To ensure that the youngest members of our community benefit from the rapid advances being made for chordoma – and cancer more generally – the Chordoma Foundation is making pediatric chordoma research a top priority.

The need for pediatric chordoma research

Pediatric chordoma patients aren’t simply miniature versions of their adult counterparts. For example, we know that pediatric and adult chordomas share some features, but the behavior of childhood chordomas suggests that their biology may differ in important ways. Understanding how pediatric chordoma is unique is critical for determining whether kids will benefit from the same therapies as adults or whether entirely distinct approaches are needed.

To answer these questions, accelerate the development of better treatments, and improve outcomes for this underserved patient population, we’re investing in high-impact research to:

Create pediatric chordoma disease models

Cell lines and mouse models are critical for cancer research. However, until recently, a lack of models representing pediatric chordoma meant that little could be done to understand the disease in this population or develop treatments. Initial investments in our Pediatric Initiative resulted in the world’s first pediatric chordoma cell lines and mouse models, which are already being put to work across an array of chordoma research projects. Going forward, more in-depth characterization of all currently available pediatric models is needed to increase their utility for evaluating new targets and therapies.

Test existing drugs in pediatric chordoma models

Identifying existing drugs that could work against pediatric chordoma offers the best chance of providing better treatment options in the near term. To that end, we’ve tested about a dozen of the most promising drugs and combinations identified by the research community in pediatric mouse models, including agents already known to be active in adult chordoma models. Some of these have shown signs of efficacy, providing evidence to support forthcoming clinical trials. But for some of these drugs follow-up studies are needed to confirm activity in additional models, optimize dosing, and/or identify biomarkers that predict response. And there is a constantly growing queue of new concepts that need to be tested. Additional funds will allow us to evaluate these drugs without delay and will shorten the timeline to determining whether existing drugs can help pediatric chordoma patients.

Understand the biology and potential targets of pediatric vs. adult chordomas

Work is underway in world-class research labs to study pediatric tumor samples from many angles in order to understand their biology and potential targets. For example, pediatric samples are being included in a study at Dana-Farber Cancer Institute looking at epigenetic regulatory genes commonly altered in chordoma, a project at University College London Cancer Institute to understand the link between gene dysregulation and chordoma, and an immunotherapy target discovery project at MD Anderson Cancer Center. With further investment, we’ll support research using the most powerful approaches available to explore differences and similarities between pediatric and adult chordoma, enabling us to determine whether targets and potential treatment options differ for pediatric patients.

Study the natural history of chordoma

Through our partnership with the National Cancer Institute, chordoma is among the first cancers to be extensively studied through their Natural History Study of Rare Solid Tumors. This initiative is collecting clinical and biologic information to better understand how chordoma develops and grows over time, and determine how patients respond to various treatments. This will lead to a much more detailed picture of the clinical course of pediatric chordoma, and ultimately to more personalized care.

Additional future priorities are to include pediatric chordoma in all of our immunotherapy projects and enable children to participate in chordoma clinical trials.

You can improve the odds for children with chordoma 

Initial progress in our Pediatric Initiative was made possible thanks to generous support from three families of young children with chordoma. New contributions will further accelerate the pace of pediatric chordoma research. To join us in ensuring that every child with chordoma benefits from research advances, contact development@chordoma.org.

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