Originally published on March 25, 2014.
Cure SMA is leading the way to a world without SMA by advancing a comprehensive research program, of which basic research is a key component. Cure SMA has awarded 79 basic grants for a total of $9,507,612 in the past 10 years. Dr. Rashmi Kothary at The Ottawa Hospital Research Institute will receive $100,000 to explore non-SMN mediated mechanisms to treat SMA.
The Non-SMN Mediated Benefits of The HDAC Inhibitor Trichostatin A
Objective: Our goal is to better understand how a small molecule (TSA) that is a global gene regulator ameliorates the disease symptoms and pathology in a mouse model of SMA.
Research Strategy: We will study what aspects of muscle growth and maintenance are targeted by TSA, both at the biological and molecular levels.
Significance: These studies are directed towards gaining a better understanding of the mechanism of action behind the beneficial effects of TSA on our intermediate mouse model of SMA. Our data currently points to a SMN independent mechanism for the drug. Therefore, this work has the potential to find novel therapeutic strategies for SMA by generating additional pathways to target for drug discovery beyond SMN enhancement.
Funded by Cure SMA Canada.
Meet Dr. Kothary:
Who are you?
Dr. Kothary is Senior Scientist at the Ottawa Hospital Research Institute and Professor at the University of Ottawa. His interests are in understanding the mechanisms that maintain/alter muscle and nervous system integrity. In parallel, they are assessing ways to alleviate the pathology of neuromuscular diseases.
How did you first become involved with SMA research?
His interest in SMA research stemmed from a collaboration with Dr. DiDonato on using mice as models to study the genetics of the disease. This was in 1999 and Dr. Kothary has been involved in various aspects of SMA research ever since.
What is your current role in SMA research?
Dr. Kothary’s current research focuses on studying pre-clinical models of SMA to better understand disease pathogenesis and to develop novel therapeutic strategies. An area of interest is to identify and develop non-SMN mediated therapies.
2014 Cure SMA Research Mission and Funding
The overall mission at Cure SMA is a world without SMA. Making this happen requires scientific research investments. Thus, our main research goal at Cure SMA is to accelerate the discovery of an effective treatment and cure for SMA by funding and advancing a comprehensive research program, including:
• Basic Research to reveal the best ways of making SMA drugs,
• Drug Discovery to make new drugs,
• Developing Clinical Trial infrastructure to help test new drugs, and
• Clinical Care Research to improve the quality of life in SMA patients.
In addition to the basic research funding announced in this edition of Compass, Cure SMA will invest in more research in 2014 to advance our vision of a world without SMA. Please be on the lookout for two upcoming Requests for Proposals (RFPs) for additional research funding from Cure SMA in 2014 in the following research areas: 1) Drug Discovery – Overseen by the Translational Advisory Council, and 2) Clinical Care – Overseen by the Medical Advisory Council.