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MDA NEWS

Fixing ALS in the Public Consciousness

Throughout May, public attention will be directed to a deadly disease that strikes thousands of Americans every year: ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease).

As part of the 18th annual National ALS Awareness Month, MDA will conduct activities and programs across the country to heighten public awareness of the disease and the vital MDA-supported research efforts under way to stop it in its tracks.

ALS plays no favorites, striking healthy adults from all walks of life, often in the prime of their lives, by killing nerve cells that control muscles. The result is gradual paralysis of the body's voluntary muscles, including those used for breathing and swallowing. Death often occurs within three to five years of diagnosis, although lives are being extended in some cases through technologies such as assisted ventilation and feeding tubes.

ALS: “Anyone’s Life Story”

Each day in May the story of a different person with ALS will appear on the MDA ALS Division Web site. The stories are profiles in courage, character and love.

Phil Kelleher was a proud fire fighter who had begun building a dream home for his family. Robert Paulson was a top patent attorney for 40 years, and loved to sing. Susan Fox used to ice skate, play the piano and dance. ALS took all that way. In the 31-day series “Anyone’s Life Story,” you'll hear from these three and 28 others about the ways ALS has changed, and yet also enriched, their lives.

The spirit and optimism of the people profiled in the series is part of what drives MDA's relentless quest, now nearly half a century old, to defeat ALS. MDA-funded researchers around the world are working to uncover clues that will lead to treatments and a cure for this cruel disease.

Nancy O'Dell New MDA ALS Ambassador

Nancy O'Dell, longtime host of the entertainment-news show “Access Hollywood,” has accepted a new role as MDA National ALS Ambassador.

O'Dell will spread awareness of MDA's fight against ALS through media interviews, public appearances and public service announcements. Her work will complement the work of MDA ALS Division Co-Chairs Augie and Lynn Nieto.

For a second year, O'Dell will co-host the Jerry Lewis MDA Telethon this Labor Day weekend.

In June 2008, ALS claimed the life of O'Dell's mother, Betty Humphries, who was 74.

Latest ALS Video

MDA has produced a full-length informational DVD for people newly diagnosed with ALS and their families. With Hope and Courage: Your Guide to Living with ALS is available at MDA local offices and is a resource for those confronted by the uncertainties, health challenges and decision-making associated with the disease.

Caregiver's Guide Remains Popular

The MDA ALS Caregiver's Guide, a 210-page spiral-bound book released a year ago, continues to get high marks from both caregivers and the people they assist. The book addresses topics such as nutrition, breathing, communication, finances, insurance accessibility and much more. It includes comments from actual caregivers and those with ALS. The guide is a supplement to MDA's award-winning 2005 publication Everyday Life with ALS. Both books are available in their entirety online. Hard copies are available free to those with ALS registered with MDA, and $15 for others.

Augie's Quest Forges Ahead

More than three years ago, Augie and Lynn Nieto worked with MDA's ALS Division to create Augie's Quest, an aggressive cure-driven fundraising initiative for ALS. The Nietos, of Corona del Mar, Calif., became co-chairs of the ALS Division after Augie, 51, learned he had the disease in 2005. In conjunction with MDA, the Nietos established a goal of raising $18 million in three years to fund ALS research at the ALS Therapy Development Institute of Cambridge, Mass., where cutting-edge technologies are applied to ALS drug development.

Through a series of high-profile fundraising events across the country, Augie's Quest met and surpassed its goal. But the Nietos didn't stop there. “When we launched Augie's Quest, I wasn't willing to accept the prospect of not meeting our goal, even though it was a great deal of money,” Nieto said. “Now that we've accomplished what we set out to do, it only makes me more determined than ever to continue forging ahead. The $18 million is only a stepping stone.” (Due to the progression of ALS, Augie relies on assistive devices to help him with breathing, eating, communicating and mobility.)

The Nietos' story and the saga of Augie's Quest continue to capture media attention. This spring the couple made appearances on both the “Today Show” and “Access Hollywood” to tell how their lives have changed since Augie learned he had ALS. The Augie's Quest MySpace page has links at the bottom to both interviews.

Research Supporters Have Many Faces

In 1978, famed University of Southern California linebacker Eric Scoggins was a member of the USC National Championship football team. In 2007, Scoggins learned he had ALS. Soon after, his former teammates and friends came together to support him and his family and raise funds for Eric's Vision, to help support Augie's Quest. The group's first major fundraiser, the Champions of Hope gala, took place on March 14 on the USC campus and generated nearly $150,000 to help with ALS research. Scoggins had planned to attend, but ALS prevented him. He died on Jan. 9.

ALS RESEARCH FINDINGS

FDA Gives Thumbs Up to Iplex Trial

The Food and Drug Administration has announced it will work with biopharmaceutical company Insmed of Richmond, Va., to develop a clinical trial of Iplex for patients with ALS. Iplex is an experimental medication that could help protect nerve cells from damage under some circumstances. Earlier tests of the medication — in a different formulation — failed to show a benefit against ALS. In a related area, Insmed is testing Iplex, with MDA support, in a clinical trial for myotonic dystrophy.

ON OTHER RESEARCH FRONTS

Teams Find Rare Muscle Stem Cell

MDA-supported scientists have isolated a rare type of skeletal-muscle stem cell in mice that may be particularly suited to repairing muscles damaged by diseases such as muscular dystrophies. Teams at the University of Colorado at Boulder and the University of Colorado Health Sciences Center in Aurora conducted the investigation. MDA grantee Bradley Olwin at Boulder coordinated the teams which included MDA grantee Dawn Cornelison, now at the University of Missouri at Columbia.

Olwin and colleagues call the newly identified cells “satellite SP cells.” They say they believe these cells are precursors of special muscle-repair cells called satellite cells that reside near muscle fibers and move in to make repairs as needed. When they injected satellite SP cells into leg muscles in mice lacking dystrophin, the protein missing in patients with Duchenne muscular dystrophy, they saw extensive muscle regeneration and replenishment of dystrophin.

Both Olwin and Cornelison have MDA funding to continue work in this area.

Gene Therapy Trial Results are a Positive First

Other researchers supported by MDA and the National Institutes of Health say results of a gene therapy trial for three people with limb-girdle muscular dystrophy (LGMD) are the first to show promise beyond showing only that they are safe. Although the primary goal of the small trial was to establish the safety of injecting the alpha-sarcoglycan gene into a foot muscle, the investigators also evaluated how long gene activity persisted in the muscle, the level of protein produced by the gene and the response of the immune system to the gene. No adverse effects, such as rejection of the therapy by the immune system, occurred during the trial. Moreover, all three trial participants produced four to five times the amount of alpha-sarcoglycan protein in the gene-injected foot muscle compared to the muscle in their other foot. (In their type of LGMD, alpha-sarcoglycan is deficient.)

Neurologist Jerry Mendell, co-director of the MDA clinic and director of the Center for Gene Therapy at Nationwide Children's Hospital in Columbus, Ohio, received MDA support to coordinate the study team. “This is the first gene therapy trial in muscular dystrophy demonstrating promising findings, setting the stage for moving forward with treatment of this catastrophic group of diseases,” the researchers said.

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