Early-phase Trial Demonstrates Shrinkage in Pediatric Neural Tumors

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].

Early-phase trial demonstrates shrinkage in pediatric neural tumors

MIA is a Potential Biomarker for NF1 Tumor Load

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].

Blood test

The 2010 NF Conference – Connecting the Public with the Research

Neurofibromatosis (NF) encompasses a set of genetic disorders that cause benign and malignant tumors to grow along various types of nerves; it can also affect the development of bones and skin. There are three main types of NF tumors: neurofibromatosis type 1 (NF1), neurofibromatosis type 2 (NF2) and schwannomatosis. NF1 is the most frequent of the three; one in every 3,000 children is born with the disease.

The Children’s Tumor Foundation (CTF) is the leading non-governmental funder of scientific research into neurofibromatosis and has funded NF studies for over 25 years. Their goal is to identify NF drug therapies and improve the lives of those living with the disorder. The Foundation also endeavors to increase public awareness of NF and provides resources for NF patients and their families.

2010 NF Conference

10 Things to be Aware of on NF Awareness Day

May is NF Awareness Month, and in a particular effort to help get the word out about NF, today — Thursday, May 20th — is NF Awareness Day.

NF Awareness Day

Neurofibromatosis (NF) is a genetically inherited disorder that predisposes individuals to the development of a variety of benign and malignant tumors in the central and peripheral nervious systems. The disorder affects neural crest cells and causes tumors to grow along various types of nerves and can also affect the development of bones and skin.

Neural crest cells: specialized cells that contribute to the formation of multiple tissues, including sensory nerves, the enteric nervous system that controls the gut, pigment cells of the skin, various skeletal and connective tissue of the head, and part of the valves and blood vessels of the heart. During early development, neural crest cells — a transient, multipotent cell population — migrate to many different locations and differentiate into many cell types within the embryo.

The Children’s Tumor Foundation (CTF) is the leading non-governmental funder of scientific research into neurofibromatosis. The CTF has funded NF research for over 25 years with the goal of identifying NF drug therapies and improving the lives of those living with the disorder. The Foundation also endeavors to increase public awareness of NF and provides resources for NF patients and their families. Straight from the CTF, here are ten things to be aware of about NF:

Neurofibromatosis: From Genes to Complications to Treatments

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

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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.