MDA AWARDS $375K GRANT TO COLUMBIA UNIVERSITY RESEARCHER WORMING IN ON THERAPY FOR ALS

TUCSON, Ariz., Aug. 18, 2010 – Oliver Hobert, hopes to land a big fish using the worms he rears in his lab at Columbia University Medical Center.  No, the Howard Hughes Medical Institute investigator at Columbia University Medical Center isn’t planning to bait any fishing hooks with night crawlers.  Instead, he’s conducting genetics experiments with microscopic nematodes (or roundworms), called C. elegans, with an eye to uncovering the causes of amyotrophic lateral sclerosis.  The molecular biologist is taking advantage of the tiny worm’s simple nervous system to help speed discovery of new therapeutics for ALS (known also as Lou Gehrig’s disease), a neurodegenerative disorder that damages motor neurons in the brain and spinal cord, and which is usually fatal within five years of diagnosis. 
Hobert has been awarded $374,511 by the Muscular Dystrophy Association to solve the mystery of a gene called TDP-43, recently identified as a cause of at least some forms of amyotrophic lateral sclerosis.

Hobert explains that researchers don’t understand yet how TDP-43 works.  “It’s a gene that encodes a protein that has something to do with RNA, and that’s all we know.  We don’t know what the normal function of this gene is and we don’t know what’s going wrong if this gene is mutated,” he said.

Because the nematode has a comparatively simple nervous system that provides a convenient experimental system for teasing out how nerves function, Hobert is using them for his research.  In addition, the worm’s short life cycle affords fast and comprehensive genetic analysis that would take years in humans.

“It’s amazing that a tiny worm might help us understand, and possibly treat, the ravages of ALS,” said Stanley H. Appel, M.D., Chairman of the MDA Medical Advisory Committee and himself, an ALS research pioneer.  “Indeed, some of the most significant, Nobel prize-winning biomedical discoveries in recent history have come from research on C. elegans.

“It’s good news that one of the top ‘worm researchers’ in the field has joined the fight against ALS,” added Dr. Appel.  “We have high hopes that his MDA-supported studies will help define the pathway by which TDP-43 mediates motor neuron injury and ultimately lead to effective therapies.”

Hobert is one of 38 research leaders who’ve been awarded grants from this new round of MDA grant funding, totaling just over $14 million.  MDA annually invests almost $39 million in neuromuscular disease research projects around the world.  Since 2001, Hobert and his lab have received $924,788 in MDA research grants. To learn more about Hobert and his research listen to his podcast.

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 towards 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, 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 Americans are affected by neuromuscular diseases, and an estimated 35,000 are living with ALS.  New York residents affected by any of the more than 40 muscle diseases covered by MDA’s program can receive excellent medical care at a dozen MDA Clinics statewide; and those affected by ALS are encouraged to visit The Eleanor and Lou Gehrig MDA/ALS Research Center at Columbia University Medical Center; The MDA/ALS Center at SUNY Upstate Medical University in Syracuse; the MDA/ALS Center at the University of Rochester Medical Center and St. Peter’s ALS Regional Center in Albany.

MDA is the nonprofit health agency dedicated to curing muscular dystrophy, ALS and related diseases by funding worldwide research (see outstanding B-roll 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: http://www.mda.org/

For more information about these new grants, visit MDA's "Grants at a Glance" http://www.mda.org/research/gaag/2010, an online slideshow that showcases each grant with photos and detailed information.