Kits to Quantitate and Analyze Mitochondrial Proteins

DESCRIPTION (provided by applicant): Mitochondrial dysfunction underlies the pathology of many disorders, including inherited mitochondrial diseases, many degenerative diseases, and the adverse effects of certain therapeutic drugs. At present there are no reliable standardized methods for detection and analysis of these defects. Our goal is to meet these needs with a set of novel assay kits that simplify analysis of key mitochondrial proteins and add powerful new analytical capabilities. These innovative kits are based on a unique set of proprietary reagents and associated intellectual properties developed by the Principal Investigators. The reagents are monoclonal antibodies (mAbs) that immunocapture intact, functional mitochondrial enzymes, and the intellectual properties are patents (pending) that cover these mAbs and their application. The Phase II STTR grant will drive commercialization of these technologies in three areas: 1) pharmaceutical drug safety screening for mitotoxicity, both to guide new drug development and to monitor adverse effects of licensed therapeutic drugs; 2) diagnosis of mitochondrial disorders (inherited and acquired) with simple standardized tests; and 3) research applications ranging from basic research into mitochondrial structure/function, to applied research that will help identify biomarkers (pathogenic and diagnostic) of mitochondrial disease. We have successfully met our Phase I goals and also accomplished additional work proactively. Working prototypes of all three types of kits are in hand and we have documented their basic performance characteristics. We are currently marketing early versions of some kits, and have established that there is a strong scientific and commercial demand for them. Phase II work will complete the development of these and other tests and validate their technical utility, making them ready for commercialization. Relevance in lay language: Mitochondria are/the powerhouse of cells and produce 95% of the energy needed for life. The proposed kits will enable personalized mitochondrial medicine, including measurement of an individual's mitotoxic burden (the degree to which his/her mitochondrial energy production system is damaged), and allow assessment of an individual's sensitivity to mitotoxic agents (including adverse effects of therapeutic drugs and environmental toxins). The kits will also be used to detect and monitor the progression of inherited mitochondrial diseases and degenerative diseases such as Parkinson's and Type-2 diabetes, and this will help clinicians determine whether or not new therapies for these disorders provide beneficial results. Finally, they will offer a way to for new therapeutic drugs that can protect mitochondria from toxic agents, and thus potentially prevent or treat mitochondrial disorders.