Researchers from Saint Louis University, Missouri, USA, have identified a new drug that has the potential to treat facioscapulohumeral muscular dystrophy (FSHD).
The drug – called apabetalone – is able to switch off the DUX4 gene. This gene is inappropriately switched on in people with FSHD and produces DUX4 protein in their muscles. DUX4 protein is damaging to muscles and is thought to cause inflammation and muscle wasting in people with FSHD.
Switching off the DUX4 gene with a drug like apabetalone stops DUX4 protein being produced and could therefore be a potential treatment for FSHD.
Although the testing of apabetalone is in relatively early stages, we will get in touch with apabetalone’s manufacturer, Resverlogix, to get further information on their plans. We will keep you informed.
Update 22/2/17 – Reservalogix is “currently working on delivery technology. We hope to launch a clinical trial for FSHD in 2018.”
Update 18/4/17 – The FSH Society have published an informative Q&A with Dr Fran Sverdrup from Saint Louis University, who carried out some of the research testing apabetalone and other BET inhibitors. In the interview, he said “it is too early in the process to predict which BET inhibitor might be the best candidate for clinical trials or when a clinical trial might start.”
For more information, read Resverlogix’s press release.
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