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Hopkins-sponsored research on how to stop ALS, repair its damage or even prevent it altogether ranks among the world’s best because it’s carried out under a unique blueprint that’s taken shape as The Robert Packard Center for ALS Research at Johns Hopkins. The Packard Center is the only institution of its kind dedicated solely to the disease.

Over 100 scientists from Hopkins, other universities, government labs and biotech companies work together, sharing insights and materials to understand the biology of ALS and find a cure.

Housed at Hopkins’ East Baltimore campus, the Center is supported largely through private philanthropic funding, as well as more traditional grant support from the National Institutes of Health. It began as the brainchild of the Neurology department’s ALS researchers, especially the Center’s present director, Jeffrey Rothstein. They met with a small group of patients and philanthropists as well as forward-thinking Neurology administrators, to open the Packard Center in 2000.

By emphasizing teamwork and sharing—with no need to wait until lab results are published—by requiring progress, meeting milestones and inviting open collaboration, and also by carefully selecting the most promising areas of research and top scientists in those areas, the Packard Center has advanced the field dramatically.

Its scientists have helped uncover new genes for familial ALS. They’re actively working on genetic risk factors—reasons why patients with sporadic ALS may be susceptible to the disease. They’ve developed a necessary variety of animal models for testing therapies and exploring ALS biology. Also, they’ve shed light on nerve repair, uncovered natural, built-in nerve protective systems, advanced stem cell therapy and are studying natural ways to counter muscle atrophy. Concerned about more immediate help for patients, Center scientists have screened potential new drugs and are ushering them down the testing pipeline. The first five years have seen more than a dozen clinical trials for candidate drugs or other therapies.

Finally, Packard Center scientists have become world-respected in increasing understanding of what ALS does to cells. Both that knowledge, along with the Center’s drug discovery program, extend insight and treatment possibilities for other neurodegenerative diseases including Parkinson’s disease, Alzheimer’s disease, spinocerebellar ataxia, Huntington’s disease, multiple sclerosis and the peripheral neuropathies.