Since the Natural History Study of Rare Solid Tumors began enrollment in January 2019, over 400 participants have joined the study in an effort to advance our knowledge about rare solid tumors. To highlight the study’s third anniversary of opening enrollment, we created a video discussing our research progress thus far. Importantly, MyPART’s progress in rare tumor research would not be possible without the participation of our patients and their families. In this anniversary video, we also share the unique perspectives from two young adult study participants. Learn about why they chose to participate in cancer research and how they have navigated their cancer journeys.

Watch the video here.

The new year brings an exciting new development for the Chordoma Foundation: the organization is launching their own laboratory space dedicated entirely to chordoma research! The lab will focus on translational research with the aim of discovering better treatments for chordoma. The lab will also allow the Chordoma Foundation to follow up on findings from their partners more quickly and easily than is currently possible. Josh Sommer, Co-founder and Executive Director of the Chordoma Foundation, states, “The idea is to allow our academic and industry partners to focus on what they are uniquely suited to do, while our central lab specializes in bridging common gaps along the process of rigorously evaluating therapeutic concepts.”

The lab is located in the Research Triangle area of North Carolina and led by Dr. Dan Freed, the Head of Target Discovery and Translational Research for the Chordoma Foundation. Josh adds, “We’re excited about the possibilities [the lab] will create to more rapidly translate biologic insights into new treatments for chordoma – and, based on the lessons we learn along the way, the case study it might provide for other cancers as well.” The foundation has already hired its first full-time lab position. We look forward to learning about the research developments that come out of the Chordoma Foundation’s new laboratory and are excited to continue our partnership as we head into the new year.

Learn more about the Foundation’s new laboratory space and their key highlights from 2021 here.

Dr. Pam Wolters is a licensed psychologist and co-directs the Behavioral Health Core in the Pediatric Oncology Branch (POB). Dr. Wolters is also the director of the Health Psychology and Neurobehavioral Research Program in POB. In her role with MyPART, she works with MyPART psychologist Dr. Robin Lockridge to develop psychological studies and clinical outcomes of interest for the Natural History Study of Rare Solid Tumors. Dr. Wolters adds, “I am particularly committed to developing and implementing outcome measures for children to be sure that we hear their voices in research, which are often overlooked. As an example, I was integrally involved in the pediatric trial of selumetinib for children with neurofibromatosis type 1 where my team and I led the patient-reported outcome assessments. These outcomes helped to document improvements in pain and quality of life and demonstrate clinical benefit, which contributed to FDA approval of the first drug for children with NF1 and plexiform neurofibroma tumors.”

Dr. Wolters came to the NCI as a doctoral student in POB in 1988. She conducted neuropsychological assessments for children being treated for acute lymphoblastic leukemia and HIV infection on clinical trials to better understand the effects of the disease and treatments on their brain functioning. She says, “I learned so much from my mentors about clinical trial research and loved working as part of multidisciplinary teams that were so devoted to the care and well-being of children with chronic and life-threatening medical conditions... One study led to another and here I am still enjoying my various roles in conducting clinical research at the NIH over 30 years later!”

Today, her work focuses on evaluating neurocognitive and patient-reported outcomes as part of clinical trials and natural history studies to better understand the relationship of these outcomes to the course of disease, environmental factors, and psychological factors. Dr. Wolters explains, “I also strive to develop new assessment tools and use novel technologies to monitor changes in studies over time. I feel that it is extremely important to capture the symptoms and daily functioning of patients, including children, and to ensure that these patient outcomes are an integral part of clinical research studies and publications.” When asked about her favorite part of MyPART, Dr. Wolters says, “I think that one of the most special parts of the MyPART network is the partnership with patients and advocacy groups and their involvement in the research process. Studies are significantly improved when we obtain input from patients on everything from their needs, what outcomes are important, ways to make research more feasible, and the implications of the results from their perspectives.”

Learn more about the MyPART team here.

• The 2022 Pediatric and Wild Type GIST Clinic will be held in person on September 14-16^th at the NIH Clinical Center in Bethesda, Maryland. Fill out an application here.
• The NIH RASopathy study, coordinated through the NCI Division of Cancer Epidemiology and Genetics (DCEG) is now open and aims to learn more about the development of tumors in people with RASopathies, a group of syndromes caused by changes in the genes involved in the Ras/mitogen-activated protein kinase (Ras/MAPK) pathway. POB Chief and MyPART leader Dr. Brigitte Widemann and POB clinician-researcher Dr. Andrea Gross are part of the study team. Learn more about the study here.
• Pediatric Oncology Branch clinician-researcher, Dr. Jack Shern, participated in the 2022 NF Congressional Briefing on Jan 7^th and presented his talk “New tools for early cancer detection in patients with Neurofibromatosis Type 1.”
• Pediatric Oncology Branch researcher Taylor Sundby participated in the Children’s Tumor Foundation virtual patient led panel discussion about MPNST biomarkers on Jan 27^th.
• New publication by Pediatric Oncology Branch clinician-researcher, Dr. Rosandra Kaplan, out now: “Targeting tumor microenvironment and metastasis in children with solid tumors.”
• Global Genes Rare Compassion Program is a program open to rare disease patients and families and medical students. Patients and families are matched with several medical students to share their perspectives on having a rare disease. Applications (for patients/families and medical students) are open through February 25^th.