Dr James Dowling is announced as recipient of 2017 Myotubular Trust award

Combinatorial therapy as a novel treatment strategy for myotubular myopathy

We are delighted to announce a three year grant award to Dr James Dowling of Sick Kids Toronto. Two existing drugs, currently licensed for use in children who have other conditions, have been identified by Dr Dowling’s team as showing encouraging signs of improving the symptoms of myotubular myopathy.  This grant from Myotubular Trust will allow Dr Dowling and his team to investigate the effectiveness of both of these drugs, either alone or to complement other therapies. 
Repurposing medicines that are safe and have been used and studied in children, is a very exciting goal for rare diseases. We are delighted to be making our 2017 grant of £216,000 to Dr Dowling to pursue this promising project.
Because these drugs have previously been used in children, we feel that the pathway for clinical development and testing of them can be feasibly accomplished in a timely and cost effective manner.” Dr Dowling

During the course of this grant, Dr Dowling’s laboratory will also continue to develop on previous awards by the Myotubular Trust. Loss of MTM1 results in the accumulation of a lipid called PI(3)P in someone with myotubular myopathy. This project will continue to develop inhibitors of PI(3)P that work well in humans, and can be translated to clinical use.