Chordoma Foundation

Help researchers learn more about chordoma and improve treatment options by participating in NCI’s Natural History Study

The chordoma community is one of a number of rare cancer communities that has been invited to participate in the Natural History Study of Rare Solid Tumors, currently underway at the U.S. National Cancer Institute (NCI). The purpose of this important study is to collect clinical and biologic information including tumor tissue samples from patients with rare solid tumors, including chordoma, to understand better how these tumors develop and grow.

Help researchers better understand how chordoma tumors develop and grow

Patients who take part in natural history studies help speed up the development of new treatments by giving researchers the information they need to make progress. By sharing your medical records and tissue samples and answering questionnaires about your medical history and quality of life, you can help clinicians and scientists learn more about why and how chordoma progresses, ultimately aiding in the identification of new treatments to test. And because travel is not required, you can participate in this study from wherever you are in the world!

Unlike trials to test treatments, natural history studies don’t test the safety or efficacy of treatments for a disease. Instead, they compile information from a large number of patients to better understand — as the name implies — the “history” of a disease over a long period of time. This information provides a comprehensive, detailed understanding of the natural course of a disease from the time it forms in your body, to the time you first notice symptoms, and through every clinical stage after that. Researchers will use data from this natural history study to gain new insights into the biology of chordoma and develop treatments that are tailored to chordoma patients.

Why does this matter?

Knowledge of the natural history of chordoma is required to meaningfully assess the effect of treatments in clinical trials. In order to conclude that a new treatment benefits patients with chordoma one has to show that the treatment changes the natural history of chordoma. Designing adequate and well-controlled clinical trials requires a strong scientific foundation — a foundation built through natural history studies like this one.  

For example, the first effective treatment for another rare tumor called plexiform neurofibroma (PN), which occurs in children with neurofibromatosis type 1 (NF1), was identified after a natural history study like this one had characterized how PN formed and grew from childhood through young adulthood and what symptoms they cause. Learnings from this natural history study informed a meaningful clinical trial design of a new medicine, ultimately leading to FDA approval of this drug for children with PN. Dr. Brigitte Widemann, Chief of the Pediatric Oncology Branch at NCI’s Center for Cancer Research, spearheaded the effort in PN. 

Dr. Widemann and her team at NCI’s My Pediatric and Adult Rare Tumor (MyPART) Network recently received funding to build on their experience with PN and expand their work into other rare cancers such as chordoma. The MyPART team is now leading the Natural History Study of Rare Solid Tumors and we are thrilled to partner with them on including chordoma as one of the first cancers to be more extensively studied. Over the past several months, their team has consulted with dozens of leading chordoma specialists — including members of the Chordoma Foundation Medical Advisory Board (MAB) — to design the chordoma portion of the study, ensuring that the right data is collected to answer the most important questions about chordoma.

This represents a significant investment in chordoma by the NCI, and we are deeply grateful to Dr. Widemann and the MyPART team for applying their expertise and resources to chordoma and look forward to continuing to collaborate with them to accelerate understanding of our disease.

“Studies like these are crucial to our understanding of how rare cancers progress and in building the scientific foundation upon which drug development programs are formed. Any chordoma patient, including those in active treatment, is welcome to participate in the Natural History Study from the comfort of their home. They are also welcome to come to the NCI to meet with our experts directly.”

Dr. Brigitte C. Widemann

Chief of the Pediatric Oncology, NCI’s Center for Cancer Research

To learn more about the Natural History Study, visit cancer.gov/mypart.

To enroll, email the team at NCICCRRareTumorClinic@mail.nih.gov.



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