TRANSLATIONAL SCIENCE TRAINING PROGRAM 2013

MARCH 18 – 19, 2013

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.

The program will be held over two full days, in a “boot camp” format, from 8am to 5pm each day.

**Attendees will be limited to NIH Postdoctoral Fellows and NIH Ph.D. Students.  While all fellows interested in a career in translational science would benefit from attending the boot camp, it will be most beneficial to late graduate students and early postdoctoral fellows. 

Participants will:

Gain insight into the drug discovery and development processes.
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. National Center for Advancing Translational Sciences) 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.

Read what past TSTP Fellows have to say about the program!

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 dedicated networking event with scientists and project leaders at NCATS.
 
Speakers:

Rajesh Ranganathan, Ph.D., Director of NINDS Office of Translational Research, NIH and Co-Developer and Director of 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
Brenda Gehrke, PhD., Pharmacologist, Division of Hematology Oncology Toxicology, OHOP, CDER, 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 proposal detailing how you could incorporate translational sciences into your current research project. Indicate which of the disciplines described below would be most relevant to your translational proposal (Medicinal Chemistry, Drug Discovery Biology/HTS Assay Development, Pharmacodynamics/Pharmacokinetics, Molecular Probes, Rare or Neglected Diseases). If your proposal is better suited to one of the ongoing programs at NCATS or DPI, feel free to include that information in your proposal as well (i.e. Tox21, RNAi, TRND).The proposal is an exercise to get you thinking about translational sciences.  OITE will select the best 30 applicants that will be grouped according to the discipline selected, to meet in small groups with an NCATS expert in that discipline. The NCATS scientists will not only give you feedback on the proposal submitted, but also provide assistance for how to think about and incorporate translational scientific approaches in your current and/or future research. To best use the time with these experts, we strongly suggest that you submit an original application tailored to translational sciences rather than submitting a recent abstract. The proposal is the only information we have to select the cohort that will be able to participate. This is not a grant/collaboration application, nor is it a request for NCATS scientists to do part of your project! This is an opportunity to discuss translational science with experts in the field and network, but not a guarantee that you will get to work with the NCATS scientists.

Applications are due 6 weeks before the start of the workshop: Friday, February 22, 2013.

For more information on the TSTP program, please contact Phil Ryan (ryanp@od.nih.gov) or Philip Wang (wangph@mail.nih.gov).

Participation is limited to Postdocs and Graduate Students.  Please submit your application here.

NCATS OVERVIEW
 
The new National Center for Advancing Translational Sciences (NCATS) at NIH was founded to develop innovative approaches to reduce, remove, or bypass costly and time-consuming bottlenecks in the translational research pipeline in an effort to speed the delivery of new drugs, diagnostics, and medical devices to patients resulting in tangible healthcare benefits to the public. To carry out its mission, NCATS has established an organizational structure that spans the traditional drug discovery and development pipeline including, but not limited to, basic disease research, assay development, lead identification and optimization, medicinal chemistry, and drug metabolism and pharmacokinetics by combining the Division of Preclinical Innovation (DPI) and Clinical Innovation (DCI). NCATS provides resources and expertise to support basic research through drug development and into the clinic. Additionally, DPI is home to several high-profile programs including the Molecular Libraries Program (MLPCN), Tox21, Trans-NIH RNAi Screening Facility, Therapeutics for Rare and Neglected Diseases (TRND) program, and Bridging Interventional Development Gaps (BrIDGs) Program. NCATS DPI has a portfolio of drug development projects that have matured and are ready for future commercialization partnership and collaborators.

DRUG DISCOVERY AND DEVELOPMENT DISCIPLINES

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 / High Throughput Screening Assay Development

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.
 
Rare and Neglected Diseases

Over 7000 diseases are known to result from genetic mutations, and many of them have a clearly defined gene or protein perturbation responsible for the disease. Despite this, only 300 of these diseases have therapies available to treat them due to the difficulty in targeting the gene/protein responsible, small patient populations, and high risk of investment by pharmaceutical companies. These diseases are often the focus of basic scientific research although very few are successfully treated in the clinic for a variety of reasons. Neglected diseases are similar but different; neglected diseases often have huge patient populations, but the populations reside in tropical regions and poor countries that lack the means to treat the disease with expensive therapies. Again, there is a high risk associated with the development of therapies to treat diseases rampant in the developing world due to the lack of return on investment. The scarcity of treatment options in both of these types of diseases leads to a huge need and gap in drug discovery.