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

September 27, 2011

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/article/pii/S0887899411003146

Related Stories

Recommended for you

New mechanism discovered behind infant epilepsy

September 3, 2015

Scientists at Karolinska Institutet and Karolinska University Hospital in Sweden have discovered a new explanation for severe early infant epilepsy. Mutations in the gene encoding the protein KCC2 can cause the disease, hereby ...

Neuron responsible for alcoholism found

September 2, 2015

Scientists have pinpointed a population of neurons in the brain that influences whether one drink leads to two, which could ultimately lead to a cure for alcoholism and other addictions.

Deciphering the olfactory receptor code

August 31, 2015

In animals, numerous behaviors are governed by the olfactory perception of their surrounding world. Whether originating in the nose of a mammal or the antennas of an insect, perception results from the combined activation ...

Scientists see motor neurons 'walking' in real time

September 2, 2015

When you're taking a walk around the block, your body is mostly on autopilot—you don't have to consciously think about alternating which leg you step with or which muscles it takes to lift a foot and put it back down. That's ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.