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Two robust fragile X syndrome findings intersect

Last Friday, the New York Times reported successful results in an early-stage clinical trial of a drug for the treatment of fragile X syndrome. This genetic disorder is the most common inherited cause of retardation and autism.

The drug, which led to improved behavioral outcomes, was tested only in adults. But many believe, according to the Times, "that such compounds may prove most effective in young children, whose brains are far more likely to respond rapidly... "

There's an ethical problem here. You would never want to run this kind of test in young children with (or without) fragile X syndrome, since you don't really know whether the drug is working - or whether it may, instead, be causing damage to their still developing brains.

Now along comes word that Stanford's Allan Reiss, MD, and his colleagues have been able to image the brains of very young boys (ages 1-3) with fragile X syndrome and compare those images to those of the brains of normal, like-aged counterparts.

The study showed significant developmental differences taking place in fragile X brains, compared with normal ones, over a two-year period between imaging sessions. Because this MRI-based imaging method is noninvasive, it could be safely used to determine whether prospective drug candidates are causing young patients' brain development to more closely resemble that of normal children's.

Thus, the imaging technique could guide therapy: If successive images show that a patient's brain is converging toward normality and that the drug therefore appears to be working, stay with it - if not, switch to another one quickly, before the drug can inflict lasting harm.


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