Brigitte C. Widemann, M.D., Chief of the Pediatric Oncology Branch, has been named a Top Ten Clinical Research Achievement Awardee by the Clinical Research Forum and has received a Distinguished Clinical Research Achievement Award for her study “Selumetinib in Children with Inoperable Plexiform Neurofibromas.” The Distinguished Clinical Research Achievement Awards are presented to the top two of the 10 studies that show creativity, innovation, or a novel approach that demonstrates an immediate impact on the health and well-being of patients.
Plexiform neurofibromas are tumors that grow along nerves and occur in approximately 50 percent of children with neurofibromatosis type 1 (NF1), a genetic disorder that affects one in 3000 individuals. These tumors are challenging to treat and can cause disfigurement, chronic pain, breathing difficulties and other symptoms depending on the tumor location. Until recently, there was no effective medical treatment for plexiform neurofibromas. Based on the findings from Widemann’s study, the Food and Drug Administration approved selumetinib for children two years and older with NF1 and symptomatic, inoperable plexiform neurofibromas. Selumetnib provides the first approved medical therapy for these tumors.
The award-winning studies recognized by the Clinical Research Forum exemplify major advances resulting from the nation’s investment in research to benefit the health and welfare of its citizens and reflect the influential work being conducted by investigators at research institutions and hospitals across the United States, as well as with partner institutions from around the world.
The top 10 studies were selected based on the degree of innovation and novelty involved in the advancement of science; contribution to the understanding of human disease and/or physiology; and potential impact upon the diagnosis, prevention and/or treatment of disease.
All awardees were honored at the 2021 Top Ten Clinical Research Achievement Awards Virtual Event on March 30. Watch this video to learn more about “Selumetinib in Children with Inoperable Plexiform Neurofibromas” from Widemann and colleague Andrea Gross, M.D., Assistant Research Physician in the Pediatric Oncology Branch.
In addition to these clinical achievements, Widemann was recently elected to the Association of American Physicians (AAP). She will be recognized during a virtual gathering on April 9 with members from the AAP, the American Society for Clinical Investigation and the American Physician Scientists Association.