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October 2008In This Issue: -Once Again, the Telethon Surpasses Expectations
RESEARCH NEWS-MDA Establishes a Clinical Research Network-Repligen Gets MDA Funding for Friedreich's Research -PTC, Genzyme Team Up to Produce PTC124 for DMD -MDA/ALS Centers Will Help Identify ALS 'Signature' -Researchers Make Motor Neurons from ALS Skin Cells Feedback Privacy Policy Previous Issues: August 2008 May 2008 March 2008 February 2008 December 2007 Pass it along! |
Welcome to the MDA® e-update, the Muscular Dystrophy Association's online newsletter that reports MDA's research breakthroughs and other information to friends whose support helps to make our programs possible. MDA NEWS
Once Again, the Telethon Surpasses ExpectationsThe Jerry Lewis MDA Labor Day Telethon continues to break records, thanks to the magnetism of Jerry Lewis and the incredible largesse of the American public. At the end of the 43rd Labor Day Telethon, broadcast from the South Point Hotel, Casino & Spa in Las Vegas, the tote board read $65,031,393 — some $1.2 million more than last year. Thank you to everyone who helped MDA achieve this success! Sharing With Humanitarian Hurricane AidAs the Telethon was getting under way, so was Hurricane Gustav. Gulf Coast residents evacuated by the millions. Telethon star Jerry Lewis urged families affected by muscular dystrophy and related diseases — some 5,000 in the Gulf states — to seek MDA assistance wherever they eventually touched down. Just as when Hurricanes Katrina and Rita were wreaking havoc in 2005, Lewis again asked Telethon viewers to donate not only to MDA, but also to the Salvation Army's Emergency Disaster Services. Gustav, fortunately, didn’t deploy its full potential for destruction. Empire State Building Salutes
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In special recognition of MDA and the Telethon this year, the Empire State Building put on a lighting display from Aug. 29 through Sept. 1. For three nights from sunset to midnight, the upper third of the building was bathed in alternating white and blue light panoramas. "The Empire State Building is an American icon," said MDA National Chairman Jerry Lewis. "It was an honor to see the MDA blue up in lights throughout Telethon weekend."
Arizona Man Earns Top Recognition
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Tom Bush |
Tom Bush of Oro Valley, Ariz., has received the Robert Ross National Personal Achievement Award for 2009. The award recognizes people with muscular dystrophy and related neuromuscular diseases who have demonstrated exceptional personal achievement and community service involvement. Bush retired from a 30-year career as director of the New Jersey Office of Disabilities Management. His work included implementing the then-newly enacted Americans with Disabilities Act (ADA). Next, Bush worked 10 years at MDA national headquarters in Tucson, Ariz., developing and managing the Association’s Web site, before his "real" retirement. He also has been very active with a large number of community organizations, especially those dealing with transportation and employment issues for people with disabilities.
Abbey Returns as Goodwill Ambassador
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Abbey Umali |
At Jerry Lewis' request, 9-year-old Abbey Umali will serve a second term as MDA's National Goodwill Ambassador for 2009. Throughout 2008, Abbey and her parents, Joel and Wendi, have done a fantastic job traveling the country speaking at fundraisers and other special events. The charming young lady from Redlands, Calif., captivated audiences everywhere, including on stage in Las Vegas at the national broadcast of Telethon. When not on the road, she attends Montessori School, where her favorite subjects are reading, math and geography and, "of course," she says, "recess!"
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Tee Off in the
Jerry Lewis Open
MDA and World Golf Tour are bringing the greens to you in stunning high definition to help Jerry’s Kids. Now is your chance to join the first online Jerry Lewis Open, enjoy virtual play on famous courses, compete and win prizes — all while raising funds for MDA. Details and registration instructions are on the Web site, http://www.jerrylewisopen.org/ec/.
RESEARCH NEWS
MDA Establishes a Clinical Research Network
The network is designed to speed testing and development of new treatments for two neuromuscular diseases. MDA selected 10 of its 225 clinics to serve as Clinical Research Network Centers and committed $1 million annually to fund them. Five centers will coordinate clinical trials and other studies in ALS (amyotrophic lateral sclerosis), and five will focus on Duchenne muscular dystrophy (DMD). The network eventually may expand to cover additional diseases.
Repligen Gets MDA Funding for
Friedreich's Research
MDA recently awarded $1 million to Repligen Corp. of Waltham, Mass., to develop treatments for Friedreich's ataxia (FA). In FA, severe cardiac and neurologic problems result from a deficiency of the protein frataxin, caused by a mutation in the gene for that protein. Repligen research will include determining if a class of compounds known as HDAC inhibitors may be able to activate silent frataxin genes and restore frataxin protein production.
PTC, Genzyme Team Up to Produce
PTC124 for DMD
MDA previously awarded PTC Therapeutics of South Plainfield, N.J., a grant of $1.5 million to begin developing PTC124, a medication with promise for treating DMD. Now PTC and Genzyme Corp., a biopharmaceutical company based in Cambridge, Mass., have teamed up to further develop and commercialize PTC124. In DMD, the muscle protein dystrophin is missing in muscle cells, causing their progressive degeneration. In about 15 percent of DMD cases, a specific genetic error called a "nonsense mutation" inhibits functional dystrophin production. PTC reports that in clinical trials about half of DMD patients with this genetic error who took PTC124 began producing dystrophin.
MDA/ALS Centers Will Help
Identify ALS 'Signature'
MDA is currently involved, through its Augie's Quest research initiative, in funding a three-year, $18 million scientific collaboration with the ALS Therapy Development Institute (ALS TDI) of Cambridge, Mass. Now the two organizations are teaming up to collect blood and tissue samples from people with ALS who are treated at MDA/ALS centers in Texas, California and New York. The intent is to identify a molecular "signature" of ALS that will help with diagnosis and monitoring, and possibly provide new targets for experimental treatments.
Researchers Make Motor Neurons
from ALS Skin Cells
Scientists supported by MDA at Harvard and Columbia Universities have created motor neurons (muscle-controlling nerve cells) from the skin cells of a woman with a familial form of ALS. An eventual goal of such experiments is to replace patients' disease-affected cells with healthy ones from their own bodies. Scientists cautioned, though, that several hurdles must be crossed before the concept can become reality. Among them: being sure that replacement cells go to the right place in the brain or spinal cord, and connect to muscles and other nerve cells correctly. A near-term goal is to study what goes wrong in disease-affected cells. MDA-supported researchers in the work included Hiroshi Mitsumoto, director of the Eleanor and Lou Gehrig MDA/ALS Center at Columbia.
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