Pre-Clinical Drug Screening
The pre-clinical drug testing facility at Children’s National Medical Center is a unique resource for performing preclinical efficacy drug trials in murine models of muscular dystrophy (e.g. mdx and A/J mice). The facility is equipped to perform a battery of functional (e.g. Rotarod, Grip strength), behavioral (e.g. open field digiscan), histological (e.g. H&E), biochemical (e.g. Serum muscle enzymes), immunological (e.g. lymphocytes, macrophages, and dendritic cells), and molecular assays to assess efficacy of various pharmaceutical and other therapeutic interventions in murine models of muscular dystrophy. The current project is aimed at testing the efficacy of drugs that are likely to affect inflammation, membrane stabilization and myoferlin upregulation. It is anticipated that some of these drugs are likely to reduce muscle fiber damage and improve muscle function in dysferlin deficient mice.
Outcome: None of the 5 compounds chosen for testing produced significant improvements in muscle function in dysferlin deficient mice. In addition the project revealed that determining robust outcome measures to test in dysferlin deficient animals is a significant challenge.