Common cholesterol drug safe, may improve learning disabilities in patients with neurofibromatosis

Researchers at Children's National Medical Center have found that a cholesterol-lowering statin drug appears to be safe in children with neurofibromatosis type 1 (NF1) and may improve learning disabilities, including verbal and nonverbal memory. This is the first time that the drug lovastatin has been studied in children with NF1. The study, led by Maria T. Acosta, MD, a pediatric neurologist and researcher at Children's National and clinical director and cognitive director of the Gilbert Family Neurofibromatosis Institute, appears in the October 2011 issue of Pediatric Neurology.

Lovastatin is currently approved by the U.S. in adults and children for the treatment of high cholesterol. The drug works by inhibiting a specific enzyme in cholesterol biosynthesis. Previous animal studies have found that lovastatin affects a related molecular pathway that may be linked to cognitive deficits in neurofibromatosis.

"While we originally set out to determine the safety of lovastatin in NF1 patients, we also saw statistical improvements in memory and visual attention, which is a big step towards helping improve our patients' quality of life and in evaluating which may be effective therapies for NF1," stated Dr. Acosta. "While this is a relatively small study, we now have strong baseline information, and we are working with other institutions in the country and throughout the world to perform a definitive study to replicate these findings on a larger scale."

This Phase I study looked at the safety and efficacy of lovastatin as a treatment for patients with , which accounts for the majority of NF cases. Over a period of three months, 24 patients between ages 10-17 years received treatment with lovastatin. Patients were given cognitive functioning tests before and after treatment. All patients maintained normal cholesterol levels throughout the study, and there were some cognitive improvements in memory, , and efficiency following treatment.

"The implications for all children with learning disabilities – not only those with NF1 – are of interest to the greater pediatric community, so we hope to move forward quickly through the Consortium to advance this research in NF1 patients in a timely manner," commented Roger Packer, MD, Senior Vice President of the Center for Neuroscience and Behavioral Medicine, Director of the Gilbert Family Neurofibromatosis Institute at Children's National, and Group Chair of the Department of Defense Neurofibromatosis Clinical Trials Consortium.

More information: Lovastatin as Treatment for Neurocognitive Deficits in Neurofibromatosis Type 1: Phase I Study, www.sciencedirect.com/science/… ii/S0887899411003146

Related Stories

Cholesterol-lowering drug boosts bone repair

Jul 31, 2008

Lovastatin, a drug used to lower cholesterol and help prevent cardiovascular disease, has been shown to improve bone healing in an animal model of neurofibromatosis type 1 (NF1). The research, reported today in the open access ...

Recommended for you

Birthday matters for wiring-up the brain's vision centers

14 hours ago

Researchers at the University of California, San Diego School of Medicine have evidence suggesting that neurons in the developing brains of mice are guided by a simple but elegant birth order rule that allows them to find ...

How is depression related to dementia?

Jul 30, 2014

A new study by neuropsychiatric researchers at Rush University Medical Center gives insight into the relationship between depression and dementia. The study is published in the July 30, 2014, online issue of Neurology, the me ...

User comments