In 2005, scientists at the University of California, Los Angeles used the cholesterol-lowering drug lovastatin to successfully treat a learning disability in experimental mice. The study, led by Dr. Alcino J. Silva, sparked intense research into how we think, and whether this drug could help those suffering from a genetic learning disability called neurofibromatosis Type 1, or NF1. In 2010, researchers reported that the drug improved cognitive function in a small group of children with NF1. Larger trials are underway.
Neurofibromatosis Type 1
NF1 is a learning disability found in about 1-in-3,000 people worldwide. People often inherit the disorder, but cases also occur because of a spontaneous genetic mutation. Many children born with this condition have larger than normal head circumference and are shorter than average. In addition to many physical problems, these children may have poor language skills and may struggle with reading, spelling and mathematics. Attention deficit hyperactivity disorder (ADHD) is common. Symptoms may worsen over time, but these children generally live a normal lifespan. There is no effective medical treatment.
Lovastatin
Lovastatin is an FDA-approved drug used to treat high cholesterol. It is sold under the brand names Altocor, Altoprev and Mevacor. It belongs to a class of medications that inhibit production of cholesterol in our bodies. Cholesterol builds up on the walls of blood vessels and can cut off the supply of blood and oxygen to the heart, brain and other parts of the body. Lovastatin may cause side effects, which can be severe. They include muscle pain, fatigue, nausea, rash, hives, itching and swelling.
2005 Research Discovery
Seminal research on lovastatin and cognition in 2005 focused on experimental mice with the NF1 genetic defect. Researchers understood that people with NF1 produced too much of a protein called Ras, which is used in nerve function. Ras is made up of fatty molecules called lipids. Lovastatin lowers lipids. Mice were given lovastatin for three months, and their cognitive function improved remarkably. The question since then has been whether this mice study could be applied to humans.
Early Research Reveals Cognitive Secrets
In July 2010, Dr. Silva's research team at UCLA published more studies showing that mice carrying NF1 mutations had higher levels of the nerve transmitter GABA in the brain area for working memory. The findings imply that too much GABA hinders brain activity and interferes with memory. Meanwhile, researchers at the Children's National Medical Center in Washington reported that a small study of 24 children aged 10 to 17 with NF1 were given lovastatin for three months. All showed improvement in visual/spatial memory and cognition. Still, that's a very small study, and a much larger, international study is underway.
