·        Experimental therapeutics is the application of scientific rigor to the design and conduct of clinical trials.  The objective of the experimental therapeutics program is to provide a home for physician-scientists engaged in performance of clinical trials regardless of the funding source of these trials and to develop a training program in this discipline as applied to pediatric disorders.

·        The experimental therapeutics  program would explicitly include all clinical trials conducted by CMRC -affiliated investigators. These clinical trials may be investigator initiated or not.  Sponsors may include the NIH, pharmaceutical industry, philanthropic  or other organizations.  It is the goal of this program to expand the breadth of studies conducted at CMCR and to assure the highest scientific standard for all clinical research.

·        All physician-scientists should be full members of CMRC with the rights and privileges associated with CMRC membership.

·        The experimental therapeutics program will complement the core programs and centers of CMRC with respect to its translational function.

·        As part of this program we plan to develop an NIH funded training program in pediatric experimental therapeutics. This program would train investigators in the experimental and biostatistical methodology for the design, conduct, and analysis of therapeutic trials related to pediatric disorders.   There will also be an emphasis on research ethics and the protection of human subjects.

·        The establishment of a program in experimental therapeutics will provide much needed infrastructure for clinical trials.  This will build on the current infrastructure provided by the NIH sponsored General Clinical Research Center (GCRC).  This program will be an important asset for the CMRC translational medicine initiative by serving as a vital bridge between drug discovery and development in our laboratories and the needs of our pediatric patient populations.  This program will encourage the development of multi-center patient cohorts in pediatric sub-specialty areas and the use of these well characterized cohorts to conduct therapeutic trials.   Over time the experimental therapeutics program will encourage collaborations between investigators and provide multiple funding opportunities from diverse sources.

·        This program will allow investigators to develop and evaluate treatment and prevention measures for pediatric disorders and adult onset disorders with childhood antecedents, such as stroke and epilepsy.  By giving our patients and other children an opportunity to participate in clinical trials of novel or modified therapies for a range of serious childhood disorders, we ultimately will be able to give all children the benefit of new, better and less toxic medications.  As scientists reveal new information leading to innovations in detecting and treating diseases from cancer to cerebral palsy, we will be able to test these promising new treatments and ensure they reach those who most need them.