Drug Research Finding May Help Children with Down Syndrome

For years researchers have fought to find a treatment for countless children born with Down Syndrome each year. Finally, all the years have come to mean what may be the largest scientific breakthrough in years. "It's a very exciting piece of work, "said David Patterson, a Down Syndrome researcher at the University of Denver who was not involved in the study. "This is really the first time that I've seen such a striking effect . . ."

Researchers studying the effects of an approved epilepsy drug on Down Syndrome affected mice have recently found their ability to learn increases while on the drug.

The drug pentylenetetrazole or PTZ for short was administered to the mice and the positive results were measured for up to two weeks after administration. This finding leads researchers to believe the drug has effects similar to those of other psychiatric drugs. The long-term effects may also lead to long-term effects on the brain.

"This treatment has remarkable potential, "said Craig Garner, a professor of psychiatry and a director of the Down Syndrome Research Center at California's Stanford University. The finding may also lead to future understanding of what causes the mental retardation associated with Down Syndrome. "Ou8r findings clearly open a new avenue for considering how cognitive dysfunctions in individuals with Down Syndrome might be treated."

It has taken researchers years of searching drug after drug to find a positive result. "So many other drugs have been tried that had no effect at all, "Garner said.

Down Syndrome effects patients by inhibiting learning processes in the brain from a moderate to sever level. PTZ causes more GABA, a message-carrying chemical, to be available in the brain. On the other said of the coin, overdoing this can cause seizures in patients. PTZ was previously approved by the FDA for treating epilepsy in humans but is no longer approved on the market for this purpose. Researchers are hoping to harness the excitation effect produced by the increased GABA to benefit people with Down Syndrome in the same way it has been able to affect the mice in the trial.

After merely 17 days on the medication the mice were able to learn more on the level of normal mice. The mice's ability to learn was demonstrated by their ability to learn mazes and shape recognition. This may suggest the drug has a brain changing effect which would open the door for more tests and studies into PTZ.

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