Scientific Publications and Presentations

NCI intramural program approach to rare tumors: Natural history study of rare solid tumors in children and adults: A longitudinal, comprehensive data and biospecimen collection protocol

June 21, 2023

Wedekind, Reilly, Del Rivero, Lockridge, Allen, Raygada, Bernstein, Thomas, Vivelo, ... , Sandler & Widemann
Journal article in Pediatric Blood & Cancer

Rare tumors contribute significantly to the burden of cancer in the United States, yet are often poorly understood and have inadequate research support. To address this situation, the National Cancer Institute’s (NCI) My Pediatric and Adult Rare Tumor Network (MyPART) was developed. MyPART’s goals include connecting participants and investigators through shared infrastructure and networks; accelerating understanding of rare tumors; and developing clinical trials for rare tumors through national/international collaborations of participants, advocates, clinicians, and researchers. A critical part of this effort, a natural history study is described in this paper.

Natural history studies are important for understanding the course of disease over time and can inform drug development. The primary objective of the Natural History and Biospecimen Acquisition Study for Children and Adults with Rare Solid Tumors Protocol (NHRSTP) is to evaluate the natural history of participants with rare tumors and genetic cancer predisposition syndromes. They also aim to evaluate biological relatives and individuals with genetic cancer predisposition syndromes to better understand the natural history and biological features of these conditions. As part of the study, tumor tissue and other samples collected from participants are stored for future analysis. The goal of the effort is to use this data to develop therapeutic interventions, screening guidelines, clinical trials, and patient-reported outcome measures to improve the lives of patients with rare tumors.

All of the clinical and laboratory evaluations performed in this study are funded and do not cost anything for the participants. Through this research, NCI MyPART hopes to provide better support and resources for patients with rare tumors and develop new clinical trials for these conditions.

The full article describing the plan and protocol for NHRSTP study can be read here.

Note: FCF is listed in the paper as an advocacy partner of NCI’s MyPART team.