Throughout the month of September, stakeholders in the pediatric oncology community take the opportunity to shine an even brighter spotlight on the needs of patients and families experiencing childhood cancer. Advocacy organizations hold events and encourage the community to engage in efforts to increase awareness, from the challenges of researching rare cancers, to the need for improved clinical trial design and new treatments.

The NCI Center for Cancer Research Pediatric Oncology Branch (POB) and the MyPART Network treat every day as if it’s a part of Childhood Cancer Awareness Month! The POB actively engages with childhood cancer stakeholders, including members of Congress and advocacy organizations, to communicate our research efforts and highlight the need for continued support to accelerate progress. MyPART partners with three childhood and young adult focused advocacy organizations: Joey’s Wings, Stupid Cancer, and Teen Cancer America. Through these partnerships, we exchange resources, hold meetings and workshops, and communicate research priorities to ensure we are meeting the needs of the childhood cancer community.

This month, POB is hosting members of the Alliance for Childhood Cancer, which includes a wide array of childhood cancer community stakeholders, from advocates to research organizations. MyPART will also host a meeting on TFE/Translocation Renal Cell Carcinoma in collaboration with Joey’s Wings and Cincinnati Children’s Hospital. In addition to meetings, several POB staff will be featured for their work in childhood cancer. MyPART’s own Dr. Mary Frances Wedekind is featured in the NCI Childhood Cancer Data Initiative (CCDI) “Community Stories.” POB investigators, Drs. Nirali Shah and Christine Heske, are featured in a HealthCast Podcast for Childhood Cancer Awareness Month.

Collaboration across research institutions, advocacy organizations, and healthcare providers is crucial, not just throughout Childhood Cancer Awareness Month, but every month, for accelerating progress in childhood cancer research and treatments. While activities throughout September help shine a spotlight on childhood cancer, POB and MyPART are dedicated to improving the lives of those touched by childhood cancer every day throughout the year. Learn more about POB here and access support resources for families experiencing childhood cancer here. Follow us on “X” at @NCI_CCR_PedOnc as we feature stories and resources throughout Childhood Cancer Awareness Month.

On August 2^nd, MyPART officially launched a Spanish version of the MyPART website! Each of our 39 rare tumor pages has been translated into Spanish, along with the support resources, featured clinical trials, and other rare tumor news and information featured on the English-language website. Further, the Spanish version of the MyPART Partners page links to the Spanish versions of our advocacy partners’ websites, where available. We hope that the Spanish version of the MyPART website will provide access to important rare tumor information and increase our reach to the Spanish-speaking rare cancer community.

View the MyPART website in Spanish here.

Anne Liu joined the MyPART Team as a postbaccalaureate fellow! Anne graduated from Carnegie Mellon University in 2023 with her BS in Neuroscience. She’s always loved science, and through previous research experience, found that she is specifically interested in pediatrics and oncology. As part of the MyPART Team, Anne will help with medical records for the Natural History Study of Rare Solid Tumors and help organize MyPART’s annual rare tumor clinics, which bring patients and families together with researchers and expert physicians here at the NIH Clinical Center. Anne is particularly interested in contributing to the ongoing research efforts in chordoma.

When asked about her favorite part of MyPART, Anne exclaimed, “I love the people! The supportive and collaborative atmosphere motivates me to work harder and encourages my learning. As MyPART is such a diverse team, I’m super excited to get to know everyone one by one.” In the future, Anne hopes to pursue her medical degree and work in global health. Welcome, Anne!

Learn more about the MyPART Team here.

On August 24^th, a joint virtual meeting hosted by the Foundation for the NIH (FNIH), the US Food and Drug Administration (FDA), and NCI was held to discuss opportunities for forming a public-private partnership with the goal of developing therapies for ultra-rare cancers. While no official definition for ultra-rare cancer exists, meeting participants used an incidence of ≤ 1 per 1 million people as the general definition for ultra-rare cancer. The meeting kicked off with remarks from Drs. Marc Theoret and Rick Pazdur of the FDA Oncology Center of Excellence (OCE) and the NCI Director, Dr. Monica Bertagnolli. Dr. Bertagnolli underscored the importance of representation of underrepresented groups in clinical research and to learn from every individual with a rare cancer. She highlighted how NCI and FDA are collaborating on the Clinical Trials Innovation Unit, launched in February 2023, to streamline clinical trial designs. Dr. Theoret highlighted the programs within the FDA OCE focusing on rare cancers and the need for novel clinical trial design strategies. Dr. Pazdur summarized that making progress in ultra-rare cancers will require understanding the underlying mechanisms of the cancer, developing a more flexible regulatory framework for clinical trials in rare cancers, and international collaboration. Dr. Stacey Adam, an Associate Vice-President at FNIH, outlined FNIH’s role to connect research leaders at NIH with experts in academia, biopharma, and non-profit organizations to accelerate biomedical breakthroughs, and presented the goals for the public-private partnership. The partnership aims to create a non-competitive “open-science” environment focusing on drug development for targets and in cancers that are not economically feasible for the private sector. By harnessing resources available across government, academia, and advocacy to a common goal, the partnership would lower the risks of the drug development process for all stakeholders, with the goal of having a first-in-human trial for at least one ultra-rare cancer therapy by the first two years of the partnership. Through establishing pilot-programs with ultra-rare cancer champions, the hope is that this collaboration will lead to the creation of drug development strategies that can be applied to other rare cancers. 

MyPART co-leader, Dr. Karlyne Reilly, presented a talk on the challenges of drug development for rare cancers and highlighted the existing resources at NCI and NIH that can be harnessed to accelerate progress. Dr. Martha Donoghue, Associate Director of the Rare Cancers Program in FDA OCE, described the incentives and programs currently available at FDA to support drug development for rare tumors. Jim Palma, Executive Director of the TargetCancer Foundation, a MyPART advocacy partner, presented on the patient and advocacy perspective of why this partnership is needed. He highlighted the need to bring patients and advocates to the table early on, not at the end of the clinical trial design process, to ensure a patient-centered approach that meets the needs of patients with rare cancers. Other speakers included representatives from NCI programs, the National Center for Advancing Translational Sciences (NCATS), and the pharmaceutical industry.

The virtual meeting concluded with over an hour of open discussion among the participants, with audience members able to ask their questions and provide their input. This virtual meeting is only the beginning of a collaborative effort across FNIH, FDA, NCI, and other stakeholders to advance therapies for ultra-rare cancers. MyPART looks forward to participating in future discussions and collaborations. Learn more about this virtual meeting here.