MyPART: My Pediatric and Adult Rare Tumor Network - Cures

Division Of Basic Sciences - Nci

Investigators

Linked publications & trials

Abstract

Through collaboration between MyPART, the Center for Cancer Research (CCR) Rare Tumor Initiative (RTI), and the CCR Pediatric Oncology Branch (POB), we have developed a rare tumor natural history and biospecimen trial to study rare solid tumors in pediatric, adolescent, and adult patients. The Natural History Study of Rare Solid Tumors (NCT03739827) has been open for enrollment since January 2019 and has enrolled more than 500 patients with a variety of very rare solid tumors. Patients can enroll by providing information from their home (field cohort) or by coming to the NIH Clinical Center to be seen by expert clinicians and other healthcare providers (clinic cohort) for a more extensive work up. Anyone with a rare solid tumor can participate in the field cohort and patients will be invited to come the NIH Clinical Center to join the clinic cohort based on the details of their medical history. The ability to enroll participants remotely allowed us to continue to remain active during the COVID pandemic where we were not in the position to bring most patients to the NIH Clinical Center for evaluation. Sub-protocols established under the Natural History Study allow the infrastructure of the master study to be used by both MyPART and non-MyPART investigators to carry out more in-depth studies of specific rare tumor types. A sub-protocol for chordoma has opened and has enrolled over 100 participants. Participants in the main study and sub-protocols are followed longitudinally over time. The protocol database is established and holds a wide range of information including patient reported outcomes, medical history, tumor specimens, imaging information, and clinical evaluations performed at the NIH Clinical Center. A research analysis pipeline for blood and tumor samples has been developed for nucleic acids, immune characterization, and cytokines. All information provided is reviewed and a clinical recommendation is given, when feasible. In addition, we have established a molecular tumor board and we are returning molecular tumor data to patients. Research tumor sequencing is ongoing. For patients who undergo surgery at the NIH Clinical Center for clinical reasons, we are comprehensively analyzing tumors including single cell sequencing and we are establishing preclinical models (patient derived xenografts and organoid lines). Our collaboration established with the NCI Developmental Therapeutics Clinic is active and we have 5 treatment trials opened, including a trial of the PD-L1 inhibitor atezolizumab that has opened for children and adolescents with the rare sarcoma alveolar soft part sarcoma. Based on results from this trial directed by Dr. Alice Chen the FDA recently granted approval of atezolizumab for patients 2 years and older with inoperable alveolar soft part sarcoma. In addition to histology-specific trials, the Developmental Therapeutics Clinic has early clinical trials open to patients with many different types of rare tumors. Several additional clinical trials for very rare tumors are in development. A phase II trial of a TIGIT antibody and a PD-L1 inhibitor for children and adults with SMARCB1- or SMARCA4-deficient tumors is open for enrollment. This trial is a collaboration between MyPART and the NCI-CTEP Pediatric Early Clinical Trials Network, with MyPART investigators serving as PIs and several NCI Center for Cancer Research (CCR) Investigators contributing to enrollment on 6 rare solid tumor strata. In 2019, 2020, 2022 and 2023 MyPART held specialty rare tumor clinics for patients with "wild-type" (SDH-deficient) GIST (gastrointestinal stromal tumor), medullary thyroid carcinoma, and pediatric chordoma. These clinics assemble clinicians, researchers, patients, families, and advocates from across the US and the world. Patients and families benefit from receiving medical opinions from world experts in their tumor while also having the opportunity to meet others with the same rare tumor diagnosis, often for the first time. Clinicians and researchers benefit from sharing their knowledge about a particular rare tumor, which has also led to increased collaboration between groups. Due to the COVID pandemic several clinics were held remotely, but in 2023 we have been back to in person clinics at the NIH Clinical Center. Additional rare tumor clinics are planned. MyPART has also made progress in pre-clinical rare tumor research. Work is ongoing to engineer a mouse model of SDH-deficient GIST that can be used to learn more about this rare tumor and test potential therapies. With its focus on patient engagement, MyPART has established a robust communications program. We have a website (www.cancer.gov/mypart) that receives over 60,000 unique visitors per month and provides information on a number of rare tumors, the Natural History Study of Rare Solid Tumors, support for patients and their caregivers, and links to additional resources. We also send out a monthly newsletter and have a Twitter account we share with the POB. MyPART has established partnerships with 22 advocacy organizations in order to build strong relationships with the rare tumor community, to learn what is important to them, and to provide education and information to rare tumor patients and their families about the importance of participating in rare tumor research to accelerate the development of new and effective therapies. We developed a manuscript on a landscape analysis of rare tumor efforts nationally and internationally. We recently published manuscripts highlighting our chordoma clinics and our approach to studying rare pediatric and adult solid tumors on our natural history study. A manuscript describing results for the first 200 study participants was just accepted for publication. MyPART efforts align well with the goals of the NCI Childhood Cancer Data Initiative (CCDI) and MyPART members are actively engaged with several CCDI working groups. We are in the early stages of working with the NCI CCDI on the development of a national CCDI coordinated strategy to study rare tumors.