CMD Drug Discovery Meeting

The CMD High Throughput Screen meeting at National Chemical Genomics Center (NCGC) provided the opportunity to discuss CMD targets, development and optimization of target specific assays and expanded screening to NCGC. The meeting set the goal to identify 2 CMD target assays and begin high throughput screening using NCGC resources by 2011.

Carsten Bonnemann provided a CMD overview: clinical background and targets identified at the July “Therapeutic Targets in the CMDs” conference. Dean Burkin presented proof of concept and his preliminary data from an alpha1beta7 integrin assay. The assay which uses 2 reporters (lacZ and luciferase) has identified compounds which upregulate integrin expression. Paul Martin discussed his work on Galgt2 in the mdx and dyw mouse. His current research has focused on both transgenic and AAV mediated upregulation of Galgt2. An assay he developed to identify small molecules that upregulate Galgt2 will be further optimized prior to HTS. Valerie Allamand presented data from a nonsense mediated decay (NMD) assay developed in France and rationale behind pursuit of NMD and premature codon read through in the collagen VI myopathies. Her presentation highlighted the necessity in predicting mutation effect on protein function and structure as a prerequisite to pursuing NMD and/or PTC read through. Denis Guttridge discussed the alternative and classic signaling pathways in the context of muscular dystrophy and NFkB as a target. He also presented recent data highlighting an NFkB inhibitor peptide as a potential therapeutic in conditions of muscular dystrophy involving the classical pathway. Sweta Girgenrath reviewed current work in the dyw mouse looking at IgF and IPLEX. Chris Austin presented an overview of high throughput screening and NCGC capabilities. Wes Friesen and John Babiak discussed the importance of secondary assays and the development of Ataluren. Jim Dowling provided a comprehensive review of current CMD animal models, murine and zebrafish. John Porter closed the meeting with a rousing talk exhorting all to place a premium on rigor, objectivity and early target dissection to ensure the development of the optimal lead candidates and mobilization of resources for meaningful targets.

Meeting participants included:
Dr. John Porter, NINDS
Dr. Petra Kaufmann, NINDS
Dr. Amelie Gubitz, NINDS
Dr. Glen Nuckolls, NIAMS
Dr. Christopher Austin, Director, NIH Chemical Genomics Center (NCGC)
Dr. James Inglese, Deputy Director, NCGC
Dr. Wei Zheng Group Leader, Cellular Signaling Assay
Dr. Ron Johnson, Team Leader, Molecular Genetic Assay Technologies
Dr. Catherine Chen, Postdoctoral Fellow
Dr. John Babiak, PTC Therapeutics
Dr. Wes Friesen, PTC Therapeutics
Dr. Carsten Bonnemann, Childrens Hospital of Philadelphia
Dr. Dean Burkin, University of Nevada
Dr. Paul Martin, Nationswide Children
Dr. Valerie Allamand, Institute of Myology, Paris, France
Dr. Denis Guttridge, Ohio State University
Dr. Sweta Girgenrath, Boston University
Dr. Jim Dowling, University of Michigan
Dr. Anne Rutkowski, Cure CMD
Denise Philippi, NCGC staff

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