Action Duchenne supported a project with Professor Matthew Wood, Oxford University. This one year contract, which started in September 2013, looked at a small molecule drug (BGP-15) which activated the heat shock response (a hsp72 agonist) and, therefore, has the ability to protect cells (e.g. muscle cells) from stress (such as that resulting from muscle degeneration). This therapy has showed benefit in the mdx mouse and dystrophin and utrophin deficient DKO mouse.

BGP-15 was being investigated in diabetes and a range of other disorders and had been shown to be well-tolerated. Of course such an approach cannot repair or replace dystrophin, but by helping to protect degenerating muscle cells it may help to create a much more favourable environment for other therapies, including exon skipping to work. This pump priming project therefore aims to carry out a preliminary study to determine whether or not BGP-15 has any synergistic benefit when co-administered with current state-of-the-art antisense oligonucleotides for exon skipping, peptide-conjugated PMOs.