Posted by Friends of FSH Research on Nov 14, 2018
We started enrollment with our first patient in the Clinical Study using MRI to measure immune response. The study is mainly a safety and feasibility study on using an immunosuppressive regimen including tacrolimus and prednisone to attempt to the reverse the MRI signal. Our prior work with the Wellstone Center suggest that the T2 MRI signal is correlated with an immune response. A secondary outcome in our study is to determine whether our immunosuppressive drug regimen can reverse the T2 MRI signal, therefore suggesting, that this biomarker is reversible and therefore a potentially useful biomarker for future drug interventions for FSHD, and that the signal represents an immune reaction.
Our goal is to recruit up to 6 patients, for which we are actively recruiting and will start to screen patients who do not have prior muscle MRIs to determine their eligibility for the study.
Our immunosuppression study was based on our findings in the Wellstone FSHD study of 36 patients with MRI-guided biopsies, where we found that MRI STIR imaging was associated with inflammation or active myopathy in ~70% of the muscle biopsies versus 25% of the STIR negative muscles. Therefore, a hypothesis is that muscle STIR hyperintensities represent inflammatory changes. To test the hypothesis, we tested the use of immunosuppressive medication tacrolimus and prednisone for the treatment of FSHD and used muscle MRI as an outcome measurement. We found that the treatment did not change the outcome measure, which itself is an important discovery and will be addressed in an upcoming publication.