JOHNS HOPKINS NEUROSCIENTIST PURSUES ALS WITH NEW $348K MDA GRANT
TUCSON, Ariz., Aug. 18, 2010 — Johns Hopkins University researcher Shanthini Sockanathan, Ph.D., has been awarded $347,842 by the Muscular Dystrophy Association to study why motor neurons in the brain or nervous system that are healthy at birth begin to die off, causing neuromuscular diseases such as ALS (amyotrophic lateral sclerosis, or Lou Gehrig’s disease). Motor neurons control muscle activities such as walking, breathing and swallowing. When ALS strikes, motor neurons are ravaged, progressive paralysis of voluntary muscles ensues and people generally succumb to the disease within five years of diagnosis.
Sockanathan, an associate professor of neuroscience at the Solomon H. Snyder Department of Neuroscience at Johns Hopkins, already has obtained preliminary results that implicate a developmentally regulated gene in motor neuron survival, and her efforts could yield valuable insights for a variety of other neurodegenerative disorders, including spinal muscular atrophy (SMA), and Alzheimer's, Huntington’s and Parkinson’s disease, as well as fragile X, a genetic developmental disorder.
“My research is focused on understanding pathways that correct motor neuron development and survival so we can understand how motor neurons work, how they are generated and how they die. With this knowledge, we can understand what goes wrong with them in the diseased state. Use this simple analogy: If your car breaks down, it’s difficult to repair it if you don’t understand how it works in the first place and that’s how I view our work,” she said.
Sockanathan is one of 38 research leaders receiving new multiyear awards from MDA. Her three-year project is part of the $14.1 million in new research funding approved during the Association’s July board meeting. Including this grant, which is her third from MDA, Sockanathan has been awarded close to $1 million in MDA funding since 2001. MDA is the world’s leading nongovernmental funder of ALS research. To learn more about Sockanathan and her research, listen to her podcast or read her transcript.
Sockanathan credits MDA for encouraging her, as a young scientist, to pursue research into new areas. “Most of my work is on basic research of motor neuron development, and I am really grateful that MDA has been so forward-thinking in trying to fund this sort of research.”
“Dr. Sockanathan’s discovery of the crucial role of specific factors in motor neuron survival offers an entirely novel line of investigation on what causes the neurodegeneration that is the hallmark of ALS,” said Stanley H. Appel, M.D., chairman of the MDA Medical Advisory Committee and an ALS research pioneer.
“Her MDA-funded project will not only shine light on the molecular underpinnings of motor neuron degeneration, but the research tools her team is generating will become valuable tools for the entire community of ALS scientists.”
Research grant applications are peer-reviewed twice yearly by MDA’s Medical and Scientific Advisory Committees, comprising world-renowned experts in neuromuscular disease research. The most promising of some 500 applications received each year are recommended for funding to the MDA Board of Directors.
According to R. Rodney Howell, M.D., chairman of the MDA Board of Directors, “Generous Americans responding to the annual Jerry Lewis MDA Telethon, and to thousands of other special events benefiting the families served by MDA, deserve much of the credit for the rapid progress being made toward treatments for neuromuscular diseases. It’s their strong belief in MDA’s capable stewardship of public funds that’s enabling so much to be accomplished — even in a sluggish economy.”
Often credited for its leadership in building the field of neuromuscular disease research, MDA has simultaneously enhanced clinical care for individuals affected by muscle disorders, achieving important quality of life and longevity gains. The Association, which has invested almost $39 million in 2010 in research worldwide, is the first nonprofit to earn a Lifetime Achievement Award from the American Medical Association (“for significant and lasting contributions to the health and welfare of humanity”).
MDA-funded scientists have uncovered the genetic defects that cause several forms of muscular dystrophy; Charcot-Marie-Tooth disease (CMT); a form of amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease); childhood spinal muscular atrophy (SMA) and other neuromuscular conditions. Now entering a period of increasing numbers of clinical trials of potential therapeutics, the Association’s network of approximately 200 hospital-affiliated clinics is instrumental in identifying appropriate candidates for clinical trials, and in helping to refine outcome measures for those clinical trials.
More than 1 million people in America are affected by neuromuscular diseases.
In the greater Baltimore area, people affected by ALS can seek treatment and support at the MDA/ALS Center at Johns Hopkins University, co-directed by Daniel Drachman, M.D. and Jeffrey Rothstein, M.D. MDA also sponsors a clinic covering the more than 40 other neuromuscular diseases at the Johns Hopkins University Outpatient Center.
MDA is the nonprofit health agency dedicated to curing muscular dystrophy, ALS and related diseases by funding worldwide research, view video of MDA research at http://www.mda.org/research/gaag/2010/b-roll.html
The Association also provides comprehensive health care and support services, advocacy and education. For more information on MDA research and programs, go to www.mda.org.
For more information about these new grants, visit MDA's "Grants at a Glance" at an online slideshow that showcases each grant with photos and detailed information. |
