Fibrolamellar patient participation requested in National Cancer Institute (NCI) study

Fibrolamellar patients are invited and encouraged to participate in the Natural History Study of Rare Solid Tumors, currently underway at the National Cancer Institute (NCI) in Bethesda, Maryland. The purpose of this study is to better understand how tumors develop and grow. To do this, the study collects information, data, and tissue samples from patients with rare solid tumors, such as fibrolamellar. Natural history studies like this will help researchers learn more about fibrolamellar and other rare cancers and help shape the design and development of clinical trials to test new treatment options.

Unlike standard clinical trials, natural history studies do not test the safety or efficacy of new therapies. Instead, they compile information from patients and their medical records to better understand the “history” of a disease over time. Researchers use natural history study results to identify disease trends and get new ideas on how to treat rare cancers. Patients who are involved in these studies can help accelerate the development of new treatments by giving researchers critical information they need to make progress.

This study is sponsored by NCI’s MyPART (My Pediatric and Adult Rare Tumors Network), a group that drives research across a range of pediatric, adolescent, and young adult rare tumors.  MyPART and its Natural History Study of Rare Solid Tumors are a core part of NCI’s “Cancer Moonshot” portfolio. In 2016, President Obama launched the national “Moonshot” initiative to accelerate cancer research efforts and eliminate barriers to progress by broadening access to important data, and facilitating collaboration between researchers, patients, advocacy organizations and pharmaceutical companies.  To fund these efforts, Congress passed the 21st Century Cures Act in December 2016, authorizing $1.8 billion in funding over 7 years, including $195 million funding for the NCI in fiscal year 2020.  Participation of fibro patients in this study creates a valuable opportunity to focus some of that sizeable “Moonshot” research effort on fibrolamellar.

Importantly, given the current realities of COVID-19 and social distancing, patients can participate in this natural history study from home without needing to travel. As an additional benefit, participating patients and their doctors will be notified by the NCI research team if any clinically actionable mutations associated with their disease are discovered in the study’s genetic testing process.

To learn more about this opportunity to contribute to research, click here to view additional details about the study and a series of frequently asked questions.