Rare Disease Day at NIH was a virtual event held March 1^st to highlight rare disease research and share perspectives and stories from rare disease stakeholders. MyPART Executive Director, Dr. Abby Sandler, hosted a panel on rare cancers alongside several MyPART advocacy partner leaders and an oncologist who works with advocates on his rare cancer clinical trials.

Panelists shared their personal stories and how they became involved in rare cancer advocacy. Lennie Woods, Co-Founder and Executive Director of Sara’s Cure, and Jeanne Whiting, Co-Founder of the Desmoid Tumor Research Foundation, explained how their advocacy organizations literally began with a couple of people sitting around the kitchen table or the living room floor desperate to make a difference. “I was eager to do anything in my power to help,” Josh Sommer, Co-Founder and Executive Director of the Chordoma Foundation explains as he shared his story of joining a research lab to study chordoma after being diagnosed with the rare tumor as a college freshman. Panelists also discussed the successes of their advocacy organizations, which included an array of accomplishments, such as the establishment of virtual tumor boards to help inform patients of treatment options, the creation of patient registries, and the launch of new clinical trials. Dr. Eric Sherman, an oncologist from Memorial Sloan Kettering Cancer session, shared his own experience working with advocacy foundations on clinical trials for rare types of thyroid cancer. He stressed the important role of connections between oncologists and advocates in helping these trials meet their accrual goals, which is very challenging when trials focus on rare cancers.  

Panelists also discussed the challenges that come with rare cancer research, such as the lack of available tumor tissue for study and the existence of silos – different groups working on the same problems independently, instead of joining forces. Other panelists highlighted the challenge of balancing resources between growing their advocacy organizations and supporting research efforts. In the case of clear cell sarcoma, the focus of Lennie Woods’s organization Sara’s Cure, this tumor didn’t even have an ICD code when Lennie began her efforts. Now, there are three clinical trials studying the efficacy of different drugs in treating clear cell sarcoma.

While each panelist’s organization had a different rare cancer focus, they all shared the perspective that success in the rare cancer research and advocacy space comes from making connections with others and pushing through obstacles to make progress. “It’s a marathon, not a sprint,” Josh Sommer explained, but along the way, they found opportunities to connect with patients and other rare cancer stakeholders, which helped advance their missions of supporting patients and finding potential treatments. “It’s so important patients feel they are not fighting this alone,” Jeanne Whiting, Co-Founder of the Desmoid Tumor Research Foundation stated.

See below for the list of panelists and to learn more about their organizations:

• Lennie Woods, Co-Founder and Executive Director, Sara’s Cure
• Jim Palma, Executive Director, TargetCancer Foundation
• Jeanne Whiting, Co-Founder, Desmoid Tumor Research Foundation
• Timothy Babich, Co-Founder and Director, the RUNX1 Research Program
• Josh Sommer, Co-Founder and Executive Director, Chordoma Foundation
• Eric J. Sherman, M.D., Medical Oncologist, Memorial Sloan Kettering Cancer Center

Visit our Twitter account, @NCI_CCR_PedOnc, to read the live tweets from the day and visit the Rare Disease Day at NIH website to learn more about the event.

The Phase I study Combination Nilotinib and Paclitaxel in Adults with Relapsed Solid Tumors is a clinical trial for patients with solid tumors that are no longer responding to treatment. The study is testing the safety of using two drugs, paclitaxel and nilotinib, to treat solid tumors. In pre-clinical studies, nilotinib was shown to have greater anti-tumor activity when used in combination with paclitaxel. This trial is now open to children and young adults ages 12 years and older. To be eligible to participate in this study, patients must have completed any radiation or surgery treatments at least 3 weeks prior to starting this study. To enroll patients ages 12-17, please contact MyPART physician, Dr. Mary Frances Wedekind Malone, at maryfrances.wedekindmalone@nih.gov.

To learn about other rare solid tumor clinical trials, visit the MyPART web page on featured clinical trials here.

Dr. Liny John is a clinical fellow in pediatric hematology and oncology who works with the MyPART team on several research projects. Dr. John grew up in Maryland, rooting for the Baltimore Ravens. She stayed in Baltimore for her undergraduate education, earning a bachelor’s degree in Biomedical Engineering from Johns Hopkins University. After earning her master’s degree (also in Biomedical Engineering from Johns Hopkins), she moved to Pittsburgh, PA, where she completed medical school at the University of Pittsburgh Medical Center and started residency in pediatrics at Children’s Hospital of Pittsburgh.

She is currently finishing her last year of residency as Chief Resident at Children’s National Hospital. With her interest in rare solid tumors, she knew she wanted to spend the research-focused years of her residency with a mentor who shared her interests. She explains, “As soon as I met Dr. Brigitte Widemann, I knew I had found my perfect match. I wanted to be involved with clinical trials related to pediatric solid tumors and as she described the work [being done by] MyPART, I knew I would be joining a team that was a part of cutting edge and exciting research!” Her research projects are focused on learning more about the natural history of chordoma, rare bone tumors that grow in the skull base and spine. She is particularly interested in poorly differentiated chordoma and dedifferentiated chordoma, which are two rare sub-types of chordoma that are more commonly found in pediatric populations. The ultimate goal of her research is to develop novel clinical trials to test new and improved treatments for chordoma. She also helps plan the annual Chordoma Clinic, which brings patients together with expert doctors and researchers.

When asked what she finds special about MyPART, she explains, “I love that this team [allows] patients, advocates, scientists and clinicians from across the globe to come together with a common goal of improving outcomes for patients with rare tumors. Through natural history studies, specialty clinics, new clinical trials, and so much more – so much has, and continues to be, accomplished.”

Learn more about the MyPART team here.

The Paula Takacs Foundation for Sarcoma Research is a non-profit organization committed to supporting sarcoma research and elevating sarcoma awareness. The organization was founded in 2010 by Paula Takacs in Charlotte, NC. During her treatment journey for sarcoma she realized she had to help her fellow patients access more and better treatments. It is that legacy of activism that the Foundation continues today.  In order to support the mission, the Foundation hosts a variety of events including the Sarcoma Stomp, one of the largest walk/run sarcoma events in the U.S. This year, the event will be held virtually on April 24^th, 2021. The Paula Takacs Foundation website also provides numerous family and patient resources to help with treatment navigation and emotional support. The MyPART Network is proud to partner with the Paula Takacs Foundation to improve the lives of people with sarcoma, and to advance sarcoma research and awareness.