Pediatric Chordoma Initiative

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’ve invested in an array of complementary research projects. As a result, we've:

• Developed and validated 12 pediatric chordoma disease models, and conducted the first deep characterization of pediatric chordoma biology.
• Tested numerous treatment concepts in these models to assess the activity of drugs or drug combinations that have shown promise in adult chordoma models. This includes screening all approved drugs (≈ 6,000) against pediatric cell lines.
• Learned more about the natural history of chordoma through our partnership with the National Cancer Institute (NCI), where scientists are collecting patient data to better understand how chordoma develops and grows over time, and determine how patients respond to various treatments.
• Connected families to teams of experts through the NCI’s annual pediatric chordoma clinic.
• Created pediatric-specific resources and provided personalized support to families to help children get the best care and outcomes possible.

Today, our goal is to build on these strong initial advances to make additional progress against pediatric chordoma, with the goal of catalyzing clinical trials that include this subset of our community.

Our current priorities include:

• Collecting additional pediatric chordoma tumor samples. Studying tumor samples is the primary way in which researchers generate ideas about new ways to treat cancer, and a lack of pediatric chordoma samples represents the biggest barrier to advancing this research.
• Developing and characterizing new pediatric disease models. While tumor samples facilitate the generation of new ideas for how to treat pediatric chordoma, cell lines and mouse models are critical to our ability to test these ideas. We’ll build on our successful model-development capabilities to establish, validate, and analyze additional models that more fully reflect the heterogeneity of the pediatric chordoma population.
• Testing drugs against 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. We’ll include pediatric chordoma models in ongoing drug screening experiments, whose pace is accelerating thanks to Chordoma Foundation Labs. The data generated will be critical for determining the best treatment approaches for children and designing and justifying new clinical trials.
• Determining whether pediatric chordomas differ from adult chordomas. Researchers are working to create multi-layered maps (“multi-omic” analyses) of the various aspects of chordoma tumor biology in order to understand drivers of this disease. By including pediatric samples in these studies, we’ll finally be able to answer the important question of whether pediatric chordoma is biologically distinct from chordomas in adults, and potentially uncover unique vulnerabilities to target.
• Studying the natural history of pediatric chordoma. Our partnership with the NCI has led to chordoma being among the first cancers to be extensively studied through their Natural History Study of Rare Solid Tumors. We’ll promote and facilitate additional pediatric patient participation in this study, which will lead to a much more detailed picture of pediatric chordoma, and ultimately to more personalized care.