The new year marks the first birthday for the Natural History Study of Rare Solid Tumors! The study began enrolling patients in late January 2019. After only one year, the study has enrolled over 100 patients with over 10 different types of rare tumors. The Natural History Study began under a project funded by the Cancer Moonshot initiative to accelerate the discovery of cancer treatments by increasing patient engagement in research. This study, coordinated by the MyPART team, seeks to enroll pediatric and adult patients with rare solid tumors.

Natural history studies are an important tool to study rare diseases because they coordinate the collection of data from many people with the same rare disease. This allows scientists to find things these patients have in common, identify potential causes of the disease, and study the progression of the disease over time. When studying the effect of a new treatment, natural history study data can sometimes be used to compare to data from patients who receive an experimental treatment. These data can help determine the effect of potential treatments in a population where it may be difficult to enroll enough patients for a large clinical trial. Earlier this year, the FDA released draft guidance information for researchers about how to conduct natural history studies to aid the development of safe and effective treatments for rare diseases, further highlighting the important role that these studies play in understanding rare disease.

From the Natural History Study of Rare Solid Tumors, we hope to understand why rare tumors form and how they grow and progress. The data from this study will be shared with rare tumor researchers and clinicians all over the world to facilitate the development of new treatments and improve the care of patients with rare tumors. The Natural History Study of Rare Solid Tumors embodies the mission of MyPART: to connect rare tumor patients, families, caregivers, advocates, and researchers to support the development of rare tumor treatments for pediatric and young adult patients. Learn more about the Natural History Study of Rare Solid Tumors and how to participate here.

The National Leiomyosarcoma Foundation (NLMSF) was founded in 1997 as a resource for patients, families, and caregivers experiencing leiomyosarcoma (LMS) – a type of sarcoma that affects smooth muscle tissue. In addition to the main NLMSF website, the foundation hosts a research focused website and a website for emotional support, called “NLMSF Life Change.” NLMSF also hosts a monthly conference call to connect patients and caregivers with others affected by LMS. Launching on January 20th of this year is the NLMSF Patient-Caregiver Advocacy Network, which gives patients the opportunity to provide guidance for LMS patient care and research. In addition, NLMSF participates in the LMS Research Roundtable and Scientific Think Tank, which brings together researchers from across the globe to determine the priorities in LMS research and standardize guidelines for patient care. Learn more about NLMSF and the variety of resources they offer here.

Next Advocacy Spotlight: Desmoid Tumor Research Foundation

• January 29 – MyPART Team Retreat – Bethesda, MD. The morning session of the retreat is open to the public and will highlight MyPART research and programs. 
• February 11 – The Children’s Inn Congressional Networking Reception – Washington, DC. Staff from the Pediatric Oncology Branch and MyPART will attend this annual reception hosted by The Children’s Inn to connect policy makers with patients, families, researchers, and staff from The Children’s Inn to highlight the importance of supporting child health.
• February 19-21 – NCI CCR Medullary Thyroid Carcinoma Clinic – Bethesda, MD. The second annual MTC clinic is hosted by the Pediatric Oncology Branch to serve patients and families affected by MTC.
• February 28 – Rare Disease Day at NIH – Bethesda, MD. Rare Disease Day is observed globally to raise awareness of rare diseases for policy makers and the public. Each year, the NIH Clinical Center and NCATS sponsor Rare Disease Day at NIH. This year, MyPART staff will help coordinate a clinical trials resource booth. The event is open to the public and video of the event will be live-streamed.