MDA AWARDS $375K TO ARIZONA RESEARCHER SEEKING ALS THERAPIES

TUCSON, Ariz., Aug. 18, 2010 – All it took was one meeting and one inspiring story for Dr. Daniela Zarnescu to know she was in the right place and right profession -- doing research on amyotrophic lateral sclerosis, better known as ALS or Lou Gehrig’s disease.

While at the University of Arizona’s ALS clinic – one funded in part by the Muscular Dystrophy Association – Dr. Zarnescu was invited by clinic director Dr. Katalin Scherer to meet some patients.

“It was heartbreaking to see a smart and talented neurologist (Dr. Scherer), able only to offer her professionalism and kindness to an endearing and accepting old man surrounded by his equally powerless, loving sons,’’ Dr. Zarnescu said. “Getting to know that patient provides a strong motivation to work on ALS.’’

Dr. Zarnescu is now advancing important ALS research with an MDA grant for $375,000.  She is one of 38 research leaders now receiving multi-year 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.

“In this funding climate, it is critical,’’ she said of the award money. “The availability of funding from the funding agencies such as MDA is the equivalent of ‘life support’ for biomedical research in this country.’’
To learn more about Dr. Zarnescu and her current project, listen to or read transcript from her recent MDA Q&A podcast.

Dr. Zarnescu said she will work with the fruit fly model (Drosophila) because it “has multiple excellent features that can be exploited for rapid discovery of novel genes and potential therapeutic approaches for ALS.’’

She added that while the genetic causes of ALS are just beginning to be discovered, the pathological features of the disorder contain the common denominator, TDP-43. Dr. Zarnescu hypothesizes that the defects due to altered TDP-43 protein “can be corrected by genetic (therapies) and pharmacological intervention and that those genes and compounds that rescue the TDP-43 phenotypes may provide novel therapeutic approaches to ALS.’’

“Dr. Zarnescu is a talented young geneticist, having already made her marks in the fields of fragile X disease and breast cancer research,” said Stanley H. Appel, M.D., chairman of the MDA Medical Advisory Committee and himself an ALS research pioneer.  “We are thrilled that, through this new MDA research grant, Dr. Zarnescu will use genetic approaches to determine how TDP-43 can be corrected to better select potential therapies for ALS.”

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, which has invested almost $39 million in 2010 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 some 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 one million people in America are affected by neuromuscular diseases.
Residents of the Tucson area who are affected by any of the 43 muscle diseases covered by MDA’s program can receive excellent medical care at The Mucio F. Delgado Clinic and Children’s Clinics for Rehabilitative Services.

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.  The Association also provides comprehensive health care and support services, advocacy and education.  For more information, visit 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.