ALS Philadelphia Summer 2013 Newsletter

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Research Update ALS Association-Supported Study Shows Combining Two Growth Factors May Have Advantage in ALS Treatment May 31, 2013 A study published in the scientific journal Molecular Therapy, which was supported by the Wisconsin Chapter of The ALS Association and led by University of Wisconsin at Madison researchers, shows in ALS that two growth factors may be better than one. Growth factors are proteins produced by one set of cells in the body that help promote the health of other cells. Previous work has shown that both VEGF (vascular endothelial-derived growth factor) and GDNF (glial-derived neurotrophic factor) can promote the survival of motor neurons in models of ALS. Motor neurons are the cells that die in ALS and lead to paralysis. “This study provides support for the idea that delivering growth factors to muscle may be beneficial in ALS,” said Lucie Bruijn, Ph.D., Chief Scientist at The ALS Association. “Further work will be needed to explore this option, but it has the advantage of being less invasive than delivering the same growth factors to the spinal cord.”

Ben Ohrenstein learns about research from Lucie Brujin, PhD.

Masatoshi Suzuki, Ph.D., and colleagues tested whether the two growth factors together could provide more benefit than either alone. These researchers engineered stem cells to produce both growth factors and then injected these cells into the muscles in a rat model of ALS. The combined treatment increased lifespan and improved features of the motor neurons at the point where they contact muscle. On May 13, Lucie Bruijn, PhD., Chief Scientist at The ALS Association and James D. Berry, MD, MPH, Unit Chief at Massachusetts General Hospital spoke in Philadelphia about improving understanding and expanding treatment horizons in ALS. Scientists are making significant progress in understanding the potential causes of ALS in both the familial and sporadic forms as well as new directions for possible treatments. You can watch video of Dr. Brujin’s presentation and learn more at www.alsphiladelphia.org/research

Executive Director Jim Pinciotti continued from page 3 This year, Pat & I celebrated our 38th wedding anniversary while surrounded by over 7,000 friends. While we may not know all 7,000 of the Phillies fans by name, they are certainly friends in our quest to defeat ALS. Being with Pat at the Phillies Phestival was a reminder that our families are never alone in the battle against ALS. While I’m proud of the support we provide through our clinics, our in-home care program, assistive technology, durable medical equipment, and groundbreaking research, moments like these are a strong reminder of how important it is for us to make sure people are able to spend time with their families as well. We talk a lot about how we are together in hope, but we are also together in love and support for one another, even in the face of a terrible disease. I hope that you get many opportunities to spend time with your families this summer. If you get a chance to be with your family at a Phillies game, make sure to tell them thank you!


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