Milestones in ALS Research

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MDA’s ALS Research Mission

ALS (amyotrophic lateral sclerosis, or Lou Gehrig’s disease) is a multisystem neurodegenerative disorder that primarily affects motor neurons, the nerve cells that control voluntary muscle movement. The loss of motor neurons causes the muscles they control to become weak and then paralyzed. Death, which can occur as early as three to five years after diagnosis, usually is due to respiratory complications caused by paralysis of the muscles used in breathing.

MDA is the world leader in funding ALS research. Its worldwide program supports research efforts ranging from basic (early-stage) science, to preclinical testing and therapy development, to human clinical trials. Since its inception, MDA has dedicated $324 million to ALS research, services, education and advocacy programs.

Current MDA-supported ALS research is focusing on several areas, including:

• ALS-associated genes SOD1, TDP43, FUS, UBQLN2 and C9ORF72;
• potentially toxic protein clumps called aggregates;
• oxidative stress
• facts that cause support cells to attack motor neurons instead of nourishing and protecting them;
• disruption of the cellular waste-disposal system
• the role of the immune system in ALS;
• ways to nourish and protect motor neurons;
• mitochondrial energy production;
• "antisense" therapy designed to block toxic protein production;
• stem cell therapy; and
• drug discovery and development.

Other critical MDA-funded ALS research contributions include:

• maintenance of an MDA ALS Clinical Research Network to streamline and support tests of experimental treatments, with locations at five centers: Methodist Neurological Institute (Houston), Massachusetts General Hospital (Boston), Columbia University (New York City), Emory University (Atlanta), and California Pacific Medical Center (San Francisco);
• sponsorship and hosting of national and international scientific meetings on ALS research;
• support of the MDA Neuromuscular Disease Registry, which aims to expedite clinical trtials, and improve survival and quality of life in ALS and other diseases; and
• advocacy efforts focused on speeding up the regulatory pathway to approval for experimental ALS therapies.

As you will see in this report, ALS has so far resisted the best efforts of decades of dedicated researchers. But the disease is slowly giving up its secrets. MDA continues to be inspired by the words of the late Michael E. DeBakey, world-renowned heart surgeon and an MDA national vice president, who said, “There are no incurable diseases. There are only diseases for which no treatment has yet been developed.”

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