Hopkins Launches New ALS Research Center with a $25 Million Gift
Nicholas Maragakis is a Packard-funded investigator and a member of the Packard Center's Operating Committee.
A $25 million gift has enabled Johns Hopkins to establish a new center to develop novel therapies for the neurodegenerative disease known as amyotrophic lateral sclerosis, Lou Gehrig’s disease, or ALS. Much of the center’s research will focus on using stem cells individually derived from ALS patients to develop new model systems to investigate how nerve cells degenerate, as tools to screen new drug therapies, and to develop stem cell therapies as transplants to potentially slow or reverse the disease.
The new center, formally known as the Michael S. and Karen G. Ansari ALS Center for Cell Therapy and Regeneration Research at Johns Hopkins and dedicated March 21, 2012, is named for its benefactors: Michael and Karen Ansari. Michael Ansari is the founder, chairman and CEO of M.I.C. Industries.
The gift, representing a five-year commitment, will fund a variety of efforts that aim to eventually cure ALS. The disease targets motor neurons, a type of nerve cell that controls muscle movement, and affects about three out of every 100,000 individuals.
“Despite knowing about this disease for decades and the large number of clinical trials that have been completed, we still have little in our arsenal to treat it,” says Nicholas J. Maragakis, M.D., an associate professor of neurology at the Johns Hopkins University School of Medicine, co-medical director of the ALS Clinic and director of the new center. “We are now able to think out of the box about this disease. The goals of a center will focus on the use of stem cells as tools to foster aggressive programs in discovering the underlying mechanisms behind what causes ALS and rapidly translating these discoveries to the patients in our clinic.”
The new center will test a number of different approaches against ALS with emphases on neuroprotection and regeneration. These include guiding stem cells produced directly from ALS patients and animal models to develop into specific neural cell subtypes to study their vulnerability. The research will also compare stem cells from patients with hereditary ALS to those with the more common sporadic disease (that doesn’t run in families) to understand how these forms differ; dose stem cells from ALS patients with environmental toxins to see how these influence cell survival; use stem cells from ALS patients or animal models to screen drugs that may work to treat ALS; and ultimately use stem cells to understand how to enhance nerve cell regeneration.
Other Johns Hopkins University School of Medicine researchers who will serve as principle investigators at the new center include Jeffrey Rothstein, M.D., Ph.D., professor of neurology and neuroscience, director of the Robert Packard Center for ALS Research at Johns Hopkins and co-director of the MDA/ALS Clinic; Ahmet Hoke, M.D., Ph.D., professor of neurology and neuroscience and director of the Neuromuscular Division; and Charlotte Sumner, M.D., associate professor of neurology. In addition to the efforts in the labs of these investigators, the group will embark on new collaborations with other researchers within the Hopkins community, including the Institute for Cell Engineering, Brain Science Institute, and Packard Center for ALS Research. Projects beyond the Hopkins community with nationally and internationally recognized institutions are also in progress.
“When my wife was diagnosed with ALS, a single question formed in my mind: How can we overcome this illness?” posed Michael Ansari. “The Ansari Center is a single purpose center: to stop the progress of ALS, reverse it, regenerate nerves, and enable patients to regain their strength.”
“This extraordinary gift represents a game-changer in ALS, bringing together in the Ansari Center at Hopkins four renowned physician-scientists whose sole focus is on finding a substantive treatment for ALS,” says Justin C. McArthur, M.B.B.S., M.P.H., F.A.A.N, professor of neurology, pathology and epidemiology and director of neurology at the Johns Hopkins University School of Medicine. “This gift allows us the flexibility to explore options in understanding and attacking ALS that we wouldn’t be able to pursue any other way. Our goal is nothing less than overcoming ALS.”