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On May 3-4th, the fourth annual Pediatric and Young Adult Chordoma Clinic was held at the National Institutes of Health (NIH) Clinical Center in Bethesda, Maryland. Experts in chordoma, a rare bone cancer that arises in the spine or skull base, travelled from across the country to gather with NIH physicians and scientists, advocates from the Chordoma Foundation, and most importantly, patients and their families. The Pediatric and Young Adult Chordoma Clinic is one of several rare tumor specialty clinics held at the NIH Clinical Center. The last in-person clinic for chordoma was held in 2019, with virtual clinics held during the pandemic.
NIH’s rare tumor clinics, coordinated through the MyPART Network, are a unique opportunity made possible by the resources available at the NIH Clinical Center to bring a group of patients with the same rare tumor together with experts who treat and scientists who study that tumor. This allows physicians and researchers to learn from a group of patients, facilitating potential discoveries and allowing physicians to see more people with the same rare tumor in a short time than they would be able to see at their own individual institutions. Patients also receive treatment recommendations from the group of experts in their rare tumor. Importantly, rare tumor clinics also provide patients and families with the opportunity to meet others experiencing the same disease, which can have a profound effect for patients who are feeling alone in their journey.
The Pediatric and Young Adult Chordoma Clinic began with a scientific workshop on chordoma. Dan Freed, PhD, Head of Target Discovery and Translational Research at the Chordoma Foundation Labs, presented his talk, “Chordoma Preclinical Research Landscape” and discussed the lab’s efforts to test therapeutic effects of existing FDA approved and investigational drugs using different assays. In collaboration with Children’s Hospital of Philadelphia, over 250 tumor samples have been collected. The lab has developed cell lines, patient-derived xenograft (PDX) models, and genomics datasets with the intention to share these models and data with the research community. After experts listened to the current preclinical research efforts in chordoma, Gregory Cote, MD, PhD, medical oncologist and physician investigator at Massachusetts General Hospital, led a discussion on the latest clinical trials in chordoma. Because chordoma is so rare, creative clinical trial designs must be implemented to explore the safety and efficacy of potential therapeutics in a small number of patients. Experts discussed the details of current trials and posed questions on how to better conduct these trials.
Following the scientific workshop session, the clinical portion of the chordoma clinic began! Patients met with the NIH team and the external experts. The experts present at the clinic ranged in medical disciplines from neurosurgeons to radiation oncologists to medical and pediatric oncologists, all with an interest in chordoma. Each physician brought their own unique expertise and perspective, and collectively, they formed a multidisciplinary team to review patient cases and provide treatment and follow-up recommendations. In addition to seeing the expert physicians, patients and families had the opportunity to meet with NIH teams that specialize in pain, nutrition, psychosocial needs, genetics, and more. One young adult patient who attended the clinic reflected, “the fact that I remained in the same room, and the [experts] just all came to me, made me feel very attended to...[it was] such an amazing opportunity. I've never really met with a geneticist before or [had] a psychosocial or pain consult... So that was really cool.”
The first full day of the clinic came to a close with a small dinner hosted at The Children’s Inn by the Chordoma Foundation. Patients and families mingled with the NIH team, external experts, and Josh Sommer and Shannon Lozinsky from the Chordoma Foundation. At the end of the evening Josh Sommer, Chordoma Foundation Executive Director, gave a small speech to thank everyone for attending and to share his thoughts. He remarked, “For me, [the] overriding feeling I have here just is one of hope… If I think about where we've come since the last time we were here in 2019, the field has advanced by light years. I think [this] really happened because there's this critical mass of researchers and clinicians that have come together, and in particular, for pediatric [and] young adult chordomas… there's just no way you can generate that sort of critical mass at any given institution. So really, it requires an effort like this at the NCI to make it happen.”
The 2023 Pediatric and Young Adult Chordoma Clinic was a successful step in the direction of learning more about chordoma, prioritizing research goals, and developing improved standards of care. MyPART looks forward to our continued collaboration with the Chordoma Foundation to help patients with this rare tumor.
Learn more about the NIH rare tumor clinics coordinated by MyPART here.
 Left: A family hugs MyPART physician and PI of the Natural History Study of Rare Solid Tumors, Dr. Mary Frances Wedekind. Top Right: External experts and the NIH team gather to discuss the current state of clinical trials for chordoma at the Scientific Workshop on May 3rd. Bottom Right: External experts and NIH staff gather for a group photo in the courtyard of the NIH Clinical Center.
MyPART physician and POB Clinical Director, Dr. John Glod, ran into MyPART advocacy partner, Dr. Corrie Painter, at the 2023 Rare Cancer Research Symposium, hosted by MD Anderson and the Broad Institute on May 4, 2023, in Houston, TX. Dr. Corrie Painter is President of Angiosarcoma Awareness Inc., a patient advocacy organization, and previously led the Angiosarcoma Project, an effort to learn more about angiosarcoma through collecting tumor samples, medical records, and patients’ experiences. The Angiosarcoma Project is part of Count Me In, an initiative from the Broad Institute to engage patients as partners in research.
Dr. John Glod (left) with Dr. Corrie Painter (right) at the Rare Cancer Research Symposium in Houston, Texas.
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MyPART Neuropsychologist, Dr. Robin Lockridge, featured by the Childhood Cancer Data Initiative: Dr. Lockridge (left) discusses why it's important to incorporate psychological data in addition to other clinical data collected by healthcare teams to better address the needs of children and young people with cancer. Read her feature here.
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MyPART postbac fellow Erika Kaschak presented a poster at the American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, IL on June 2-6th. Learn more about the 2023 ASCO meeting here.
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Recording available from FDA Oncology Center of Excellence: “Beginning the rare cancer journey: charting the best path to an accurate diagnosis.” MyPART co-leader and POB Chief, Dr. Brigitte Widemann participated in this panel, which included rare cancer survivors, to discuss the challenges in diagnosing rare cancers. Watch the recording here.
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Rare Cancer Research Funding opportunities through the DoD Rare Cancers Research Program: Three funding mechanisms are available and pre-application letters of intent deadlines range from late June to mid-July. Learn more about these funding opportunities here.
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