Rare Tumor Workshops

We hold workshops in the Center for Cancer Research to help scientists learn what to study about rare cancer. During these workshops:

• Scientists come together to share their research.
• Advocates come to share issues that are important to patients.
• Clinicians come to share their experience treating the rare cancer.
• FDA representatives come to help researchers design experiments and clinical trials with the best chance of drug approval.

Workshops we have held in the past include:

• Chordoma
• Merkel Cell Carcinoma
• Malignant Peripheral Nerve Sheath Tumor (MPNST)

Chordoma Workshop

The NCI Chordoma Workshop was held on June 15, 2018. It was sponsored by the Center for Cancer Research and planned with the help of the Chordoma Foundation. Thirty-eight experts gathered from across the U.S. to discuss plans for a chordoma clinic in the NCI Center for Cancer Research. The group reviewed how chordoma presents in children and families and how it differs from chordoma in adults. Doctors from the Center for Cancer Research described how rare tumor clinics work. The experts discussed four topics:

• How to design clinical trials for chordoma
• New therapy options for chordoma
• Measuring quality of life in chordoma patients
• Molecular changes in chordoma and ways to test new treatments

As a result of these discussions, the group agreed to move forward with a Pediatric Chordoma Clinic, which was held in April 2019. The clinic was planned based on the suggestions of doctors, scientists, advocates, and FDA representatives at this workshop.

Merkel Cell Carcinoma Workshop

An international workshop on Merkel Cell Carcinoma was held in Rockville, MD on March 5-6, 2018. It was co-sponsored by the NCI Center for Cancer Research, National Institute of Arthritis and Musculoskeletal and Skin Diseases, and the University of Michigan. Fifty-two experts from the U.S. and four other countries gathered to:

• Find the most important research questions
• Build plans for research collaborations

Giovanna Tosato (NCI), Connie Trimble (Johns Hopkins University), and Bernie Fox (Oregon Health and Sciences University) gave keynote talks.

The attendees broke into smaller groups to discuss issues in Merkel cell carcinoma research. The three groups were:

• Basic research
• Translational research
• Clinical research

The results from the meeting were published for the scientific community in an article by Harms et al., 2018.

Malignant Peripheral Nerve Sheath Tumor (MPNST) Workshop

 “MPNST State of the Science: Outlining a Research Agenda for the Future,” was a workshop held in Bethesda, MD on October 6-7, 2016.

The goals of the workshop were to:

• Summarize findings on MPNST since the last consensus meeting in 2002
• Find barriers and knowledge gaps
• Find the best chances for future progress
• Make plans for working together

The workshop was co-sponsored by the Center of Cancer Research and the Division of Cancer Epidemiology and Genetics, NCI, and the Children’s Tumor Foundation. Before the workshop, the organizers wrote a summary of MPNST research to date and published it in an article by Kim et al., 2017. Fifty-six experts came from the U.S. and four other countries. Dr. Sasha Jumbe, from the Bill and Melinda Gates Foundation gave a keynote talk on solving health problems. Seven pathologists from around the country met the day before the workshop to look at tumor cases. They came to agreement on how to describe tumors changing from benign to malignant and published their findings for the scientific community in an article by Miettinen et al., 2017.

There were five topics that the experts focused on at the workshop:

• Clinical Trials Methodology
• Diagnosis/Imaging/Primary Management
• Genomics/Biomarkers
• Preclinical Models
• Pathology/Histology

The experts agreed on the top priorities for research in each of these areas. The group published their findings for the scientific community in an article by Reilly et al., 2017.