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

Study links enzyme to autistic behaviors

10 hours ago

Fragile X syndrome (FXS) is a genetic disorder that causes obsessive-compulsive and repetitive behaviors, and other behaviors on the autistic spectrum, as well as cognitive deficits. It is the most common ...

A new cause of mental disease?

15 hours ago

Astrocytes, the cells that make the background of the brain and support neurons, might be behind mental disorders such as depression and schizophrenia, according to new research by a Portuguese team from ...

Molecular basis of age-related memory loss explained

Jul 22, 2014

From telephone numbers to foreign vocabulary, our brains hold a seemingly endless supply of information. However, as we are getting older, our ability to learn and remember new things declines. A team of ...

The neurochemistry of addiction

Jul 22, 2014

We've all heard the term "addictive personality," and many of us know individuals who are consistently more likely to take the extra drink or pill that puts them over the edge. But the specific balance of ...

User comments