As part of the Chordoma Foundation’s commitment to ensuring that all chordoma patients benefit from advances in research, we are happy to announce the launch of a multi-year Pediatric Chordoma Initiative designed to accelerate the development of better treatments and improve outcomes for this underserved patient population.
During the first phase of the initiative, which will run through 2020, we plan to invest at least $1.5M to jumpstart pediatric chordoma research and tailor the Foundation’s educational and support services to meet the unique needs of pediatric patients and their families. Results from these initial research investments are expected to culminate in the launch of at least one clinical trial for pediatric patients, and will also help to guide subsequent investments in pediatric chordoma drug development.
Seeded with generous contributions totaling $1.3M from three families of young children with chordoma, we have started a Pediatric Fund at the Foundation to support this vital work. Together, these extraordinary gifts are helping us begin to level the playing field for pediatric chordoma research, with positive initial results.
The need for the Pediatric Chordoma Initiative
Making up fewer than ten percent of chordoma cases, pediatric chordomas pose a special set of challenges for research, for patient care, and for the families of whose children are affected. But while chordoma research, clinical care, and support services have advanced significantly in the past decade, progress for the pediatric subset of the patient population has lagged. Consequently, among the seven new chordoma clinical trials planned or in progress, none includes children. Neither do recently developed chordoma treatment guidelines address pediatric chordoma.
The reasons for these disparities are multiple. Most importantly, however, very little is known about the biology and behavior of chordoma in children and to what extent they are similar or different from adult chordomas. Though we know that adult and pediatric chordoma share certain common defining features – e.g., brachyury expression – there are hints that different biological processes may be at play in pediatric tumors. These differences could have important implications for treatment, including surgery, radiation and drug therapies. Thus, gaining a better understanding of pediatric chordoma is critical for guiding the development of better treatments.
Until now, however, research on pediatric chordoma has been hampered by many of the same barriers that once held back the broader chordoma research field: lack of available tumor tissue and disease models, lack of a clear research agenda, no critical mass of engaged researchers. We have seen over the past decade the progress that can be unleashed by overcoming these barriers, and, through the Pediatric Chordoma Initiative, we aim to utilize the infrastructure and know-how that we’ve developed to catalyze similar advances for this subset of our community.
Goals and focus of the Pediatric Chordoma Initiative
The first phase of the Pediatric Chordoma Initiative will focus on overcoming barriers to pediatric chordoma research and defining the molecular and immunologic differences between adult and pediatric chordomas. To that end, key aims of the first phase of the initiative include:
- Recruiting researchers to study pediatric chordoma
- Creating pediatric chordoma disease models, including cell lines and patient-derived xenograft (PDX) models, that researchers need to study the biology of the disease and test drugs
- Funding grants to study the biology of pediatric chordomas in comparison to adult chordomas
- Testing drugs in pediatric chordoma models through our Drug Screening Program
- Initiating clinical trials designed to include pediatric chordoma patients – at least one by 2020
Achieving these aims will position pediatric chordoma to benefit from advances being made for chordoma and cancer more generally. It should also help determine whether children with chordoma are likely to benefit from the same therapies as adults or whether altogether different treatment approaches are needed. As this knowledge is generated, it will help to point the way to subsequent priorities in drug discovery and/or clinical research.
Thanks to the initial contributions to the Pediatric Fund, we have already made significant headway and brought a new level of attention to pediatric chordoma.
Notably, during the recent International Chordoma Research Workshop we hosted a special breakout session focused on pediatric chordoma, which was chaired by Dr. Brigitte Widemann, Chief of the Pediatric Chordoma Branch at the National Cancer Institute. This session brought together more than 30 doctors and researchers to identify key unanswered questions and research priorities regarding pediatric chordoma.
Additionally, in 2017 we announced a new $25,000 prize for pediatric chordoma cell lines, which has already spurred several labs to begin work on this important challenge.
We’ve also invested a portion of the funds in developing the first-ever pediatric patient-derived xenograft (PDX) models of chordoma with tissue contributed through the Foundation’s Tumor Donation Program. Already, these investments have resulted in the creation and validation of several pediatric PDX models, two of which have been included in our Drug Screening Program and are being used to assess the activity of drugs or drug combinations that have shown promise in adult chordoma models.
In addition to inspiring movement in the research community, early investments as part of the Initiative have also enabled the launch of a new pediatric group within Chordoma Connections, our private, online community for patients and loved ones, and are supporting the development of additional pediatric-specific content on the CF Website. By working to ensure that parents and patients are able to connect and learn about the disease, we aim to create a future in which no family has to face chordoma alone.
Help improve the odds for children with chordoma
With additional contributions to the Pediatric Fund, we can further accelerate the pace of pediatric chordoma research and do more to help those who are facing the disease today. We invite you to join us in ensuring that every young person with chordoma is able to benefit from advances in research, and overcome their disease. To learn more or to make a contribution please contact firstname.lastname@example.org.