Molecular Therapeutics

分子治疗 分子治疗学计划旨在整合基础和转化科学，以开发具有强大科学依据的新型治疗药物和临床试验。该计划将与癌症治疗分子方法相关的四个广泛领域的工作人员编织在一起：致癌信号，结构生物学，药理学和药物遗传学以及药物发现和递送。比如说， 结构生物学家联合收割机与药物化学家一起进行小分子药物发现工作，然后与动物模型专家一起测试这些药物。我们的物理科学家使用这些相同的模型来测试新的治疗方法，我们的药理学家研究这些新疗法的递送和功效。项目成员的研究得出了令人兴奋的发现， 其中包括创新的纳米递送方法、新型碳纳米管x射线源、治疗毒性的新生物标志物，以及增强对癌症信号传导途径的理解和建模。 该计划通过将物理学家，化学家，小鼠遗传学家，生物学家，药理学家以及临床和转化研究人员聚集在一起，共同参与新药发现，阐明治疗相关化合物的作用机制以及创新的临床试验，为中心增加了价值。提供试点资金，以推动有前途的治疗方式向前发展，同时注意向 临床和商业化。42名项目成员来自三所学校的七个部门： 医学院、药学院、文理学院。成员是4个项目赠款的主要参与者。2009年的费用供资总额为3 170万美元，包括国家癌症研究所供资960万美元。在过去六年中，成员们出版了1 120份出版物，其中8%是方案内出版物，21%是方案间出版物。加里约翰逊博士，过去7年的项目共同负责人，是药理学的主席，并以其定义MAPK信号转导通路及其生物学意义的研究而闻名。 博士Norman Sharpless，过去2年的项目共同负责人，是一位临床活跃的医生-科学家，在转化研究方面享有全国声誉，在小鼠癌症模型方面具有特别的专业知识。通过战略招聘和分子治疗计划的规模显着增加，比前一时期的资金。该计划准备扩大努力，为癌症患者开发创新的治疗药物，诊断和设备，并与临床研究和乳腺癌计划一起进行测试。

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.