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Much of the necessary methodology exists; however, the power of the Center for Precision Medicine is its dynamic platform that engages creative participation of scientists, clinicians, technology developers, data scientists, patient groups, and others. This initiative will also require new resources; these should not compete with support of existing programs, especially in a difficult fiscal climate. With enough resources and a strong, sustained commitment of time, energy, and ingenuity from the scientific, medical, and patient communities, the full potential of precision medicine can ultimately be realized to give everyone the best chance at good health.
The Center for Precision Medicine will provide unique opportunities to collaborate with the NIH, including but not limited to the Therapeutics for Rare and Neglected Diseases (TRND) Program. With an open environment, permitting the involvement of established experts on a given disease, the TRND program is designed to enable certain promising compounds to be taken through the preclinical development phase — a time-consuming, high-risk phase that pharmaceutical companies refer to as the “the valley of death.” Besides accelerating the development of drugs to treat rare and neglected diseases, the TRND program is designed to identify molecularly distinct subtypes of some common diseases, which may lead to new therapeutic possibilities, either through the development of targeted drugs or the repurposing of drugs by identifying subgroups of patients likely to benefit from them.
The Center for Precision Medicine will also collaborate with existing tissue banks and biorepositories on campus at UCMC - an important step toward linking them to clinical outcomes. Such a resource will allow for a much broader assessment of the clinical importance of genetic and “omic” variations across a range of conditions. The Center for Precision Medicine will also be a touch point for the CCTST Program- one of 46 centers funded by the NIH Clinical and Translational Sciences Award program and potentially the Mark O. Hatfield Clinical Research Center (the country's largest research hospital, in Bethesda, MD) to translate basic research findings into clinical applications. The Hatfield Center can provide specialized diagnostic services for rare and neglected diseases, offer a state-of-the-art manufacturing facility for novel therapies, and pioneer clinical trials of other innovative biologic therapies, such as those using human embryonic stem cells or induced pluripotent stem cells.
Real progress will come when clinically beneficial new products and approaches are incorporated into clinical practice. To this end, the following milestones will be the focus of our efforts:
A major misconception among physicians, scientists and the American public is that rare diseases of the heart exist in a virtual vacuum. In fact, this is not true. Many fundamental advances in medicine have their origins in the study of rare diseases. For example, the human genome project was stimulated by the identification of a gene responsible for muscular dystrophy. Similarly, the field of epigenetics began with early descriptions of rare diseases and the potential contribution of environmental and psychosocial conditions. Accordingly, the scientific and translational foundation of studying rare heart and lung disease is scalable and can be used to advance the understanding of common diseases, disorders and conditions and train the next generation of researchers.
CDCN's research model. The CDCN has taken a four-step approach to drive forward "omics" research: 1a) The CDCN identified and connected the research community and (1b) provided supportive resources to patients and loved ones. Then, the CDCN turns to the research community (2a) and patient community (2b) to prioritize key research studies into an International Research Agenda as well as collect and centrally store clinical data and tissue samples. With the top priority projects identified, the CDCN solicits funding from patients and loved ones for specific projects. Then, the CDCN provides two mechanisms for funding (3a) investigator-initiated research grants through traditional requests for proposals and (3b) strategically directed research grants where the DRO recruits the top experts within the community or outside of the community to do the studies. 4) Pharmaceutical companies are more eager to contribute tissue samples and funding for clearly defined projects with the greatest likelihood of return.
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