Hear Professor Marnie Blewitt from WEHI talk about her project to identify chemicals that activate one of the two major (silenced) PWS gene clusters, Magel2. A few years ago, Professor Blewitt stumbled across a chemical that silences an enzyme Schmd1 and as a consequence activates the Magel2 gene cluster i.e. a potential medicine for Prader-Willi.
Because she had previously been introduced to PWRFA, Marnie was interested in investigating whether her accidental discovery could be turned into a therapy for Prader-Willi. We initially seed funded her $25,000 to validate her finding and she has then taken her work on to proof of concept of a gene therapy and developed a first chemical candidate for a pharma company to develop into a drug. Pharma companies need more than one chemical candidate to successfully develop a drug, so Professor Blewitt’s work has now been fast-tracked to the National Drug Discovery Centre in the search for more compounds that will silence Schmd1 and activate the silent Magel2 gene cluster. There’s still a way to go and no guarantees but this world-leading research offers so much hope for a medicine for our children.
Complete the signup form here to secure your copy of the Webinar, held on 28 October 2020, with Professor Blewitt discussing her work.