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To the Editor: Dr Krab and colleagues¹ investigated the possible benefit of statin therapy in children with neurofibromatosis type 1 (NF1), a genetic disorder associated with learning disabilities.² In this randomized, double-blind, placebo-controlled trial, it was found that simvastatin, a 3-hydroxy-3-methylglutaryl coenzyme A (HMG-CoA) reductase inhibitor, did not improve cognitive function in children with NF1.¹ The motivation for this clinical trial came from the beneficial effect of lovastatin (another HMG-CoA reductase inhibitor) on cognitive function in the Nf1^+/– mouse model NF1.³ These mice are heterozygous for a null mutation in neurofibromin, exhibit behavioral disorders that resemble those found in humans, and display deficits in physiological correlates of memory.³

However, the potential mechanism for an effect of statins on cognitive performance in the Nf1^+/– mouse is not clear.⁴ Statins are cholesterol-lowering drugs, but neurofibromin is not involved in cholesterol metabolism as it is a tumor suppressor protein. Mutations in neurofibromin are . . . [Full Text of this Article]

Jacobus F. A. Jansen, PhD
jansenjfa@gmail.com
Department of Medical Physics and Radiology
Memorial Sloan-Kettering Cancer Center
New York, New York

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