In mid-November, the MyPART team published an overview of results from the first 200 participants enrolled in our Natural History Study of Rare Solid Tumors. The paper, entitled “Longitudinal Natural History Study of Children and Adults with Rare Solid Tumors: Initial Results for First 200 Participants,” highlights lessons learned from an analysis of data collected thus far, and shows that a longitudinal study of people with rare solid tumors utilizing a remote, field cohort model is feasible. Anyone with a rare solid tumor or cancer predisposition syndrome can enroll in the study, regardless of diagnosis or treatment. This longitudinal, observational study collects a saliva sample, imaging data, medical records, individual and family medical history, pathology and tumor tissue from any previous biopsies or surgeries, and potentially blood (for patients who are invited to visit the NIH Clinical Center). All participants also provide in depth information about how having a rare solid tumor affects their life through patient-reported outcomes questionnaires.
Analysis of the first 200 patients showed that actionable mutations were found in 55% of patients. Clinically significant anxiety was found in 20-35% of adults. Most study participants did not enroll with a new diagnosis, but instead enrolled several years after diagnosis. Over 60% of participants had metastatic disease at the time of enrollment. Researchers can apply for access to the data, including sequencing data, through the NIH Database of Genotypes and Phenotypes (dbGaP) here.
As of October 2023, MyPART has enrolled over 550 participants in the Natural History Study of Rare Solid Tumors. We will continue to analyze and share the data as the study continues! Learn more about MyPART’s Natural History Study of Rare Solid Tumors here.
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The US Food and Drug Administration (FDA) approved nirogacestat for adults with desmoid tumors that are progressing and require systemic treatment. Desmoid tumors are aggressive tumors that form in the connective tissue throughout the body. While the tumors do not spread to other parts of the body, they can grow into surrounding tissues and organs. Surgery has historically been used to treat desmoid tumors, but there is a risk that the tumors will return. Because of this risk, systemic therapies have been the focus of clinical trials for desmoid tumors. Nirogacestat is the first FDA approved treatment for desmoid tumors. The phase 2 study of this treatment was originally tested through the NCI Rare Tumors Initiative as a pilot project. The Desmoid Tumor Research Foundation, a MyPART advocacy partner, has led the push for desmoid tumor treatments and was integral in the effort to reach nirogacestat's approval.
Learn more about the approval of nirogacestat for desmoid tumors here.
Members of the NCI Center for Cancer Research Pediatric Oncology Branch (POB) gathered to create a gingerbread masterpiece for the annual NIH Clinical Center Gingerbread House Contest. This year, POB ran with the theme of “Whoville by POB,” which highlights the many important research targets in childhood cancer research. Previous themes included the “POB CAR Factory” (2018), a play on the term for engineered T-cells and immunotherapy, and “One Breakthrough at a Time” (2019).
Gingerbread houses will be on display in the Clinical Center atrium from December 4th through January 3rd. View the gingerbread house creations on the NIH Clinical Center Facebook page starting December 6th here!
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Dec 7-8th: "Bridging the Gap: SMARCB1 Mutated Tumors – Rare Tumor Conference" hosted by Dr. Rajkumar Venkatramani from Texas Children’s Hospital and MyPART in Houston, TX. Learn more about the event here.
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Save the date! Upcoming Rare Tumor Clinics in 2024 at the NIH Clinical Center:
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Chordoma Clinic: May 8-9, 2024
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Pediatric and Wild-type GIST Clinic: June 12-13, 2024
- Applications for the GIST Clinic are now open! Learn more about the clinic and apply here.
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