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Support Resources
The MyPART website now has information on a variety of support resources for children and young adults with rare tumors. We also include resources for caregivers. Visit the new MyPART Support for Those with Rare Cancers webpage.
Natural History Study Enrollment
The Natural History Study of Rare Solid Tumors has enrolled its 100th patient! The study began enrolling patients with rare tumors, their family members, and people with genetic conditions at increased risk for rare tumors in late January 2019. This study will follow patients with rare solid tumors over time to learn how rare tumors develop, which can help researchers discover new treatments.
Our patients represent diverse rare tumor diagnoses and we’ve already enrolled patients with 20 distinct tumor types. For 11 of these tumor types, we have fewer than 2 patients each. To protect patient privacy, we group these 11 types together (labelled “other” in the figure below). So far, most patients are female. This is most likely because some of the tumors we study are more common in females. Our patients range in age, ethnicity, and race. We hope to see increasing diversity in our enrolled patients as enrollment increases. See the figure below for an overview of our patient enrollment.
Learn more about our Natural History Study of Rare Solid Tumors here.
  
The Fibrolamellar Cancer Foundation (FCF) was founded in 2008 when Tucker Davis was diagnosed with fibrolamellar carcinoma and decided to start an organization with his close friends with the goal of finding a cure. Fibrolamellar carcinoma is a type of rare cancer that grows in the liver of adolescents and young adults who have no prior history of liver disease. FCF has three main goals: find treatments and a cure, raise awareness, and connect the fibrolamellar community of patients and families. FCF recently hosted a research summit and invited researchers and MyPART staff to discuss priorities in fibrolamellar carcinoma research. Learn more about the Fibrolamellar Cancer Foundation here.
Next Advocacy Spotlight: National Leiomyosarcoma Foundation
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The Children’s Inn provides housing and support for pediatric patients and their families who are visiting the NIH to participate in clinical studies. The Inn regularly provides activities and events for their guests throughout the year. But during the holiday season, they go above and beyond to make sure patients and their families experience the festivity of the holiday season. Events range from trips to see the holiday lights in Washington DC to a holiday concert with Shelby Blondell. During the annual “Santa Ride Party,” members from the local police department dress as Santa and his elves and ride to the Inn on motorcycles with sirens blaring and lights flashing. The officers bring gifts and participate in activities with the children. One week of December is dedicated to “Winter Camp INNcredible,” the first time the Inn will provide camp activities for children in the afternoon during their winter break from school. The Inn also hosts holiday-themed movie nights and gingerbread house making. On Christmas Eve and Christmas morning, Children’s Inn Founder, Dr. Lori Wiener, and her family visit the Inn with Santa to make the holiday special for families who cannot go home. Families also enjoy a Christmas Day Brunch.
Learn more about the Children’s Inn and their support for families here.
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Selumetinib is a drug showing promise to reduce the size of tumors in pediatric patients with neurofibromatosis type 1 (NF1). NF1 is a genetic condition that can cause rare tumors called plexiform neurofibromas, which tend to grow around and along the nerves throughout childhood and adolescence. NCI Pediatric Oncology Branch Chief and MyPART leader, Dr. Brigitte Widemann, and her team have studied these rare tumors for many years through a natural history study, which enabled them to design clinical trials for selumetinib. Natural history studies follow individuals with a condition for a long period of time and can be challenging to coordinate. The NCI has the resources to successfully run these studies, which are important for studying rare tumors and can lead to the development of treatments.
Using a technique to precisely measure the volume of these oddly-shaped tumors, Dr. Widemann and her team were able to study the effect of selumetinib on plexiform neurofibromas. The team also studied patient reported outcomes, a term that refers to patients rating things that affect their quality of life, such as pain and weakness. The studies were conducted in collaboration with the pharmaceutical company AstraZeneca. Selumetinib reduced tumor size in almost 75% of patients. Importantly, the drug also reduced pain and improved strength in patients. These observations demonstrated that the effect of selumetinib is not only cosmetic; the drug improves quality of life. This is the first time a drug has shown any improvement in this tumor. In April 2019, the drug was granted “breakthrough” designation by the Food and Drug Administration (FDA), a designation awarded to drugs that show significant clinical benefit over current treatment options. Just recently, the New Drug Application for selumetinib was accepted by the FDA and given priority review. Pending its approval, selumetinib will be the first drug for the treatment of plexiform neurofibromas.
The story of selumetinib highlights the unique resources available at the NCI that make rare tumor research possible. In addition to carrying out exceptional basic and clinical research, we’re able to run natural history studies and undertake early phase clinical trials that are often difficult to execute. By partnering with outside organizations, we can push drug development forward and take treatments from the lab bench to the bedside of rare tumor patients. Learn more about the research that led to the development of selumetinib here.
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