This Phase I study looked at the safety and efficacy of lovastatin as a treatment for patients with neurofibromatosis type 1, 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, visual attention, 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.
|Contact: Emily Hartman|
Children's National Medical Center