The Childhood Cancer Data Initiative (CCDI), NCI’s collaborative effort to accelerate and improve data collection, sharing, and research in childhood cancer, launched the Molecular Characterization Initiative (MCI) in March 2022. MCI is a collaboration among NCI, the Children’s Oncology Group, researchers, oncologists, advocates, and children, adolescents, and young adults (AYAs) with cancer to provide state-of-the-art molecular testing to help inform treatment options and potential clinical trial participation. While this initiative shares some characteristics with the MyPART Natural History Study of Rare Solid Tumors, the MCI also has several distinct differences.

Both studies are dedicated to further our understanding of rare cancers and improve how we treat and care for patients. The primary difference is that the MCI provides state-of-the-art comprehensive molecular characterization at the time of diagnosis that helps participants and doctors select the best and most appropriate treatment while MyPART collects information and samples from people with rare tumors and their relatives, in addition to tracking their health history over a long period of time. Given these different purposes, both programs also differ in eligibility criteria and what participation entails. The MCI is currently open for children and AYAs 25 years of age or younger who have been newly diagnosed with a central nervous system tumor, soft tissue sarcoma, or rare tumor. MyPART similarly focuses on children and AYA patients, but anyone of any age and at any disease stage with a rare solid tumor can enroll in MyPART’s Natural History Study of Rare Solid Tumors. MyPART also enrolls family members of patients with a rare tumor if these patients also have a genetic tumor predisposition syndrome or the family has a medical history of people with rare tumors. Neither study enrolls patients with blood or immune system cancers.

Second, at this time, the MCI only enrolls patients who are seen at a Children’s Oncology Group (COG) affiliated health center. There are plans to continue to expand to children and AYAs outside of COG-affiliated hospitals, those with other childhood cancers, and those whose cancer has returned. MyPART can enroll patients from home and so enrolls patients from all over the world.

Further, the two programs collect genetic information from patients, but differ slightly in the data they collect and their protocol for returning data to patients. The MCI strives to return molecular testing results to patients within 21 days of enrollment and sample submission (typically peripheral blood and tumor tissue) to quickly provide guidance in potential treatment options for the patients. MyPART’s Natural History Study collects saliva samples from participants via mail to conduct genetic testing. Both studies collect tumor tissue (tumor tissue is required for the MCI and is collected when available for MyPART), which allows researchers to compare a patient’s normal tissue to their tumor tissue to find changes in the tumor tissue that may have contributed to the development and growth of the tumor.

MyPART also performs detailed medical data extraction including, but not limited to, presentation, time to diagnosis, treatment details, pathology details, disease status.  Extraction is performed by the medical team with a medical and family history provided by the patients. MyPART also collects extensive patient reported outcomes that cover multiple domains and include pediatrics, adults, and caregivers. Additionally, the MyPART team may invite participants to the NIH Clinical Center for additional testing, which usually includes the collection of blood for immune cell profiling. MyPART also returns genetic testing results to patients, though the return of results may not be within a 21-day time frame. MyPART has a dedicated team of genetic counselors and other support staff to help walk patients and their families through the results of their genetic testing. Additionally, because MyPART’s study is a natural history study, this study plans to collect data longitudinally for a long period of time. Participants in the MCI are followed longitudinally through COG’s Project: EveryChild.

While MyPART and the MCI differ in some respects, they share important qualities. Both projects value data sharing and prioritize patient privacy. Information will be de-identified before the data are shared with researchers, meaning that researchers will not be able to connect an individual with the health information collected for the study. While data sharing holds the key for accelerating discoveries in childhood cancers, NCI puts patient privacy and security first.

Both studies also share NCI Pediatric Oncology Branch (POB) staff! POB Chief and co-leader of MyPART, Dr. Brigitte Widemann, is also part of CCDI leadership, sits on the CCDI Steering Committee, and co-leads the CCDI Engagement Committee. POB scientist Dr. Karlyne Reilly, co-leader of MyPART, participates in a CCDI Working Group, as do Dr. Jack Shern, a POB physician scientist who is part of the leadership of the MCI, and Dr. Mary Frances Wedekind Malone, the PI of MyPART’s Natural History Study of Rare Solid Tumors. MyPART Executive Director, Dr. Abby Sandler, sits on the CCDI Engagement Committee alongside POB scientist Dr. Troy McEachron.

Although different in terms of patient eligibility, data collection, and return of results, both MyPART and the MCI complement each other to provide comprehensive information on childhood cancers and rare tumors. Further, both efforts are committed to sharing data with the global cancer research community, which will accelerate progress in the development of treatments and help us learn more about why these tumors form.

Learn more about the CCDI Molecular Characterization Initiative here.

Learn more about MyPART’s Natural History Study of Rare Solid Tumors here.

Overview of Both Studies:

MyPART welcomes its newest advocacy partner, cureMEC: The Myoepithelial Carcinoma Project. cureMec was founded by Naomi Natale and Michael Casaus in 2022 after their 3-year-old son Sebastián was diagnosed with myoepithelial carcinoma (MEC), a rare and aggressive cancer, in 2021. cureMEC raises awareness and supports research on MEC. MEC, named after the myoepithelial cells that form the tumor, is usually found in the salivary glands but can also form in the skin and other soft tissues. This family-led organization strives to create a community for those experiencing MEC by sharing their story and providing access to information and resources about MEC. cureMEC aims to advance a patient-driven strategy to help create a database of clinical and molecular information on MEC to share with researchers and accelerate discoveries in MEC. The cureMEC website also hopes to encourage tissue donation to facilitate MEC research. To share a message of hope for Sebastián and spread awareness, cureMEC coordinates “Sebastián’s Song,” a request for people to record versions of two songs, “Oh Bright Light” and “Que Florezca la Luz,” to share with Sebastián and the MEC community. MyPART joins cureMEC in their mission to find treatments for rare tumors and improve the lives of young people with rare tumors.  “We’re thrilled to be partnering with MyPART to raise awareness and provide education about MEC," said Naomi Natale, co-founder of cureMEC. “We look forward to connecting other MEC patients and families with researchers and clinicians who are committed to our goal to develop effective therapies to rare cancers like MEC.”

Watch this short video introducing Sebastián and cureMEC: https://www.youtube.com/watch?v=kMuePKvtrFA

MyPART welcomes Erika Kaschak, a post-baccalaureate fellow through the NCI iCURE program, to the team! NCI’s iCURE program, which stands for Intramural Continuing Umbrella of Research Experiences, is a training program for qualified students and scientists from diverse backgrounds and aims to foster diversity in biomedical research. Erika says, “I chose to apply to iCURE because of its mission to diversify the next generation of biomedical researchers. As a first generation, woman college graduate, iCURE provided a supportive atmosphere where I matched with Dr. Widemann’s team in order to continue my research before medical school and contribute to scientific advances in medicine.”

Erika graduated from Notre Dame of Maryland University with her bachelor’s degree in Biology in May 2022. In her role as a post-baccalaureate fellow for MyPART, Erika says she will support the MyPART team and “utilize my unique skills in medical record extraction and laboratory protocol to maximize the efficiency of current projects and capabilities of publications within MyPART.” Erika explains her favorite part about joining the MyPART team is the collaborative environment and the ability to learn something new and different from every member of the team. She says, “I am already astounded by the supportive team framework and the accomplishments that were made possible through this network. I truly believe that MyPART is special because we choose to do what is hard in the hopes of improving the lives of others.” Welcome Erika!

Learn more about the MyPART team here.