Scientists at RTI International join effort led by the US National Institute of Neurological Disorders and Stroke (Ninds) to accelerate development of a treatment for spinal muscular atrophy, or SMA
Spinal muscular atrophy, a paralyzing neurodegenerative disease that causes loss of motor neurons in the spinal cord, is the leading genetic cause of death in infants and toddlers, occurring in as many as one in 6000 births annually in the United States.
By causing the loss of motor neurons, the disease interferes with the ability of electrical and chemical messages to reach muscle cells.
This in turn causes the muscle cells to atrophy, leading to muscle weakness.
Muscles required for swallowing and breathing are affected as well as those of the limbs.
Under terms of the contract, worth $2.1 million, RTI researchers, led by principal investigator Edwin Garner, will study ways in which to improve tests to screen for the disease by evaluating the pharmacokinetic, pharmacologic and toxicologic properties of candidate drug compounds.
Compounds will be screened in vitro for pharmacological activity against molecular targets.
Candidates showing activity against SMA will then be screened for metabolic liabilities and potential for brain penetration prior to conducting in life studies.
Drug discovery and development is a core research activity at RTI.
For more than 40 years, RTI has worked with pharmaceutical companies and government agencies to bring new medicines to the fight against disease.
This project is one component of a $22 million national Ninds-led programme to accelerate development of a treatment for SMA.
By means of such drug discovery research, Ninds hopes to have identified and completed preclinical research and development of candidate therapeutics for treating SMA by late 2007.
