MDA awarded a research grant totaling $260,000 over a period of two years to Professor Dame Kay Davies, Dr. Lee’s Professor of Anatomy in the department of physiology, anatomy and genetics at the University of Oxford in the United Kingdom. The funds will help support Davies’ development of an effective therapy applicable to all people with Duchenne muscular dystrophy (DMD).
Davies’ approach is to identify a drug that will increase levels in muscle of the structural protein utrophin (similar to dystrophin, the protein deficient in DMD).
“A small molecule drug delivered to the whole body following oral administration can potentially target all affected muscle types, including heart and diaphragm, and would not require [as other therapies under development for DMD do] the use of an immunosuppressant,” Davies explained.
The team will screen candidate compounds for their ability to increase utrophin activity in a mouse model designed to permit real-time quantification of utrophin levels in the living animal.
Davies expects that the new screen will enable her team to rapidly identify new small molecules as lead candidates for DMD.
“Following on the grant from MDA,” Davies said, “we hope to be in a strong position to develop a clinical candidate and, ultimately, to deliver a best-in-class drug for the treatment of DMD.”
Funding for this MDA grant began February 1, 2012.