This proposal constitutes the renewal application of the Pediatric Oncology Clinical Research Training Program (POCRTP), developed by Baylor College of Medicine's Texas Children's Cancer Center. This training program is predicated on the premise that the development of pediatric oncologists and pediatric oncology nurses who are highly trained in clinical research is essential for continued progress to be made in the treatment of childhood cancer. This renewal application highlights the impressive academic accomplishments and progress of the individual Scholars recruited during the last ten years. The program is structured to provide a formal, comprehensive, multidisciplinary clinical research educational program that trains (1) pediatricians who have completed a three-year pediatric hematology-oncology fellowship training program and are board eligible or certified in that subspecialty and, (2) Ph.D. nurses with a career interest in pediatric oncology clinical research. Trainees, in this "one-track" program, are designated "Paul Calabresi Scholars". They receive a two to three year comprehensive, didactic mentored training experience in the design, implementation, conduct and analysis of clinical research trials. The program emphasizes training in therapeutic research that occurs in team research settings in which basic and clinical scientists interact to expedite the translation of basic research discoveries into patient-oriented therapeutic cancer research. The program includes participation in a core didactic course in clinical investigation and trial design, provided through Baylor College of Medicine's Clinical Scientist Training Program, and an in-depth clinical research training experience focused in one of five specialized research areas, designated "Pathways", chosen by the trainee. The POCRTP Pathways include Clinical Pharmacology, Neuro-oncology, Cell and Gene Therapy, Hematologic Malignancy or Solid Tumors. The research experience within each Pathway is tailored to meet the individual Scholar's long-term research goals. All Scholars receive comprehensive mentorship by both a laboratory research and a clinical research mentor and receive in depth instruction in clinical trial design, statistical analysis, research ethics and regulatory requirements and guidelines. In addition, Scholars design and conduct clinical trials in their respective Pathways. An elective rotation at the National Cancer Institute's Cancer Therapy Evaluation Program is a feature of this program. The experience to date indicates that Scholars who complete this program are exceptionally well trained in clinical research, very successful in obtaining NIH funding and well positioned to become future leaders in pediatric oncology clinical research.

Public Health Relevance

This Pediatric Oncology Clinical Research Training Program provides a comprehensive clinical research educational program that trains pediatric oncologists and doctoral-trained pediatric oncology nurses in pediatric oncology clinical research. The program goal is to produce the next generation of leaders in the field of pediatric cancer research. The trainees, designated Paul Calabresi Scholars, receive a mentored training experience in the design, implementation, conduct and analysis of clinical research trials with the ultimate goal of translating basic research discoveries into innovative treatments that will improve the prognosis of children with cancer.

National Institute of Health (NIH)
National Cancer Institute (NCI)
Physician Scientist Award (Program) (PSA) (K12)
Project #
Application #
Study Section
Subcommittee G - Education (NCI)
Program Officer
Damico, Mark W
Project Start
Project End
Budget Start
Budget End
Support Year
Fiscal Year
Total Cost
Indirect Cost
Baylor College of Medicine
Schools of Medicine
United States
Zip Code
Simko, Stephen J; Tran, Huy D; Jones, Jeremy et al. (2014) Clofarabine salvage therapy in refractory multifocal histiocytic disorders, including Langerhans cell histiocytosis, juvenile xanthogranuloma and Rosai-Dorfman disease. Pediatr Blood Cancer 61:479-87
Simko, Stephen J; Garmezy, Benjamin; Abhyankar, Harshal et al. (2014) Differentiating skin-limited and multisystem Langerhans cell histiocytosis. J Pediatr 165:990-6
Heczey, Andras; Liu, Daofeng; Tian, Gengwen et al. (2014) Invariant NKT cells with chimeric antigen receptor provide a novel platform for safe and effective cancer immunotherapy. Blood 124:2824-33
Horton, Terzah M; Perentesis, John P; Gamis, Alan S et al. (2014) A Phase 2 study of bortezomib combined with either idarubicin/cytarabine or cytarabine/etoposide in children with relapsed, refractory or secondary acute myeloid leukemia: a report from the Children's Oncology Group. Pediatr Blood Cancer 61:1754-60
Gramatges, Maria M; Liu, Qi; Yasui, Yutaka et al. (2014) Telomere content and risk of second malignant neoplasm in survivors of childhood cancer: a report from the Childhood Cancer Survivor Study. Clin Cancer Res 20:904-11
Berres, Marie-Luise; Lim, Karen Phaik Har; Peters, Tricia et al. (2014) BRAF-V600E expression in precursor versus differentiated dendritic cells defines clinically distinct LCH risk groups. J Exp Med 211:669-83
Muscal, Jodi A; Scorsone, Kathleen A; Zhang, Linna et al. (2013) Additive effects of vorinostat and MLN8237 in pediatric leukemia, medulloblastoma, and neuroblastoma cell lines. Invest New Drugs 31:39-45
Kilburn, Lindsay B; Kocak, Mehmet; Schaedeli Stark, Franziska et al. (2013) Phase I trial of capecitabine rapidly disintegrating tablets and concomitant radiation therapy in children with newly diagnosed brainstem gliomas and high-grade gliomas. Neuro Oncol 15:759-66
Muscal, Jodi A; Thompson, Patrick A; Horton, Terzah M et al. (2013) A phase I trial of vorinostat and bortezomib in children with refractory or recurrent solid tumors: a Children's Oncology Group phase I consortium study (ADVL0916). Pediatr Blood Cancer 60:390-5
Rabin, Karen R; Smith, Justin; Kozinetz, Claudia A (2012) Myelosuppression and infectious complications in children with Down syndrome and acute lymphoblastic leukemia. Pediatr Blood Cancer 58:633-5

Showing the most recent 10 out of 40 publications