Can a pill cure intellectual disabilities?

one capsule in small spoonby Sharyn Lincoln, M.S., C.G.C. & Jonathan Picker, MD, PhD

The ability to improve an intellectually disabled person’s IQ sounds like science fiction, and not so long ago it was – but that is changing.

The Boston Globe recently reported on one of the most exciting things happening in medicine, and one which may profoundly change the way society approaches children and adults with special needs.  Specifically, they reported on the use of a drug that looks like it is going to help individuals with fragile X syndrome think better.

Fragile X syndrome is a disorder that causes significant intellectual disability, as well as behavioral problems. As many as 1000 people in the greater Boston area are expected to have fragile X syndrome. Boys usually have more difficulties than girls, but the disorder can be devastating for both, as well as for their family and friends.

Right now, treatment is restricted to trying to control the effects of the disorder, and most individuals remain significantly impaired. Like many developmental disorders, better treatments have long been needed, but most researchers, until recently, had little hope for success.

That has all changed with the new so called ‘targeted’ drugs for fragile X syndrome.  These medications are the first to undergo large scale research trials with a specific goal of improving intellect. What makes these drugs so exciting is that they are “custom-made” to target the biochemical imbalance caused by the disorder.

Unlike traditional medicines that only treat symptoms, these drugs are specifically designed to restore the biochemical imbalance present in fragile X syndrome. As the brain chemistry normalizes, the symptoms, including intellectual disability, improve.

The advent of these medications is possible because of the research resulting from the Human Genome Project, which has allowed us to understand the molecular steps that cause various genetic conditions.  The development of targeted therapies has all happened at record speed as the scientists have worked hand in glove with the clinical researchers to move from theory to drug trials in the matter of just a few years.

And the best part is that fragile X syndrome is just one of many disorders that will benefit from this approach. While it is still early, we can at last begin to look towards the day when treating intellectual disability is as simple as taking a pill.