Malignant Peripheral Nerve Sheath Tumors in Neurofibromatosis Type 1: A Multicenter Project With 3 Clinical Trials

摘要

A major goal of this CTDA proposal is to optimize NF1 subject recruitment into 3 clinical trials related to MPNSTs. This CTDA project has been successful in being directly responsible for the implementation of 1 of the 3 clinical trials. Our initial efforts led to the submission of a clinical trial for neoadjuvant chemotherapy in MPNST (DAMD-NF043129; PI-David Viskochil). This proposal was not funded however it was revised by Brigitte Widemann M.D. as principal investigator with a dedicated focus on treatment of MPNSTs within an oncology Consortium (Sarcoma Alliance for Research through Collaboration known as SARC). This revision entitled: Phase II Trial of neoadjuvant chemotherapy in sporadic and NF1-Associated high grade unresectable MPNSTs (Proposal No. NF050022; PI-Brigitte Widemann) was approved for funding through the 2005 DOD NF Program. A meeting between investigators in this MPNST CTDA project and members of an MPNST Committee of a newly formed NF1 Consortium (DoD contract No. W81XWH-05-1-615; PI-Jeannette Lee) and a representative of SARC was held in April 2006 which allowed for the transition of aims from another CTDA-derived clinical trial (Identification of Risk Factors for MPNST in NF1) to potential implementation of its goals into an MPNST trial conducted through the NF1 Consortium. Finally website development from the CTDA study is being implemented for the recruitment and the enrollment of NF1 patients with spine abnormalities into a multi-center natural history study (RO1 NS050509-O1A1; PI- D. Viskochil).