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In an early-phase clinical trial of a new oral drug, selumetinib, children with the common genetic disorder neurofibromatosis type 1 (NF1) and plexiform neurofibromas, tumors of the peripheral nerves, tolerated selumetinib and, in most cases, responded to it with tumor shrinkage. NF1 affects 1 in 3,000 people. The study results appeared Dec. 29, 2016, in the New England Journal of Medicine [1].
Neurofibromatosis type 1 is a genetic condition that can cause tumors to form on nerves under the skin. Since these tumors can become malignant, it is important to monitor their growth closely and detect signs of malignant transformation as early as possible. However, the only way to currently detect them is with an MRI scan. New research published in BioMed Central’s open access journal BMC Medicine shows that a simple blood test for the protein melanoma-inhibitory activity (MIA) may be used to indicate the presence of neurofibromas even if they cannot be seen [1].
The 2008 NF Conference was held last weekend (June 6 — 10) in Bonita Springs, Florida. The preeminent annual meeting provides a forum for basic and clinical neurofibromatosis (NF) investigators to present their research (pronounced noor-oh-fahy-broh-muh-toh-sis). The conference was attended by over 200 researchers from around the world
This year’s theme — Genes to Complications to Treatments — highlighted the progress being made in NF research and clinical care, as well as the research programs of the Children’s Tumor Foundation. Last year’s NF Conference focused on models, mechanisms and therapeutic targets.
The annual Children’s Tumor Foundation NF Conference was held in Park City, Utah earlier this week (June 10 — 12). For three days, research and clinical investigators from around the world met to present their data and discuss the latest findings in neurofibromatosis (NF) research (pronounced noor-oh-fahy-broh-muh-toh-sis). This year the meeting focused on models, mechanisms and therapeutic targets.
The Children’s Tumor Foundation is dedicated to ending NF through research. The Foundation has sponsored research for over 25 years to understand the molecular basis of NF and to establish effective treatments and improve the lives of those living with the disease.
