U. of Florida Researcher Awarded $180K Grant to Test Sildenafil-Citrate (Viagra) in Duchenne Muscular Dystrophy
TUCSON, Ariz., Aug. 18, 2010 – Can a drug most commonly used for erectile dysfunction be effective in combating the devastating effects of Duchenne muscular dystrophy (DMD)?
There is growing interest in the theory that limited blood flow and oxygen to muscles contributes significantly to muscle damage in boys with DMD. Sean Forbes, Ph.D., is studying how sildenafil citrate, sold under the brand name Viagra, might benefit individuals with DMD, by increasing blood flow and oxygen to their damaged muscles.
Forbes, a post-doctoral fellow at the University of Florida-Gainesville, has just been awarded a $179,922 Development Grant from the Muscular Dystrophy Association to pursue that theory. MDA Development Grants are awarded to outstanding young investigators to cultivate their career development in the area of neuromuscular disease research. Another MDA-funded investigator, Ronald Victor, at Cedars-Sinai in Los Angeles, currently has a clinical trial underway to study tadalafil, or Cialis, as a therapy for DMD and Becker muscular dystrophy.
 | | Sean Forbes, Ph.D. |
“I find it tragic how quickly muscles can deteriorate and become dysfunctional in some of these diseases such as Duchenne muscular dystrophy,” said Forbes. “We expect sildenafil citrate to enhance muscle blood flow and oxygen that will reduce susceptibility to damage, to improve muscle function and ultimately improve ability to walk, for example, and improve the quality of life for children with DMD.”
DMD affects one out of every 3500 boys, causing progressive muscle weakness, eventually causing heart and breathing problems by early adulthood.
Forbes will use magnetic resonance imaging and spectroscopy to see the full picture of the muscle. By using these non-invasive techniques instead of traditional muscle biopsies, more boys may be able to take part in clinical trials and Forbes will get a more complete picture of what’s happening in the entire muscle, rather than just one tiny segment.
Forbes is one of 38 research leaders around the world who are recipients of new multi-year grants just awarded by the Muscular Dystrophy Association Board of Directors. This round of new MDA research grants totals over $14 million, reinforcing the Muscular Dystrophy Association’s role as the leader in non-governmental research funding for neuromuscular diseases. MDA annually provides almost $39 million to fund research looking for treatments and cures for over 40 neuromuscular diseases.
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.”
This is the first grant that Dr. Forbes has received from MDA. As a young researcher, it can be difficult to garner a research grant when competing with more seasoned, proven researchers. But encouraging and supporting young researchers is a priority at MDA.
“We need to keep encouraging top young scientists to devote their talents to the field of neuromuscular disease research,” said Louis Kunkel, Ph.D., chairman of the MDA Scientific Advisory Committee. “Having personally received a MDA Development Grant early in my career, I know first-hand how important early funding is and I am delighted Sean Forbes is getting early MDA support, too. He's talented and has the potential to help speed treatments and cures for muscle diseases.”
MDA-funded scientists have uncovered the genetic defects that cause several forms of muscular dystrophy; Charcot-Marie-Tooth disease; a form of amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease); childhood spinal muscular atrophy 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.
In the Gainesville area, people affected by any of the more than 40 muscle diseases covered by MDA’s program can receive excellent medical care at the MDA Clinic at Shands Hospital. In all, there are 12 MDA Clinics in Florida, including Jacksonville, Tampa, St. Petersburg, Orlando and Miami.
MDA is the nonprofit health agency dedicated to curing muscular dystrophy, ALS and related diseases by funding worldwide research, view video 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" at http://www.mda.org/research/gaag/2010, an online slideshow that showcases each grant with photos and detailed information. |
