Closer to an ALS cure
Date: May 3, 2012
Scientists have been studying patients with the neurodegenerative disease known as amyotrophic lateral sclerosis, or ALS, since 1824. The disease, which affects three out of every 100,000 people worldwide, gained some needed publicity when baseball player Lou Gehrig went public with his own diagnosis more than a century later. But despite this long history, says Johns Hopkins neurologist Nicholas J. Maragakis, researchers still don’t have a good understanding of what causes the disease, and how and why symptoms progress—information that’s pivotal to developing a cure.
Recently, Maragakis and other Hopkins researchers received a transformative gift that could turn the tide for ALS: $25 million in funding that will enable Johns Hopkins to establish a new center to develop novel therapies.
“We are now able to think out of the box about this disease,” says Maragakis. “The goals of the 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, formally known as the Michael S. and Karen G. Ansari ALS Center for Cell Therapy and Regeneration Research at Johns Hopkins, 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 support a variety of efforts that aim to eventually cure ALS. The disease targets motor neurons, a type of nerve cell that controls muscle movement.
“When my wife was diagnosed with ALS, a single question formed in my mind: How can we overcome this illness?” says Michael Ansari. “The Ansari Center is a single purpose center: to stop the progress of ALS, reverse it, regenerate the nerves and enable patients to regain their strength.”
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.
“For some time now, I have concluded that there exists a path to the solution for every challenge mankind faces—moreover, mankind can discover that path and bring about that solution,” Ansari says. “I am absolutely convinced that the right team of experts who are resolute in their conviction articulated in their mission, with sufficient resources and time, will discover the path and will overcome any challenge, including ALS. It is with this conviction that the Ansari Center has been created.”
Other Johns Hopkins University School of Medicine researchers who will serve as principal investigators at the new center include Jeffrey Rothstein, 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, professor of neurology and neuroscience and director of the Neuromuscular Division; and Charlotte Sumner, 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.
“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 McArthur, professor of medicine, 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.”
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