Heat shock hits muscular dystrophy
Australian academics have used N-Gene Research Laboratories Inc.'s BGP-15, a small molecule inducer of heat shock 70 kDa protein 1A in Phase IIb testing for diabetes, to increase muscle strength and survival in two mouse models of muscular dystrophy.1 The biotech now hopes to find a partner to help repurpose the compound for muscular dystrophy.
The lack of a functioning dystrophin (DMD) protein is the underlying cause of muscular dystrophy, but chronic muscle inflammation and loss of intracellular calcium homeostasis are what actually drive disease progression. Multiple proinflammatory signaling molecules, including tumor necrosis factor-a (TNF-a), NF-kB and c-jun N-terminal kinase (JNK), are upregulated in the muscle tissue of animal models of muscular dystrophy.2-4...