Molecular Therapeutics

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Molecular Therapeutics The Molecular Therapeutics Program seeks to integrate basic and translational science to develop novel therapeutic agents and clinical trials that have strong scientific rationales. The program weaves together workers in four broad areas related to molecular approaches to cancer therapy: oncogenic signaling, structural biology, pharmacology and pharmacogenetics and drug discovery and delivery. For example, structural biologists combine with medicinal chemists in our small molecule drug discovery effort, who then work with animal models experts to test these agents. These same models are used by our physical scientists to test novel therapeutic approaches with the help of our pharmacologists who study these delivery and efficacy of these new therapeutics. Research by program members has resulted in exciting findings, among which are innovative nanodelivery approaches, novel carbon-nanotube x-ray sources, new biomarkers of therapeutic toxicity, and enhanced understanding and modeling of cancer signaling pathways. The program adds value to the center by bringing physicists, chemists, mouse geneticists, biologists, pharmacologists, and clinical and translational researchers together to participate in new drug discovery, the elucidation of mechanisms of action of therapeutically relevant compounds, and innovative clinical trials. Pilot funding to push promising therapeutic modalities forward is provided as is attention to movement into the clinic and commercialization. The 42 program members come from seven departments from three schools: Medicine, Pharmacy, and College of Arts and Sciences. Members are major participants in 4 program project grants. Total cost funding in 2009 was $31.7 million including $9.6 million in NCI funding. Members generated 1,120 publications over the past six years, of which -8% were intraprogrammatic and 21% were interprogrammatic. Dr. Gary Johnson, program co-leader for the last 7 years, is the Chair of Pharmacology and is renowned for his research defining MAPK signal transduction pathways and their biologic significance. Dr. Norman Sharpless, program co-leader for the last 2 years, is a clinically active physician-scientist with a national reputation in translational research with particular expertise in murine cancer models. Through strategic recruitment and the Molecular Therapeutics Program has significantly increased in size over the prior period of funding. The program is poised to expand efforts to develop innovative therapeutic drugs, diagnostics and devices for patients with cancer and test them in concert with the Clinical Research and Breast Cancer Programs.