Spotlight - Volume 3, Issue 1. January 2012
In this issue of Spotlight…
In this issue of Spotlight, we highlight one of the most successful of the 19 Rare Disease Clinical Research Consortia, the Vasculitis Clinical Research Consortium, directed by Peter A. Merkel, MD, MPH. In highlighting this outstanding program, one can see the crucial role for multi-center collaboration for development of better diagnostic and therapeutic modalities in rare diseases.
On another front the increasingly close interactions between the RDCRN and the CTSA programs were cemented earlier this year when Rick Barohn became CTSA Director at The University of Kansas. His experience in the Clinical Investigation of Neurologic Channelopathies (CINCH) consortium, will be a critical component to furthering the relationship between the CTSA’s and rare disorders. (See the last Spotlight edition for an overview of the operation of CINCH)
Several benchmarks of the quality of RDCRN research were on display at our most recent RDCRN Steering Committee meeting in Bethesda. Two outstanding research outcomes from the RDCRN were highlighted, one concerning arginine and argininosuccinic aciduria (Urea Cycle Disorders Consortium) and another exploring lessons from the MILES trial (Rare Lung Diseases Consortium) reported this year in The New England Journal of Medicine.
We call special attention to the report of the MILES trial for another reason also. In this article, the full range of challenges and opportunities in rare disease clinical research emerged with exceptional clarity. All involved in rare disease research should carefully read and learn the many lessons it teaches.
Spotlight is edited by David Robertson, MD, Elton Yates Professor of Medicine, Pharmacology and Neurology and Director of the Clinical and Translational Research Center of Vanderbilt University's Clinical and Translational Science Award (CTSA). He is PI of the Autonomic Disorders Consortium. Cindy Dorminy, MEd LPN is Assistant Editor.
The staff of Spotlight are enthusiastic about the potential of our new publication to enhance communication among those interested in treatment and cure of rare diseases. We are very interested in your thoughts about how Spotlight can be improved. If you have suggestions for future issues, please let us hear from you. You can send your comments to RDNWebmaster@epi.usf.edu.