Q: Why is it that most of the UMDF-funded projects do not directly involve potential treatments and cures for mitochondrial disease?
A: They do! There are three main categories of research as it relates to drug development: basic, translational and clinical. All three of these categories are important for the effective development of treatments and cures. Researchers realize that, in order to “fix” a problem in the human body, they must first understand the basics: the structure and function of that system, organ, cell or organelle. Secondly, in translational research, they attempt to determine what happens when some part of that structure or function is manipulated. These two areas form the building blocks for clinical research. While there is still much to be learned about the basic structure and function of mitochondria, science is advancing quickly and translational tools such as cell and animal models are becoming more common. Clinical research focuses more on standardized testing and diagnosis, treatments, and eventually a cure, and here too UMDF encourages applicants to submit proposals of potential impact. An additional focus of the UMDF is to support and encourage new researchers in the field of mitochondrial disorders. Once these researchers become established, it is then more likely they will be competitive for much larger grants through bigger organizations and institutions such as the National Institutes of Health (NIH).