Statins, bone, and neurofibromatosis type 1
Neurofibromatosis type 1 (NF1) is a dominantly inherited multi-system disorder. Major features include pigmentary abnormalities, benign tumors of the nerve sheath (neurofibromas), malignant tumors, learning disabilities, and skeletal dysplasia.
The NF1 gene functions as a tumor suppressor, but haploinsuffiency probably accounts for some aspects of the non-tumor phenotype. The protein product, neurofibromin, is a Ras GTPase-activating protein, and various Ras pathway inhibitors are being tested in preclinical models and clinical trials for effectiveness in treating NF1 complications.
The paper by Kolanczyk et al in this volume of BMC Medicine describes a preclinical mouse model for one of the most difficult NF1 lesions to treat, tibial dysplasia, and provides evidence that a existing drug with a known safety profile in children, lovastatin, may be beneficial, opening the door to clinical trials in humans.
Author: Bruce R Korf
Credits/Source: BMC Medicine 2008, 6:22
Published on: 2008-07-31
Copyright by the authors listed above - made available via BioMedCentral (Open Access). Please
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