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Experimental Therapeutics
Children's has an extensive portfolio of funded clinical and experimental therapeutics research. Two focus areas of experimental therapeutics are neuromuscular disease and oncology.
The Cooperative International Neuromuscular Research Group (CINRG) based in the Research Center for Genetic Medicine is the largest neuromuscular clinical trial group worldwide (see www.cinrgresearch.org). Active clinical trials include more than 300 Duchenne muscular dystrophy patients enrolled into a series of funded drug trials and natural history studies, and clinical trials in Pompe disease, and limb-girdle muscular dystrophy. CINRG is integrated with basic and translational research in neuromuscular disease through a Wellstone Muscular Dystrophy Center, a Department of Defense Program Project, and a National Center for Medical Rehabilitation Research .
The Center is committed to the clinical investigation of new cytotoxic and biotherapeutic agents for the alternative treatment of cancer and actively pursues such trials in children with refractory disease. The leadership of Gregory H. Reaman, MD, as chair of the NIH-funded national cooperative clinical trials group, the Children’s Oncology Group (COG), with Nita L. Seibel, MD, as principal investigator for Children's and Anne L. Angiolillo, MD, as principal investigator for Phase I experimental therapeutics places Children's in a strategic position nationally, enabling full participation of members of the Center in novel national pediatric cancer clinical trials, as well as limited institutional studies. In addition, Roger J. Packer, MD, is the institutional principal investigator for the Pediatric Brain Tumor Consortium (PBTC), an NCI-funded phase I/II consortium of the 11 most active institutions involved in the treatment of childhood brain tumors. Children's has enrolled the second highest number of patients to PBTC clinical trials among all members of the PBTC. Finally, Stephan Ladisch, MD, a founder of the Histiocyte Society, the only international clinical trials group devoted to treatment of these rare diseases, remains active in the design of international histiocytosis treatment protocols.
The expanding knowledge of molecular mechanisms of oncogenesis using proteomics is resulting in the development of targeted small molecules that impact on signal transduction pathways, which are currently in ongoing or planned clinical trials. These include specific kinase inhibitors that interrupt cell cycle regulation or induce pro-apoptotic pathways. Additional novel agents inhibit micro-angiogenesis, preventing metastases as well as regulating the growth of primary tumors. A number of faculty members have made significant contributions to these clinical investigations, and to the important role of Children's National Medical Center in the Children’s Oncology Group and PBTC. They include Roger J. Packer, MD, Caterina P. Minniti, MD, Evelio D. Perez-Albuerne, MD, PhD, Naynesh R. Kamani, MD, Brett J. Loechelt, MD, Christopher Lawlor, MD, Anne L. Angiolillo, MD, Nita Siebel, MD, Michael Guerrera, MD, Tobey J. MacDonald, MD, and Brian R. Rood, MD.
Contact Information:
Max Coppes, MD, PhD
Children's Research Institute
Children's National Medical Center
111 Michigan Avenue, NW
Washington, DC 20010
202-476-3656
202-476-6014 fax
mcoppes@cnmc.org
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