Down Syndrome and the Piracetam Myth

There was bad (and worse) news last month for parents of Down syndrome children who had hoped that Piracetam would boost their sons’ or daughters’ mental abilities.

The first rigorous study of this use of the drug found that Down syndrome youngsters receiving Piracetam do not improve intellectually. Furthermore, many of them exhibit negative behaviors such as aggressiveness, agitation, irritability, sexual arousal, poor sleep habits and decreased appetite in response to the medication.

The study was conducted by researchers at the Imaging Research and Cognitive Neurology Unit at Sunnybrook and Women’s College Health Sciences Centre in Toronto, Canada. It was presented in May 1999 at the Pediatric Academic Societies/Society for Pediatric Research meeting in San Francisco, Calif., and recently published in the April 2001 issue of Archives of Pediatric and Adolescent Medicine, along with an accompanying editorial examining the role of parent advocacy groups in medical practice.

The Most Common Genetic Disorder
Down syndrome is the most common genetic disorder. Caused by a third 21st chromosome, it occurs about once in every 600 to 700 births.

Individuals with Down syndrome typically have poor muscle tone, short stature, a small nose with a flat nasal bridge, small skin folds on the inner corners of their eyes, dry skin, immune system suppression, developmental delays, speech difficulties and mental retardation.

Parents of children with the condition have long sought a medication, an educational approach, or other means of improving their child’s health and intellectual functioning.

That desire partly explains how the use of Piracetam for children with Down syndrome became so widespread without scientific evidence of its usefulness.

Popularity Ignited by the Internet, Fueled by TV
Len Leshin, M.D., is a pediatrician practicing in Corpus Christi, Texas, and the father of two sons, the younger of whom has Down syndrome. Leshin says that scientific evidence indicating Piracetam should be prescribed for Down syndrome has always been scant, at best. He asserts that promotion of the drug was never driven by the medical community, but by laypersons convinced of its efficacy due to reports from other parents and teachers of Down syndrome children.

“In the mid-1990s, a woman with no medical credentials or training began to tout the drug’s effectiveness for Down syndrome children on the Internet,” Leshin explains. “Its popularity grew so rapidly among some Down syndrome parents that ABC-TV’s ‘Day One’ aired a program covering its use. The show suggested the drug was capable of significantly improving a Down syndrome child’s I.Q. Such media coverage further fueled the demand for and use of Piracetam. But from the beginning, the only evidence supporting its use for these children was a poorly designed study conducted in Barcelona, Spain in the 1970s.”

The anecdotal reports and one questionable study two decades before were enough for many parents and teachers, and some pediatricians who began to administer the drug to children with high hopes throughout the late 1990s and into the new century.

But not every parent was convinced by what Chris Feudtner, M.D., Ph.D., a professor in the department of pediatrics at the Child Health Institute at the University of Washington, Seattle, describes as “exuberant testimonials appearing on television and the Internet.”

Canadian Mary Hird wanted more than parental anecdotes about how piracetam could work wonders. The mother of a young son with Down syndrome, Hird posted her feelings about the drug on one of the many Internet bulletin boards discussing the medication’s use in 1997. “All the great many questions regarding Piracetam should be answered, and with medically proven backup,” she said. “I feel that unless a drug has really been studied by a medical team in a fair and comprehensive manner, then a drug cannot truly be promoted.”

The Study
Hird’s son was one of 25 young boys and girls, six to 13 years old, recruited by the Sunnybrook and Women’s College Health Sciences Centre Piracetam research team headed by Nancy J. Lobaugh, Ph.D. The youngsters participated in a tightly controlled, double-blind scientific study of the drug, which 18 of them completed.

Over an eight-month period, each child tried Piracetam for four months, and a placebo (an agent that has no active ingredients) for four months. At the end of each four-month period, a thorough assessment was made of each child’s mental functioning and behaviors. The tests looked at attention span, learning, memory, fine motor skills and spatial skills among other things, using a large battery of tests. In addition, the children’s parents completed an 80-item questionnaire, and their teachers completed a similar 24-item instrument.

No Better Than Placebo, With Disturbing Side Effects
When the research team analyzed the evidence collected, they found Piracetam had no greater positive effect than the placebo on the 18 youngsters’ cognitive abilities. Their analysis did show, however, that for some children, taking Piracetam was associated with a variety of negative behaviors linked to stimulation of their central nervous systems.

Lobaugh and her team pointed out that their results are very different from the anecdotal testimonials presented in the popular press. Based on what they found, the researchers did not find any reason to recommend that parents of Down syndrome give Piracetam to their children.

As Feudtner put it, “These findings should temper our enthusiasm for Piracetam markedly.”

A Drug in Search of a Disease
Leshin refers to Piracetam as “a drug in search of a disease,” noting that it’s a best-selling drug in both Europe and Japan where normal adults use it in an effort to boost their cognitive abilities. He notes that UCB Pharma, the Belgian pharmaceutical company that manufactures Piracetam, has discouraged its use for Down syndrome and denied that it has any plans to research the drug’s use by children with the condition.

However, as Leshin points out, that doesn’t mean other studies won’t be undertaken to replicate Lobaugh’s results or extend the research. In fact, Leshin says, parents and others interested in Down’s syndrome children can expect additional studies in the near future that will explore whether Piracetam in conjunction with another agent called choline works any better than Piracetam alone.

 

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