MDA AWARDS $180K TO THOMAS JEFFERSON RESEARCHER STUDYING LOU GEHRIG’S DISEASE

TUCSON, Ariz., Aug. 18, 2010 — Dena Jacob, Ph.D., is a post-doctoral investigator being mentored by Dr. Davide Trotti at Thomas Jefferson University.  Having identified Jacob as an outstanding young researcher, the Muscular Dystrophy Association board of directors has approved $180,000 in a Development Grant for her to research amyotrophic lateral sclerosis (ALS), more commonly known as Lou Gehrig’s disease. 

“Amyotrophic lateral sclerosis is the most common adult motor neuron disease, affecting two to three adults per 100,000 individuals annually," said Jacob from Thomas Jefferson University. “The disease is marked by the selective degeneration of motor neurons in the spinal cord. Although there have been a number of drug trials to cure the mouse that models ALS, these attempts have so far failed."  

Dena Jacob, Ph.D.

Jacob said she may have an explanation for so many failed trials. A protein called P-glycoprotein (P-gp). P-gp works to pump drugs out of cells, Jacob notes. Moreover, she has shown there is more P-gp in spinal cords in ALS mice. She contends that P-gp may contribute to ALS drug resistance and her newly awarded MDA project will test that hypothesis.

“If successful," she said, “these experiments will provoke a re-examination of many previous unsuccessful clinical drug trials in the ALS mouse. They may also encourage the development of new, combined therapeutic strategies for ALS treatments."

Jacob, 33, is one of 38 research leaders now receiving 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. This is her first MDA grant.

“MDA’s leadership as a funding source is invaluable for advancing our understanding of neuromuscular diseases," Jacob said.

“In fact, our laboratory has been fortunate enough to receive funding twice in the past, allowing us to generate compelling data that has spurred this current, exciting line of research. So we’re very grateful to have the support of such an important organization." 

Research grant applications are peer-reviewed twice yearly by MDA’s Medical and Scientific Advisory Committees, comprised of 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.”

“Dr. Jacob and her team are asking a question central to all drug therapy approaches for ALS,” said Stanley H. Appel, M.D., chairman of the MDA Medical Advisory Committee and ALS research pioneer. “No matter how powerful a drug is at hitting the target inside the cell, might the cell be removing the drug before it can even get to its target?

“If that is the case,” explained Appel, “then we must be ready to launch counter measures against these cellular defenses. The research Dr. Jacob is doing with MDA funding could represent that first pre-emptive strike.”

Often credited for its leadership in building the field of neuromuscular disease research, MDA also has 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 to help refine outcome measures for those clinical trials.

More than 1 million people in America are affected by neuromuscular diseases. 

Residents of the Philadelphia area who are affected by any of the more than 40 muscle diseases in MDA’s program can receive excellent medical care at: The Children’s Hospital of Philadelphia; the Hospital of the University of Pennsylvania; and Drexel University College of Medicine. Individuals living with ALS are encouraged to visit the MDA/ALS Center of Hope at Drexel University College of Medicine.

MDA is the nonprofit health agency dedicated to curing muscular dystrophy, ALS and related diseases by funding worldwide research. 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," an online slideshow that showcases each grant with photos and detailed information.