Projects of Wellstone
A multi-center team working together within the Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center focuses on identifying biomarkers to evaluate outcomes of clinical trials for facioscapulohumeral muscular dystrophy (FSHD). In Project 1, Dr. K. Wagner (Kennedy Krieger Institute / Johns Hopkins Hospital) works on a Phase I clinical trial with Acceleron to assess the effects of its myostatin inhibitor, ACE-031, on healthy human subjects, preparatory to assessing its use in FSHD. In Project 2, Drs. L.M. Kunkel (Harvard Medical School), M. Zatz (University Sao Paolo, Brazil), and R.J. Bloch (University of Maryland School of Medicine) use biopsy samples to identify changes in RNAs and proteins in FSHD, with the aims of identifying additional disease biomarkers and learning how they are altered in healthy human muscle upon myostatin blockade. Project 3, led by Drs. C.P. Emerson Jr. (University of Massachusetts Medical School (UMMS) and W. Wright (University of Texas Southwestern Medical Center), is deriving primary and immortal myogenic cell lines from FSHD and normal biopsies to identify biomarkers and examine their role in proliferation, differentiation, and pathogenesis of muscle cells. Project 4, led by Dr. J.B. Miller (BBRI) in collaboration with Dr. Bloch, uses mouse models and cultured human cells to analyze FSHD biomarkers and identify disease mechanisms.