Exciting news - research has now been published which provides the first proof of principle for an epigenetics-based therapy for Prader-Willi syndrome. The study showed two selective inhibitors were able to activate the imprinted genes on the maternal chromosome of cells (which are usually repressed in people with PWS). These results raise the possibility that inhibitors or drugs derived from them may be able to activate imprinted genes in people with PWS.
Our usual words of caution - these are early stage results and rely on mouse models, but they have been published in a very high quality scientific journal and undergone peer review. The authors have outlined plans moving forward including comprehensive evaluation of the inhibitors in pre-clinical studies to fully explore their therapeutic potential for treating PWS.