Minutes from May 9th, 2014
Medical Exchange Club meeting
Date:
9th May, 2014
Location:
St. Botolph Club, Boston
Speaker:
Robert Brown, M.D.,
D. Phil. Chair and Professor of Neurology
University of Massachusetts Medical School
Members Present:
Barbara Gilchrest
Aram Chobanian
Pat Donahoe
Terry Flotte
Harvey Lodish
David Nathan
Michael Rosenblatt
Nick Zervas
John T. Potts
Ken Walsh
Mark Poznansky
Thoru Pederson
Joe Majzoub
Jay Vacanti
Cocktails:
Talk:
Robert was introduced and hosted by Pat Donahoe. Robert gave a wonderfully erudite talk about his life’s work to address the clinical needs of his patients and uncover the genetic defects associated with ALS. Robert presented the methods used to uncover many gene defects ( including SOD-1 and many other genes) that are believed to contribute to 10% of ALS seen in the community. These genes encode proteins which play critical roles in protein stability, chromatin biology, the cytoskeleton and responses to environmental factors. It has been clearly shown by Robert that certain mutations in SOD-1 can result in ALS and in turn potentially be targeted by gene therapy. Robert described his international efforts to identify families with ALS throughout the world and including the USA, Cape Verde, Sweden, Israel and Belgium to name but a few. Robert also described both elegant and promising screening techniques and gene therapy approaches that are under development to address genetic defects associated with ALS using gene silencing mechanisms that could be directed to the CNS. In this way, Robert described definitive work that provides undoubted optimism for the future for individuals challenged and suffering with the remarkably challenging and unpleasant disease that is ALS.
Post-Dinner Discussion:
Robert lead a wide ranging discussion about new approaches to the diagnosis and treatment of ALS with additional emphasis on the potential role of MSCs and IPS cells in therapy. The consensus was that this had been an enlightening meeting in which Robert communicated much novel discovery work relating to a clinically and scientifically challenging disease.
Respectfully submitted, Mark Poznansky, Scribe