Lost in Translation: Simvastatin for Neurofibromatosis Type 1

Clinical manifestations of neurofibromatosis type 1 (NF1) include learning disabilities, especially visuospatial impairments; MRIs show hyperintensities in the brains of NF1 patients. Previously, researchers using a mouse model of NF1 found that cognitive deficits markedly improved when the mice were treated with statin drugs. Now, researchers have examined whether the findings in mice would translate to children (two of the researchers have applied for a related U.S. patent).

In a double-blind, 12-week study, 62 children (age range, 8–16) were randomized to receive simvastatin or placebo. Groups were matched except for age, which analyses controlled for. At baseline and at 12 weeks, children underwent a comprehensive battery of visuospatial…