On March 30^th, Drs. Brigitte Widemann and Andrea Gross were honored in a virtual ceremony hosted by the Clinical Research Forum. They received the Clinical Research Forum Top 10 Clinical Research Achievement Award for their work on the MEK inhibitor selumetinib. Selumetinib is the first FDA approved treatment for children (ages 2 and older) with the rare genetic condition neurofibromatosis type 1 (NF1). NF1 can cause the growth of plexiform neurofibromas, a complex type of tumor that grows along the nerves throughout the body and can cause pain and other physical challenges. Because these tumors cannot be completely removed with surgery, they are called inoperable plexiform neurofibromas.

Drs. Widemann and Gross conducted Phase I and II clinical trials of selumetinib in children with inoperable plexiform neurofibromas that were causing physical problems such as pain, disfigurement, and motor skill impairment. The study found that selumetinib reduced the size of tumors in the majority of patients, and often improved motor function, pain, and overall quality of life.

This clinical work is a breakthrough in the treatment of inoperable plexiform neurofibromas in children with NF1. Read more about this breakthrough here.

View a short video of Drs. Widemann and Gross discussing their work here.

Teens and young adults experiencing cancer face unique challenges. In an effort to increase access to support programs for teens and young adults, new resources have been added to the MyPART website. Resources include links to advocacy organizations dedicated to young adults with cancer, camp and adventure programs, support networks, educational resources, and more.  Resources and guidance on end-of-life decisions are also available.

Learn more about the unique challenges faced by teens and young adults with cancer here.

April 4-10^th is National AYA Cancer Awareness Week. Follow us at @NCI_CCR_PedOnc as we highlight the AYA experience and share resources to raise awareness for teens and young adults with cancer.

Donna Bernstein is a research nurse for MyPART. She has worked at the NIH as a nurse for many years on different clinical studies in both the inpatient and outpatient setting. As part of the MyPART team, she enrolls and consents patients onto the Natural History Study of Rare Solid Tumors. She explains, “I enjoy [being a research nurse] because it merges both phases of inpatient and outpatient. I get to meet the patients, get to know them, and work with them on the studies.”

In her role as a research nurse for MyPART, she also works closely with different members of the team, including the genetic counselor, psychologist, doctors, patient care coordinator, and clinical research associates. She says, “we are a cohesive team and all of us work very closely together to discuss each patient.” When asked about her favorite aspect of MyPART, she highlights her work with patients and explains, “I really enjoy talking to the patients and hearing their stories. [They] are so eager to help us on MyPART and it is such a joy to work with them. They have been through a lot and I feel so grateful that they are willing to participate in our studies.”

Learn more about the MyPART team here.

TargetCancer Foundation, a MyPART advocacy partner, launched the TCF-001 TRACK (Target Rare Cancer Knowledge) trial in October 2020 and aims to recruit 400 participants with rare cancers or cancers of unknown primary. TRACK is a prospective study to see if patients with rare cancers will benefit from targeted molecular therapies that may be identified through comprehensive genomic profiling. Each patient will receive comprehensive genomic profiling of their tumor tissue and blood. Patients can easily enroll in this study through a remote consenting process. Once patients consent to the study, TargetCancer Foundation study staff will facilitate the collection of medical records and samples for comprehensive genomic profiling. The TRACK Virtual Molecular Tumor Board of expert physicians will review the profiling results and suggest treatment recommendations to patients and their personal physicians. The collection of this genomic data may also lead to a better understanding of how rare cancers develop. Learn more about how to participate in this study here. TargetCancer Foundation supports rare cancer research and raises awareness about rare cancers among scientists, physicians, and patients. Learn more about TargetCancer Foundation here.