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TRANSLATIONAL SCIENCE TRAINING PROGRAM
August 30 - 31, 2012

The Translation Science Training Program is an exciting opportunity for NIH Intramural Postdoctoral Fellows and Graduate Students to learn more about the bench-to-bedside process. The TSTP course is an innovative training program that, in one course, intertwines interdisciplinary scientific content, understanding of the drug development process, professional skills development, clinical trial terminology, and career exploration.

This year, the program will be held over two full days, in a "boot camp" format, from 8 am to 5 pm each day.

Attendees will be limited to NIH Postdoctoral Fellows and NIH Ph.D. Students.

Participants will:

  • Gain insight into the drug discovery and development processes, while various speakers utilize the case study of Imatinib (Gleevec) throughout the program.
  • Enhance depth and breadth of understanding of research areas outside of their own fields of study.
  • Increase awareness of opportunities for collaboration at NIH (e.g. Chemical Genomics Center) that could expand their current research projects.
  • Strengthen critical thinking skills through strategic analysis of the case study, as well as disruptive technologies.
  • Recognize the broad career options in translational research for someone with a Ph.D. in biomedical research, both in the public and private sectors.
  • Develop a network of mentors among a myriad of professionals who are involved in the bench-to-bedside process.

A Few Program Highlights...

  • Travel to the National Center for Advancing Translational Sciences (NCATS), tour the facility, and speak to scientists about their research
  • Valuable instruction and insight from experts and innovators in translational science, including increased awareness of careers in the field
  • A thorough look at the story of Imatinib (Gleevec), from its development to its emergence on the pharmaceutical market

Speakers:

  • Rajesh Ranganathan, Ph.D., Director of the NINDS Office of Translational Research, NIH and Co-Developer and Director of the TSTP Course
  • Gurusingham Sitta Sittampalam, Ph.D., Senior Scientist, NCATS
  • David Fink, Ph.D., Director of Entrepreneurial Services, University of Maryland, Baltimore County Research and Technology Park
  • Anthony Murgo, M.D., M.S., Associate Director for Regulatory Science, Office of Hematology and Oncology Products, Center for Drug Evaluation and Research, FDA
  • Doug Figg, Pharm.D., MBA, Section Head, Molecular Pharmacology Section and Clinical Pharmacology Program, Medical Oncology Branch, NCI-CCR
  • Brad Fackler, MBA, Co-Founder and Principal, Kinect Point, LLC

Application process:

Please submit a 1-page research abstract that includes a proposal detailing how collaboration with one of NCAT's preclinical groups could advance your current research project. The groups to which you may apply are: 1) Medicinal Chemistry, 2) Biology, 3) Pharmacokinetics, or 4) Molecular Probes. OITE will select 40 fellows to participate (ten per group), though it is our hope that, eventually, all interested applicants will have the opportunity to participate in future sessions.

Please note that a successful application does not indicate that you will perform a collaborative project with NCATS.  However, your proposal may be used as a platform for discussion during the NCATS visit; which could potentially lead to opportunities in the future.

Applications are due 6 weeks before the start of the workshop: Friday, July 20, 2012.

If you are a NIH Intramural fellow or graduate student at RML, NIEHS, Phoenix, Detroit, or Framingham the OITE will provide limited travel money.  To be considered for travel funding, please submit a 100 word description of how participating in the TSTP would influence your career to Philip Wang at wangph@mail.nih.gov by July 20, 2012 at 5 pm.  In your description, please indicate how long you have been at the NIH.

For more information on the TSTP program, please contact Phil Ryan or Philip Wang.

Program working groups

Medicinal Chemistry

Historically, medicinal chemistry dealt with the isolation and characterization of natural products from microorganisms and plants for medicinal use. In recent years, it has evolved into the application of sophisticated modern principles of organic synthesis to the design and optimization of pharmaceuticals. The medicinal chemist works in close collaboration with geneticists, biochemists, biologists, and pharmaceutical scientists in synthesizing new molecular entities (NME) or new chemical entities (NCE) with optimal drug action in cellular systems and animal models of the disease, for eventual clinical testing. The design involves building appropriate physicochemical properties of the NMEs/NCEs to ensure optimal oral delivery and pharmacokinetic and pharmacodynamic properties.

Drug Discovery Biology

Biology of normal and disease states in cells, tissues, and organs are of critical importance in understanding the pathology and in developing hypotheses for treatment modalities. These studies involve the identification of the genetic basis of diseases, biochemical targets, pathways, and epigenetics that control the cellular physiology that leads to the development and progression of diseases. It also extends to the understanding of the biology of infectious agents and their interaction with mammalian host cells and tissues that promote not only lethal infections, but also other cellular abnormalities, e.g. HIV. Knowledge gained through basic biological studies lead to the application of medicinal chemistry, screening technologies, and pharmacology to design therapeutic agents for further testing. The integration of multiple research areas in a highly collaborative model defines the discipline of translational sciences in drug discovery.

Pharmacokinetics and Pharmacodynamics

Once pharmacologically active agents have been identified through in-vitro testing, they must be tested in animal models in order to demonstrate safety and efficacy. These tests are critical to advancing molecules to the clinic for human testing. Pharmacokinetics is "what the body does to the drugs," in terms of their absorption into the blood stream or tissues following oral or intravenous administration, distribution to various tissues, metabolism in tissues and organs, and finally, excretion from the body. Pharmacodynamics is what "drugs do to the body," or simply, the action of drugs on living tissues and organs in an organism. For a drug to be efficacious, the agent has to be appropriately absorbed into the body and be distributed to the diseased tissue. At the same time, the drug should be appropriately metabolized and excreted to ensure safety and to minimize toxicity.

Molecular Probes

Molecular probes are well-defined, small drug-like molecules that interact with cellular targets and pathways and that alter cellular biology and physiology. Targets that are in cellular signal transduction pathways that can be modulated by these probes include enzymes, receptors, signaling proteins, lipoproteins, glycoproteins, DNA, RNA, and other effector molecules in the cellular milieu. Probe molecules are discovered by screening small molecule libraries in biochemical and cell-based assays specifically designed to interrogate disease pathways. The automated parallel chemical synthesis that was utilized in the late 1980s and early 1990s accelerated the development of automated High Throughput Screening (HTS) technologies that became the standard drug discovery tools in the pharmaceutical industry in this century. With the sequencing of the human genome in 2001, the NIH Molecular Libraries Initiative (MLI) was mandated in 2003 to bring advanced technologies and expertise to academic researchers who lacked the means to perform high-throughput screens and follow-up medicinal chemistry efforts as a means of identifying and optimizing small molecule probes of novel, unexplored cellular targets. These reagents offer the research community much-needed proof-of-concept pharmacological tools that may serve as starting points for therapeutic development into clinical agents.