muscle for Study
Washington, D.C. (March 25, 2014) — In work supported by The ALS Association, scientists have developed a new method for generating muscle cells from skin tissue of people with ALS, which will be valuable for research. The study was published in the journal Stem Cells Translational Medicine.
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.
The scientific team was led by Masatoshi Suzuki, Ph.D., D.V.M., of the University of Wisconsin at Madison, and included Allison Ebert, Ph.D., of the Medical College of Wisconsin. Their work was supported by the Wisconsin Chapter of The ALS Association.
The researchers showed that muscle progenitor cells could be efficiently produced from stem cells in cell culture, using a new combination of growth factors. The method works with either human embryonic stem cells or induced pluripotent stem cells (iPS cells), which are derived from skin cells. In the study, the researchers transformed iPS cells from two different people with familial forms of ALS, one caused by mutations in the SOD1 gene and the other by mutations in the VAPB gene.
“The ability to generate muscle cells from ALS-related tissue is important, because we need to understand more about the role of muscle in the disease process,” said Lucie Bruijn, Ph.D., MBA, Chief Scientist for The Association. “We know an early step in ALS is loss of contact between muscle and motor neuron. This system should allow us to determine more about that important step and determine whether, if we can delay it, we can prolong both neuron health and muscle function.”
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 http://www.alsa.org.