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MDA NEWS

Lou Gehrig

Making the Nation Aware of ALS

For the 17th year, America will observe national ALS Awareness Month in May. To heighten public awareness of this devastating disease, also known as Lou Gehrig's disease, MDA will conduct a multitude of events and programs across the country.

ALS (amyotrophic lateral sclerosis) strikes healthy adults in the prime of life, killing motor neurons (the nerve cells that control muscles) and gradually causing total paralysis of all voluntary muscles. Death often occurs within three to five years of diagnosis, although assistive technology like ventilation and feeding tubes are helping some people survive longer.

Ruth Johnson

ALS: 'Anyone's Life Story'

Ruth Johnson raised four children, then went on to become an elementary schoolteacher and principal. Ramon Antelo operated an auto parts business for 30 years, and loved to hunt and fish. Steve Courington worked 60 to 80 hours a week to make a good life for his wife and two young daughters.

Ramon Antelo

Each of these people, from different walks of life, developed ALS. Throughout May, learn how their lives, and those of 28 others with ALS, have been altered forever by this paralyzing disease, which steals the strength while leaving the mind and spirit untouched. Each day in May, visit the MDA ALS Division Web site to read a new profile in courage and character, in a 31-day series called "Anyone's Life Story."

Steven Courington

As you read these very personal, very human stories, know that MDA-funded researchers are working tirelessly on many different fronts to find treatments and cures for ALS (amyotrophic lateral sclerosis).

Events Around the Country

Also during May, look for MDA public service announcements about ALS in the news media. MDA offices nationwide will host special events and educational seminars for the public. Here are a few examples:

• May 2, Denver Zoo: "An Afternoon with the Experts" symposium discusses current ALS research and clinical trials.
• May 3, Sheraton Gateway Hotel Atlanta Airport: A guest speaker from the ALS Therapy Development Institute of Cambridge, Mass., will discuss new trends in ALS research.
• May 9, El Paso (Texas) Diablos' Cohen Stadium: "Fire Fighter Appreciation, ALS Awareness Night" will include a post-game showing of Lou Gehrig's story in "Pride of the Yankees."
• May 18, Arthur Fiedler Field, Boston: A three-mile ALS Walk is planned.

New ALS Publications

MDA will release two ALS-specific publications and an emergency health information organizer in May.

In addition to a newly updated version of the booklet, "Facts About ALS," MDA also is releasing a new publication, the MDA ALS Caregiver's Guide. This 210-page spiral-bound book contains reams of practical information to help caregivers of people with ALS cope with problems in such areas as nutrition, breathing, communication, finances, insurance, accessibility and much more. It also features dozens of quotes from ALS caregivers and people with ALS.

The caregiver guide is a supplement to MDA's 2005 publication Everyday Life with ALS, which is geared toward the person with ALS.

MDA's new emergency health information envelope keeps important documents and information posted on the refrigerator, close at hand for 911 calls or emergency hospital trips.

These items are available at no cost to families living with ALS who are registered with MDA, at local MDA offices.

Augie's Quest is Going for the Gold!

A little more than two years ago, Augie and Lynne Nieto, co-chairs of MDA's ALS Division, set a goal of raising $18 million in 36 months for ALS research through MDA's Augie's Quest ALS research initiative.

Augie & Lynne Nieto

As of early April, the tally had reached more than $14 million. With a series of fundraising events scheduled this spring, plus the help of individual donations, Augie and Lynne are hopeful they'll reach the $18 million mark.

Health Clubs
Join the Fight

Augie Nieto was a pioneer in the fitness industry, so it's logical that health clubs across the country would throw their support behind Augie's Quest.

Clubs for the Cure.com was created by health club owners to involve their peers in the fundraising effort. Each club is encouraged to hold at least one event in which their members will participate in activities like bicycle "spins" to garner pledges.

For more information about how you or your health club can get involved, contact Jess Hayes at jessicah@theatlanticclub.com or (732) 450-4412.

RESEARCH NEWS

ALS Research Findings

MDA supports first U.S. trial of lithium in ALS

An MDA-supported clinical trial of lithium carbonate, a medication commonly used to treat bipolar disorder, is set to begin in people with ALS, following reports that the drug may dramatically slow the progression of this devastating disease.

MDA's clinical trial is an effort to confirm or refute the findings of a recent Italian trial conducted by Francesco Fornai at the University of Pisa (Italy) and colleagues at other Italian institutions. The Italian trial showed that lithium significantly delayed ALS progression compared to standard treatment alone.

The 12-month MDA-supported trial will enroll 100 participants at 10 sites around the United States. Principal investigator is neurologist Robert Miller, director of the Forbes Norris MDA/ALS Research Center at California Pacific Medical Center in San Francisco.

"The results from the Italian study were the most important and positive ever reported for ALS," Miller said. "We really need to know that it's real, and this study should give us that answer.

Mutated gene found in some with ALS

In a study coordinated by MDA grantee Guy Rouleau and colleagues at the University of Montreal, mutations in the gene for a protein called TDP-43 were found in some people who had either the inherited or spontaneous forms of ALS. No TDP-43 mutations were found in people without ALS.

The findings supplement similar observations made by other researchers and support the need for continued research in this area.

Blood-spinal cord barrier breaks early in ALS

Leaks in the blood vessels of the spinal cord appear to precede damage to the nervous system in mice with a form of ALS, reports a research team that included MDA grantees M. Kerry O'Banion at the University of Rochester (N.Y.) Medical Center and Severine Boillee at the University of California-San Diego.

This finding, published in the April issue of Nature Neuroscience, provides promising clues for investigators working on treatments for this paralyzing disorder.

The barrier between the spinal cord and the bloodstream normally protects the central nervous system from toxins and injury. However, in mice with mutated SOD1 genes, the barrier became "leaky" due to reduced levels of three proteins that normally keep it together, the investigators say.

This damage to spinal cord blood vessels seems to be among the earliest events in a "toxic cascade" set in motion by SOD1 gene mutations.

The leaks resulted in inflammation, as well as reductions in blood flow and, in some areas, small hemorrhages with release of toxic products, the researchers say. They believe this contributes to the degeneration of motor neurons, which in turn causes paralysis.

Interventions targeted specifically to the endothelium (lining of blood vessels) will help gauge the link between leaky blood vessels and the death of motor neurons, and also assess how such damage affects disease onset and/or progression, the researchers note. The team currently is testing a compound that may help seal leaky blood vessels.

On Other Research Fronts

Special report on DMD and BMD

A new one-time publication, the "Duchenne-Becker MDA Special Report," describes exciting current MDA-sponsored research into treatments for these two forms of muscular dystrophy.

Coinciding with the 21st anniversary of the discovery of the flawed gene that causes Duchenne and Becker muscular dystrophies, the 12-page report was mailed to all MDA-registered families affected by DMD and BMD.

For a look at the history of DMD research, see "Milestones in Duchenne Muscular Dystrophy Research."

MDA grantee receives prestigious award

Paul Gregorevic, a longtime MDA research grantee, has received a $1 million award from Pfizer Australia to develop gene-based therapies for muscle disease.

Paul Gregorevic

Gregorevic recently moved from the University of Washington-Seattle to the Baker Heart Research Institute in Australia. From 2004 to 2007 he received MDA funding to develop gene-delivery methods for muscular dystrophy. In January, he began receiving MDA support to explore the effects of follistatin on mice with muscular dystrophy.

The 2008 Pfizer Australia Research Fellowship recognizes excellence in biomedical researchers who will contribute to that country's standing in scientific fields.

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