Abstract

The mission of the Pharmacology & Experimental Therapeutics Section (PETS) of NCI's Pediatric Oncology Branch (POB) is to develop more effective treatments for children and young adults with refractory cancers and genetic tumor predisposition syndromes (GTPS). A particular focus is on rare tumors with unmet need. Promising novel agents are studied in early clinical trials that evaluate toxicities, activity, pharmacokinetics, and pharmacodynamics. By leveraging unique NIH resources, I have applied the principles of drug development for refractory cancers to neurofibromatosis type 1 (NF1)-related tumors and built the nation's largest comprehensive NF1 clinical trials program directed at plexiform neurofibromas (PN) and malignant peripheral nerve sheath tumors (MPNST).

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Cancer Institute (NCI)
Type
Investigator-Initiated Intramural Research Projects (ZIA)
Project #
1ZIABC010801-11
Application #
9556368
Study Section
Project Start
Project End
Budget Start
Budget End
Support Year
11
Fiscal Year
2017
Total Cost
Indirect Cost

  Institution

Name
Basic Sciences
Department
Type
DUNS #
City
State
Country
Zip Code

  Publications

Gross, Andrea M; Singh, Gurbani; Akshintala, Srivandana et al. (2018) Association of Plexiform Neurofibroma Volume Changes and Development of Clinical Morbidities in Neurofibromatosis 1. Neuro Oncol :
Blakeley, Jaishri O; Wolkenstein, Pierre; Widemann, Brigitte C et al. (2018) Creating a comprehensive research strategy for cutaneous neurofibromas. Neurology 91:S1-S4
Higham, Christine S; Dombi, Eva; Rogiers, Aljosja et al. (2018) The characteristics of 76 atypical neurofibromas as precursors to neurofibromatosis 1 associated malignant peripheral nerve sheath tumors. Neuro Oncol 20:818-825
Fisher, Michael J; Belzberg, Allan J; de Blank, Peter et al. (2018) 2016 Children's Tumor Foundation conference on neurofibromatosis type 1, neurofibromatosis type 2, and schwannomatosis. Am J Med Genet A 176:1258-1269
Cai, Wenli; Steinberg, Seth M; Bredella, Miriam A et al. (2018) Volumetric MRI Analysis of Plexiform Neurofibromas in Neurofibromatosis Type 1: Comparison of Two Methods. Acad Radiol 25:144-152
Cannon, Ashley; Jarnagin, Kurt; Korf, Bruce et al. (2018) Clinical trial design for cutaneous neurofibromas. Neurology 91:S31-S37
Allen, Taryn M; Struemph, Kari L; Toledo-Tamula, Mary Anne et al. (2018) The Relationship Between Heart Rate Variability, Psychological Flexibility, and Pain in Neurofibromatosis Type 1. Pain Pract 18:969-978
Gross, Andrea; Bishop, Rachel; Widemann, Brigitte C (2017) Selumetinib in Plexiform Neurofibromas. N Engl J Med 376:1195
Higham, Christine S; Steinberg, Seth M; Dombi, Eva et al. (2017) SARC006: Phase II Trial of Chemotherapy in Sporadic and Neurofibromatosis Type 1 Associated Chemotherapy-Naive Malignant Peripheral Nerve Sheath Tumors. Sarcoma 2017:8685638
Pemov, A; Li, H; Patidar, R et al. (2017) The primacy of NF1 loss as the driver of tumorigenesis in neurofibromatosis type 1-associated plexiform neurofibromas. Oncogene 36:3168-3177

Showing the most recent 10 out of 36 publications