MDA AWARDS $360K TO TEL AVIV SCIENTIST WORKING TO IMPROVE DELIVERY OF GROWTH FACTORS AS POTENTIAL ALS TREATMENT

TUCSON, Ariz., Aug. 18, 2010 — In recent years, several biological agents called neurotrophic growth factors have proved beneficial in rodent models for ALS (amyotrophic lateral sclerosis, or Lou Gehrig’s disease).  However, none of the growth factors has measured up to expectations in people, possibly because they are rapidly degraded in the body before they can provide their therapeutic benefit.

Through a new $359,700 Muscular Dystrophy Association grant, Daniel Offen, Ph.D., at Tel-Aviv University in Israel, is now investigating the potential of a better delivery system for administering these beneficial growth factors.  The three-year study will test whether transplanted muscle progenitor cells (stem cells) that have been engineered to produce various neurotrophic factors can better slow progression of ALS. 

“This study may lead to a novel combined autologous cell and gene therapy approach for the alleviation of symptoms of ALS and the extension of lives for people living with this devastating neuromuscular disease, "Offen, head of the Neuroscience Laboratory, Felsenstein Medical Research Center at Rabin Medical Center, said.

Daniel Offen, Ph.D.

“A major hurdle in ALS therapy development has been to deliver therapeutic agents, in the right amounts and proper combinations, to the parts of the body where they are needed,’’ explained Stanley H. Appel, M.D., chairman of the MDA Medical Advisory Committee, and a pioneer in the field of ALS research. “Dr. Offen’s careful studies, using the body’s own stem cells to deliver combinations of growth factors to the tissues, should go a long way toward overcoming this hurdle."

Offen’s three-year study is one of 38 new MDA grants to research leaders included in $14.1 million in new research funding approved during the Association’s July Board meeting. This is his first MDA grant, and he will be studying a variety of growth factors, including brain derived neurotrophic factor (BDNF), glial derived neuortrophic factor (GDNF), insulin-like growth factor 1 (IGF-1) and vascular endothelial growth factor (VEGF).

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 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.

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.