Friends of FSH Research and FSHD Canada Fund Two New Projects

Posted by George Shaw on Jul 16, 2014

We are excited to announce that we have funded two very promising research projects in collaboration with the FSHD Canada Foundation. Based in Seattle, Friends has been funding innovative FSH research for over 10 years. Over the last few years, FSHD Canada has matched funding on projects that have accelerating our progress towards understanding FSH. Knowledge truly is hope, and because of the studies we’ve been able to fund, we now have a better understanding of how to steer the research towards a treatment or cure.

There is now a consensus in the research community that a protein called DUX4 is playing a central role in the initiation of muscle deterioration. Finding a mechanism or strategy to inhibit DUX4 from doing damage to muscle is a critical step towards developing treatments. To complement our current portfolio in this area, we have funded Dr. Michael Kyba at the University of Minnesota to conduct a massive screening experiment looking for novel inhibitors of DUX4. Using a novel platform developed by Dr. Ali Tavassoli at the University of Southampton, Dr. Kyba will be able to test tens of millions of potential small molecules for the ability to inhibit DUX4 activity. Dr. Kyba has an excellent track record performing large scale screens, and recently has published his first results from the first DUX4 inhibitor screening effort.

The second project we have funded was proposed by Dr. Daniel Miller and Dr. Rabi Tawil at the University of Washington and University of Rochester respectively. Drs. Miller and Tawil have collected serum samples from a group of patients who volunteered to donate blood samples to the laboratories. They will test the blood using a novel technique to search for serum biomarkers that are specific for FSH. Much can be learned through such a study. Not only might Drs. Miller and Tawil identify new and robust markers that can be used in later stage clinical trials, but by testing a wide array of serum biomarkers we may gain critical insight into the mechanisms of muscle damage.

These studies not only fuel our hope that one day we will have a treatment for FSHD, but also represent how very far we have progressed along the path to that cure. We look forward to keeping you up-to-date on the status of the current projects.

See Grant Cyclic peptides inhibitors of DUX4

See Grant Identification of Serum Biomarkers for Measurement of FSHD Progression