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A genetic disease that produces tumours around nerves could be treated with anticancer drugs after failing to respond to chemotherapy.
Researchers at the Indiana University (IU) School of Medicine report that Gleevec may be the first effective treatment for neurofibromatosis.
Neurofibromatosis results from mutations in a gene called NF1, which causes tumours to form in the cells that make up the protective sheaths around nerves.
IU research have begun a clinical test of Gleevec, treating neurofibromatosis patients with plexiform neurofibromas, which affects about 40% of people with neurofibromatosis. Such tumours often have a severe impact on patients’ quality of life and can be fatal.
Data, published in the journal Cell, shows that the mutated nervous-system cells in a pre-tumour state use molecular signals to recruit inflammatory system cells from the bone marrow to the vicinity of the nerve cells. Those inflammatory system cells – called mast cells – then help to create the cellular “scaffolding” and blood vessels necessary for the cancerous tumours to form.
Dr D Wade Clapp, from the IU School of Medicine, said: “We are very hopeful about the potential of this drug and related therapies. There are no other therapies for these tumours. These patients often suffer for years; they sometimes die from these tumours. These are very slow growing tumours that impair people’s everyday lives.”
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