MDA AWARDS $79K TO YOUNG BIOTECH FIRM IN SALT LAKE CITY

TUCSON, Ariz., Aug. 18, 2010 – John Manfredi, Ph.D., a Salt Lake City-based researcher who has already identified a series of novel drug-like compounds that promote growth and function of motor neurons, has now been awarded a $79,000 grant from the Muscular Dystrophy Association to evaluate the therapeutic potential of these designer drugs. The compounds work by a mechanism that is unique among experimental treatments of spinal muscular atrophy or SMA.

Manfredi’s 17-month study at Sfida Biologic, Inc., the biotech firm he founded about a year ago, will test the compounds using zebrafish that have been genetically engineered to simulate aspects of SMA.  The leading hereditary cause of infant mortality, SMA is an autosomal recessive condition that also is the most common inherited motor neuron disease.

John Manfredi, Ph.D.

“I was working on a project with a company which discovered a series of compounds that appeared to have activity in neurodegenerative diseases,’’ said Manfredi.  “For rational reasons we hypothesized the effects of these compounds would be even more dramatic for motor neuron diseases. When we tested that hypothesis, indeed, we found that they had the potential to be particularly effective in motor neuron diseases.’’

To learn more about Manfredi listen to his podcast.

His new MDA-funded project will quickly test compounds using zebrafish that are deficient in the SMN protein that characterizes the human disease.  Specifically, Manfredi’s team will evaluate the abilities of the compounds to increase survival, restore the integrity of neuromuscular junctions, and suppress motor axon defects due to SMN deficiency.  Possible outcomes include the identification of new chemical entities as potential SMA therapeutics; identifying a novel protein target for treatment of SMA; and validating SMN-deficient zebrafish as a reliable platform for discovery and development of small-molecule therapeutics for SMA.

“Our company will be dedicated to the development of a series of compounds which we believe will be efficacious for the treatment of motor neuron diseases in spinal muscular atrophy,’’ Manfredi said.

Manfredi said he is “eternally grateful that the MDA has bet on me and the compounds,’’ and that if his study is successful, it could lead to treating other motor neuron diseases, including amyotrophic lateral sclerosis (Lou Gehrig’s Disease).

Manfredi is one of 38 research leaders now receiving multi-year awards from MDA. His project is part of the $14.1 million in new research funding approved during the Association’s annual board meeting in July.
“Without MDA funding, we would not be able to take this next step on the ladder of development of these compounds,’’ Manfredi underscored. “The funding is absolutely critical and could allow us to compete successfully for funds from the National Institutes of Health and other organizations. Without the MDA funding, we would not be able to accumulate the data and to compete effectively for the money from the NIH.’’

“Dr. Manfredi’s strategy of quickly and cost-effectively testing a variety of promising compounds in zebrafish, a model of human disease, has significant potential,’’ noted Valerie Cwik, M.D., MDA executive vice president – research and medical director. “This is Dr. Manfredi’s first MDA grant and we are encouraged by the strategies and tactics he’s using to attack spinal muscular atrophy.’’

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 also enhanced clinical care for individuals affected by muscle disorders, achieving important quality of life and longevity gains. The Association, which annually invests almost $39 million on 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 (CMT) disease; 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 Salt Lake City area, individuals affected by any of the more than 40 muscle diseases covered by MDA can receive excellent medical care at the MDA Clinic at University of Utah Medical Center and the MDA/ALS Center at the University of Utah.  

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, go to: http://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.