The primary goals of the Molecular Therapeutics (MT) Program are to study and evaluate cancer targets, develop novel chemical probes that will lead to new therapies, and devise more effective delivery systems to treat cancer. MT has highly integrated basic science and translational themes in five areas: chemical and structural biology; drug discovery and development; drug delivery and nanotechnology; systems pharmacology; and oncogenic signaling. MTs objective is to combine these elements to develop novel therapeutics and to translate innovative discoveries into applications relevant to LCCC-initiated human trials. Interactions of MT?s members with LCCC basic, translational and clinical faculty enables many of the scientific steps needed for the discovery and development of promising therapies. This includes: (1) Discovery and validation of new targets for cancer therapies, (2) Development of chemical probes to modulate and further validate identified targets, (3) Development of faithful animal cancer models for the testing of novel therapeutics, (4) Discovery and application of novel surface chemistry and nanoparticle synthesis for delivery and formulation of promising therapeutics, (5) Characterization of PK/PD of novel therapeutics involving preclinical models and patient clinical trials. The focused development of MT during the past five years provides the requisite infrastructure and knowledge-base to truly do drug discovery in an academic setting. A major strength of the program has been the enhancement of chemical biology that has benefited virtually everyone involved in cancer research at UNC and in the Cancer Center. Examples of this success are represented by LCCC investigator lead startups developing clinical-candidate MER inhibitors with potent anti- tumor activity entering phase 1 trials, and the discovery of pharmacological quiescence where a lead compound will begin Phase II testing in small cell lung cancer in early 2015. Leadership for Molecular Therapeutics is provided by Stephen Frye, Director of the Center for Integrative Chemical Biology and Drug Discovery (CICBDD) and Fred Eshelman Distinguished Professor in the UNC School of Pharmacy and Gary Johnson, the Kenan Distinguished Professor and Chair of the Department of Pharmacology in the School of Medicine. The program fosters a strong integrated research effort through the establishment, use and advancement of core facilities and promoting highly interactive collaborations with Cancer Center investigators. The Molecular Therapeutics Program consists of 42 members associated with the Schools of Medicine, Pharmacy and Arts & Sciences. During the last funding period, program members have published 700 cancer- related articles (30% collaborative). In 2014, our program members held 89 grants and $22M (total cost) in annual extramural funding, including 36 grants and $8.8M (total costs) from the NCI.
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