Dose of hope for children with Down syndrome Medicine: Kennedy Krieger Institute will launch a clinical trial to test the usefulness of a "smart drug."

August 22, 1997|By Jonathan Bor | Jonathan Bor,SUN STAFF

The prescription for Down syndrome has long been love, patience and an acceptance of nature's imponderables. Now, scientists are joining parents in asking: Might there be more?

Baltimore's Kennedy Krieger Institute will soon start a clinical trial that could prove whether a "smart drug" can enhance the intellect of children with the genetic abnormality.

Called piracetam (pa-RA-sah-tam), the drug targets one of the brain's neural pathways. The trial will be the first to use objective measurements to assess the drug's effects on children with Down syndrome.

Researchers led by Dr. George Capone will monitor youngsters to see if the medicine improves their ability to speak, read and remember -- or simply to focus on a task.

The drug has earned glowing testimonials from many U.S. parents who order it through the mail from Europe, Canada and Mexico.

"There are some parents who are claiming that it helps," said Capone, a developmental pediatrician who runs the hospital's Down syndrome clinic. "There are also parents claiming that they don't see very much and parents who claim outright that they don't see any effects.

"Naturally, you tend to hear the ones who are most vocal about it helping."

On dozens of sites on the Internet, parents share their experiences and advice on how to get the drug. The Food and Drug Administration has not approved its sale in the United States. But a person can legally obtain a 90-day supply for personal use from countries where pharmaceuticals are more loosely regulated.

There are an estimated 300,000 families in the United States with Down syndrome children, a condition that is caused by the presence of an extra chromosome in each of the body's cells. Because the chromosome appears in the 21st position -- making a trio out of what should be a pair -- the syndrome is also known as trisomy 21.

Children typically have poor muscle tone, small facial features, a large tongue, and hands that are short and broad. Frequently, they are born with a misshapened heart, and are prone to hearing loss and acute leukemia.

They also have mental retardation, with IQs ranging from 30 to 80. (The norm is 100). While some never learn to speak and need close supervision, others can read and hold jobs.

The extra chromosome causes developmental problems from the moment of conception. There are too few cells throughout the body. In the brain, the shortage affects direction of thought and coordination of muscles.

Tantalizing idea

Like thousands of parents of Down syndrome children, Martin and Betsy Conover were tantalized by the idea that piracetam might help their daughter, Alexis.

"There's no scientific proof, but we wanted to do anything we thought would be a step in the right direction for our daughter -- something to enhance her learning ability," said Betsy Conover.

They started her on a daily dose of piracetam about a year ago. They tap various distributors, so the shipments come from England, France, Germany and the Netherlands. Recently, a supply came in a plain envelope without a package insert or even a brand name -- just a perforated sheet of capsules in plastic bubbles.

The drug, which is often sold over the counter, can cost less than $150 for a year's worth.

Alexis, who just turned 8, is an affectionate and energetic child who has a stubborn streak that can challenge her teachers.

She speaks in short but complete sentences and cruises through elementary computer games that test memory. Her parents consider her high-functioning.

They believe piracetam has made a difference: She seems to learn faster, and she has a better concept of rules and boundaries. It's been a long time since she's put a scare through the household by wandering out the door.

At the same time, the Conovers admit that they have no proof that the drug is responsible. It is possible Alexis would have made strides without it, as many children with Down syndrome have before her.

Because piracetam has no known side effects, the Conovers see no reason to withhold it. "I can see her taking the drug for the rest of her life," her father said.

Turn to medication

Although the past 30 years have seen vast improvements in educational services that help children with Down syndrome realize their potential, the idea of treating a child's mental retardation with medicine is new. The trend began with Dixie Tafoya, a Louisiana woman who rejected the notion that medical science had nothing to offer her adopted daughter, Madison, who was born with Down syndrome.

An exhaustive search through medical sourcebooks and databases led Tafoya to two conclusions. Because the syndrome hampers processing of nutrients, she surmised that it would be beneficial to give supplements of vitamins, minerals, amino acids, digestive enzymes and anti-oxidants.

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