More potential good news…. TWEAK Treatment for DM1?

A new article was published from one of the major research centers that researches myotonic dystrophy…. While drug trials are starting very few people this year will be able to access the drug and only 1/2 will get the drug and the other 1/2 will get placebo. Now comes news that a therapeutic agent that is currently available has the theoretical ability to help with myotonic dystophy muscle restoration. While the full article is not available yet here is the abstract from PubMed (below)

Why is this exciting, while it may be years before drugs are available (and paid for by insurance companies) this anti-tweak therapy is available today. It needs some vetting by the medical profession as to its exact course but may be an option for those with advanced myotonic dystrophy…

Hum Mol Genet. 2014 Dec 11. pii: ddu617. [Epub ahead of print]

TWEAK/Fn14, a pathway and novel therapeutic target in myotonic dystrophy.


Myotonic dystrophy type 1 (DM1), the most prevalent muscular dystrophy in adults, is characterized by progressive muscle wasting and multi-systemic complications. DM1 is the prototype for disorders caused by RNA toxicity. Currently, no therapies exist. Here, we identify that Fn14, a member of the TNF receptor super-family, is induced in skeletal muscles and hearts of mouse models of RNA toxicity and in tissues from DM1 patients, and that its expression correlates with severity of muscle pathology. This is associated with downstream signaling through the NF-κB pathways. In mice with RNA toxicity, genetic deletion of Fn14 results in reduced muscle pathology and better function. Importantly, blocking TWEAK/Fn14 signalling with an anti-TWEAK antibody likewise improves muscle histopathology and functional outcomes in affected mice. These results reveal new avenues for therapeutic development and provide proof of concept for a novel therapeutic target for which clinically available therapy exists to potentially treat muscular dystrophy in DM1.

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