Posted by Terry Elsas Colella on Oct 31, 2022
I would like to share a letter of thanks we received from Dr. Robert Bloch, one of our grantees. Dr. Bloch has provided key insights into FSHD, and his xenograft mouse model (human FSHD muscle tissue grafted to a mouse) has been an invaluable resource for the research community. Really, his letter would be more appropriately addressed to you, the actual funders of this and other research, which has steadily brought us closer to the goal of a treatment or cure for FSHD:
I would like to thank Friends of FSH Research again for the support it has provided to my laboratory for the past 5 years. Initially, Friends helped fund the work that led to the development of a xenograft model for FSHD, which has allowed us to study mature FSHD muscle tissue in mice. This award from Friends facilitated our work with Fulcrum Therapeutics, which helped them prepare for the clinical trial of losmapimod, now in Phase 3. We’ve been using this model to understand the pathogenesis of FSHD, to identify biomarkers, and, in collaboration with several other researchers in academia and industry, to test possible therapies. Our second award from Friends helped us initiate our studies of SLC34A2, a potential protein biomarker of FSHD, and without it, we never would have been able to advance our work to the point it is now. In addition to follow-up I funding I’ve received from the FSHD Society, Anthony Saleh of miRecule and I were able to obtain a grant from the Maryland Industrial Partnerships program to further his research aims and to continue our work on the SLC34A2 protein biomarker. I recently learned that our work on the biomarker would receive complementary funding from SOLVE FSHD, which should help us determine if it useful in diagnostics and tracking disease progression, as well as in assessing different therapies now being developed for clinical trials. I presented our work on the biomarker and its potential use in patients at the ENMC Workshop in Amsterdam on Oct. 2, 2022. It was very well received, and I was pleased to acknowledge the grants from Friends of FSH Research.
Again, thanks for all that you and your colleagues at Friends have done to support our studies of the xenograft model and of SLC34A2. I believe that, together, these approaches have great possibilities.
October 13, 2022
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