MDA awarded a grant totaling $79,277 to John Manfredi, chief scientific officer at Sfida BioLogic Inc., in Salt Lake City, Utah, for continued research into new drug compounds that promote the growth and function of motor neurons (nerve cells), and that may have potential as therapeutics for treatment of spinal muscular atrophy (SMA).
Manfredi's research team aims to determine the therapeutic potential of their compounds using a zebra fish research model that has been genetically engineered to simulate aspects of SMA. Results will determine whether the drugs merit future testing in more sophisticated and expensive models of the disease.
The compounds identified by Manfredi and colleagues "exhibit attractive pharmaceutical properties," Manfredi said. "They are not toxic; they penetrate the central nervous system; they exhibit appropriate half-lives in tissues; they can be administered orally; their permeability and solubility characteristics are drug-like; and they can be economically synthesized."
The group also will test a sample of their compounds alongside a collection of biochemically similar, commercially available drugs. If the commercial drugs show effects in the zebra fish model of SMA, the results will support the hypothesis that their SMA-relevant effects are due to shared biochemical activity with the investigators' novel drugs. Positive results also could lead to development of the commercial compounds as SMA therapeutics.
"The funding provided by MDA is absolutely critical for evaluating the potential of our company’s proprietary compounds to treat spinal muscular atrophy," Manfredi said. "Indeed, if these or derivative compounds prove to be efficacious for the treatment of SMA, MDA can legitimately claim responsibility for their success."
Funding for this MDA grant began August 1, 2010.