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MDA NEWS

Meet MDA's New "Celebrity Ambassador"

Ace Young

Singer, Grammy-nominated songwriter, actor and American Idol finalist Ace Young is MDA's new Celebrity Ambassador.

Young, 27, will make appearances on stage and at celebrity venues around the country to spread the word about MDA's battle against neuromuscular diseases. A native of Denver, he's a longtime supporter of children's causes, including Children's Hospital of Denver, site of one of MDA's 225 clinics.

MDA National Chairman Jerry Lewis said he's been impressed by Young's performing talents, and now his offer to help represent MDA.

"Ace certainly lives up to his name — he's an outstanding entertainer and humanitarian," Lewis said.

Young was nominated for Best Rock Song in the 50th Grammy Awards, currently has an album in production with Virgin Records, and in 2006 was named one of People Magazine's Hottest Bachelors.

New MDA ALS Center is Michigan's First

Michigan now has its first MDA center devoted to research and treatment of ALS. The Michigan State University MDA/ALS Center — MDA's 38th in the country — joins the Association's eight other medical clinics in Michigan.

Although all MDA clinics serve people with ALS and any of the other 40-plus diseases in the Association's program, an ALS center is notable for the ALS research undertaken there, plus the greater experience of its medical staff in dealing with the disease.

In a unique move designed to provide greater access to individuals and families, the MSU center will have two clinical sites: Mary Free Bed Rehabilitation Hospital/Saint Mary's Health Care in Grand Rapids, and MSU's Clinical Center in East Lansing.

MDA's New Orleans Office Reopens

Even after hurricanes Katrina and Rita devastated New Orleans and severely damaged MDA offices, MDA staff and doctors continued to serve the more than 700 families registered with the Association in the New Orleans area, operating from borrowed quarters.

Now, with the offices recently repaired and renovated, the MDA team again has a place to call home. The address is 4401 N. I-10 Service Rd., Suite 104, Metairie, La. 70006. Telephone: (504) 455-4460.

Best-Selling Book Profiles
Former MDA Ambassador

Best-selling author Richard M. Cohen has another book on the New York Times bestseller list. This one examines the lives of five people with chronic disabilities.

Ben Cumbo

Strong at the Broken Places: Voices of Illness, A Chorus of Hope includes a chapter about Ben Cumbo, 20, who was MDA's National Goodwill Ambassador in 1996 and 1997. Cohen spent two years researching the book and visiting frequently with Cumbo and his family. His writing delves deeply into Cumbo's frustrations, aspirations, fears and hopes.

Also profiled is Denise Glass of Thousand Oaks, Calif., who has ALS. Like Cumbo, Glass is registered with MDA. The book's other subjects include a woman with Crohn's disease, a man with non-Hodgkin's lymphoma and a man with bipolar disorder. The book is available from Harper-Collins Publishers.

Cohen has established a Web site, www.strongatthebrokenplaces.com, where others with disabilities can share their stories.

Mattie's Poetry Still Imparts Its Magic

Mattie Stepanek

The poetry of the late Mattie Stepanek, former MDA National Goodwill Ambassador, continues to earn accolades.

The Vietnam Literature Association has presented its top award to a Vietnamese translation of Mattie's Heartsongs poems. It's the first time a translation of children's poetry has received the award.

Mattie's poetry, prose and aspirations as a peacemaker received worldwide recognition, both during the last years of his young life, and after his death in 2004 at age 13, from a rare disease related to muscular dystrophy.

Celebrating Duchenne Research Milestone

A March 8 celebration at Children's National Medical Center in Washington, D.C., recognized pioneers in Duchenne muscular dystrophy (DMD) research.

Honorees Louis Kunkel, Eric Hoffman, Michel Koenig and Anthony Monaco were honored for their 1987 discovery of the dystrophin gene that, when flawed, causes DMD.

DMD scientists, families and other members of the DMD community joined MDA staff for the historical commemoration.

National Recognition for Stanley Appel

Neurologist Stanley H. Appel has received the prestigious John P. McGovern Compleat Physician Award for 2008 from the Houston Academy of Medicine.

Appel is chairman of MDA's Medical Advisory Committee, director of the MDA/ALS Center at Methodist Neurological Institute in Houston and a longtime MDA research grantee and Board member. He specializes in ALS (amyotrophic lateral sclerosis) research.

The national award recognizes a physician who embodies exemplary service to humanity and whose career reflects medical excellence, humane and ethical care and commitment to the medical humanities.

RESEARCH NEWS

Dystrophin Gene Transfer Appears Safe

In trials that began two years ago, MDA-supported researchers have sought to provide normal dystrophin genes to boys with Duchenne muscular dystrophy (DMD), who have flawed dystrophin genes and therefore lack the muscle protein dystrophin.

Now, in a trial to transfer a dystrophin gene compound (Biostrophin) to six boys with DMD, the procedure has been shown to be safe and well tolerated.

Researchers next plan to test three additional boys with a higher dosage of the compound, which is injected into an arm muscle.

Results of the trial with all nine boys will form the basis for future gene therapy trials.

MDA Awards $1 Million Grant for
Drug Development

MDA has awarded a $1 million grant to Repligen, a Massachusetts-based biopharmaceutical company, to develop compounds known as HDAC inhibitors, or transcription activators, to treat Friedreich's ataxia and type 1 myotonic dystrophy.

HDAC inhibitors coax cells to make proteins by allowing genetic instructions that a cell normally would read as "closed" to be read as "open."

Searching Outside Nerve Cells for ALS Clues

The possible role of astrocytes (a type of nervous system "support" cells) in ALS has been the focus of researchers in the laboratory of Don Cleveland at the University of California-San Diego. The team includes MDA grantee Severine Boillee.

The group found that diminished production of a toxic protein (abnormal SOD1) in astrocytes dramatically slowed disease progression in ALS-affected mice, even when SOD1 production was high in nerve cells that control muscles.

When production of abnormal SOD1 in astrocytes was high, the disease progressed more rapidly.

The researchers' findings add to several recent studies that contrast with long-held beliefs that nerve cells (which die and cause paralysis in ALS) are the only problem in the disease. Looking beyond those cells clearly is important.

Lithium Shows Value in ALS Study

A small Italian study of 44 people with ALS indicates that daily doses of lithium may delay progression of disease symptoms such as muscle wasting and paralysis.

In a 15-month controlled trial that began in October 2005, two groups of patients with ALS were given either riluzole (a drug shown to have modest benefit in increasing lifespan in ALS), or riluzole plus lithium, a drug used to treat people with bipolar disorder. By the end of the study, 30 percent of the riluzole-only patients had died, and the disease had progressed markedly in the survivors. However, all those who received the riluzole-lithium combination survived, and showed fewer signs of disease progression.

To date, no other ALS treatment has produced such dramatic positive results.

Valerie Cwik

Lithium may work by increasing autophagy, a process in which worn-out or abnormal cell components are destroyed, while the number of mitochondria (energy-producing components of cells) is increased.

Lithium can be dangerous and must be taken under a doctor's supervision, with frequent monitoring of its presence in the blood.

"Although the number of study participants is small, the results are very intriguing," said Dr. Valerie Cwik, MDA's medical director and vice president of research. "MDA already has plans under way to follow up on these results with a larger, confirmatory study."

New MMD Mouse Adds to
Understanding of Disease

In research, progress is often tied to the accuracy of the "mouse model" available to study the disease. Now a new mouse has been bred that will help scientists better study type 1 myotonic dystrophy (MMD1), as well as how it differs from type 2 myotonic dystrophy (MMD2).

The advance, by MDA grantee Thomas Cooper at Baylor College of Medicine with colleagues there and in France, ultimately could lead to new treatment approaches.

Previous mouse models for MMD1 exhibited some of the features and molecular events seen in human MMD1, but not all. Cooper and colleagues say their new model is the only one to mimic the muscle wasting that patients have and to show elevated levels of a protein called CUGBP1 in muscle cells, another characteristic of human MMD1.

They note that people with MMD2 don't have high levels of CUGBP1 and typically have milder muscle wasting than people with MMD1.

"Muscle atrophy is the primary cause of disability and death in individuals with MMD1," Cooper said. "Having an animal model that reproduces this aspect of the disease provides an important tool to understand the process and to test therapies."

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