friends of FSH Research

Funding Research for
FSH Muscular Dystrophy Dots

Scope of Research

In order to support the most innovative research, Friends fosters projects that support the process of drug discovery (even though our strategies are not always focused on traditional pharmaceutical drugs). By encouraging collaboration, open communication, and focused direction, we are thinking forward about how the projects we fund will help researchers build robust programs that they need to cure the disease.

We think of drug discovery as having four steps that each flow into one another:

PipelineBasic Science is an all-encompassing term to cover basic discovery and research. In the context of a disease, we view this as focusing on “target” identification. Targets are any molecule, pathway or system that can be intervened with to reduce disease burden. Research has shown that the successful development of clinical interventions is dependent on a solid understanding of targets. Therefore, we do not shy away from projects that will help us understand more about the underlying mechanisms of FSHD. Basic Science projects are the most risky, and often require the most support to gain traction to continue down the pipeline.

Strategy/Lead Compound Identification is the process of building strategies and systems to neutralize or modify the target identified through basic research. For example, if gene X is defined as causing FSHD, we need to devise strategies to stop gene X. If gene Y is required for gene X to cause FSHD, we can also develop strategies around gene Y. The ultimate goal of this stage is to identify a compound or strategy that can be tested to prove whether it will have a beneficial effect on the disease.

Preclinical Models are designed to test the efficacy of compounds and strategies identified in animal models. This may seem like a superfluous or unnecessary step, but oftentimes compounds identified during the first stages need to be optimized to be able to work in people. Sometimes they can be very toxic, and sometimes they just don’t work. Preclinical work is very important to establish before investing in later stage clinical trials.

Clinical Trials are designed to prove that interventions actually work. They help establish standard of care and an in depth understanding of potential side effects and risk/benefit ratios. It goes without saying, that the more you know from stages 1-3, the better the chances are that the clinical trial will work.