NBC News shared the story and collective work of Children’s Hospitals and Clinics of Minnesota’s blood and cancer disorders team, including Dr. Kris Ann Schultz, Dr. Yoav Messinger, Gretchen Williams, CCRP, and Anne Harris, MPH, among others, who have led the way in enabling the early detection and effective treatment of children in families affected by rare genetic cancers.
In 2009, while trying to understand pleuropulmonary blastoma (or PPB, a rare early childhood lung cancer), researchers leveraging data from Children’s International Pleuropulmonary Blastoma (PPB) Registry uncovered an unexpected cause: a mutation in DICER1, a master controller gene that helps regulate other genes. By leveraging those learnings and coupling it with new data from the International Ovarian and Testicular Stromal (OTST) Registry – a “sister” registry of the International PPB Registry – Children’s has recently discovered that the DICER1 gene mutation may underlie many additional rare childhood genetic cancers and could tell us something fundamental about how most cancers arise.
Children’s presented its promising findings this weekend at the American Society of Clinical Oncology (ASCO) Annual Meeting, advancing the potential for early diagnosis and proactive treatment of children in families affected by PPB, as well as other rare genetic cancers such as certain ovarian, nose, eye and thyroid tumors.
With the establishment of the International PPB Registry in 1988 and the International OTST Registry in 2011, Children’s, along with our partners, have become the world’s leading experts on how to care for children with PPB and other cancers marked by the DICER1 genetic defect. As a result, PPB could be among the first cancers routinely curable before it progresses to a deadly form.
Congratulations to the Children’s cancer and blood disorders team! Thank you for your commitment and your amazing, groundbreaking work.