MDA’s Fight Against ALS

Amyotrophic lateral sclerosis, also know as Lou Gehrig’s disease, is a progressive neurodegenerative disease that attacks, and eventually kills the nerve cells and pathways in the brain and the spinal cord resulting in muscle weakness. Simple tasks such as talking, walking and eating become increasingly difficult and then impossible as the body's muscles waste away. Yet, through it all, for the vast majority of people, their minds remain unaffected. ALS occurs throughout the world with no racial, ethnic or socioeconomic boundaries. Currently there is no known cause or cure for ALS.

The Muscular Dystrophy Association’s involvement with ALS began in the early 1950s when Eleanor Gehrig, widow of beloved Yankees first baseman Lou Gehrig, was searching for a way to fight the disease that had taken her husband’s life. Mrs. Gehrig served more than a decade as MDA National Campaign Chairperson. Since inception, MDA has dedicated more than $210 million to ALS research, serves, and informational programs.

Through its ALS Division, MDA is the world leader among voluntary agencies in fighting ALS, offering the most comprehensive range of services of any voluntary hearth agency in the nation. People with ALS are served at 37 MDA/ALS Research and Clinical Centers including the ALS Center at Emory University or any of the some 225 MDA clinics across the county. MDA’s ALS Division also leads the search for a treatment and cure through its aggressive worldwide research program.

In 2007, the largest non-commercial ALS drug development project in history was launched as a joint venture between MDA and the ALS Therapy Development Institute, creating the largest privately funded ALS drug development project in history. This is a three-year, $36 million project aimed at identifying bio chemical targets and drug therapies for ALS.

In 2008 MDA awarded Dr. Michael Benatar, Clinic Director for the MDA Emory Clinic, a $490,000 ALS research grant. The grant will fund two studies of familial ALS which is when there is a family history of the disease.

The pre-ALS study is a prospective longitudinal study of healthy people who are at high risk for developing ALS because they have a family history of the disease and have an identifiable mutation in the SOD1 gene. The goals of pre-fALS are to characterize the pre-symptomatic stages of the disease and to identify risk factors that impact the age at which ALS develops. This study involves annual visits to Emory (paid for by the study) as well as quarterly telephone interviews.

We are also about to start a clinical trial of arimoclomol in people with rapidly progressive forms of ALS due to specific mutations in the SOD1 gene. This study will involve monthly visits, but only two of these will be performed at Emory. For other visits a clinical coordinator will travel to patients’ homes.

In addition to these two studies of familial ALS, we are currently using MRI of the spinal cord, a procedure known as electrical impedance myography (a new painless form of EMG) and skin biopsy (to look at the nerves in the skin) to see whether any of these are useful biomarkers of the disease process.

For more information about MDA/ALS Division please visit www.als-mda.org.