Pediatric Cancer
Image source: NCI Visuals Online
Neurofibromatosis type 1 (NF1) is a genetic disorder that causes tumors to form on nerve tissue. NF1 is usually diagnosed in childhood, and there are no effective medical therapies for most types of tumors. Up to half of the people with NF1 develop plexiform neurofibromas (PNs). Over time, PNs may transform into atypical neurofibromas (ANFs) and then into cancerous tumors that grow along nerves. At this stage, patients respond poorly to chemotherapy.
Andrea Gross, M.D., Assistant Research Physician in the Pediatric Oncology Branch, is leading a study of a drug, abemaciclib, to treat ANFs in children. Abemaciclib works by blocking the action of an abnormal protein that signals the ANF cells to multiply, thus helping to slow the growth of tumor cells. Researchers want to determine the best dose of abemaciclib for NF1 and to see how participants respond to treatment.
Clinicaltrials.gov identifier: NCT04750928
NCI Protocol ID: NCI-21-C-0011
Official Title: A Phase I/II Study of the Cyclin-Dependent Kinase(CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type 1 (NF1) Related Atypical Neurofibromas
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