"Smart drugs;" undiagnosed.

Q We have heard a lot about "smart drugs," especially for children with Down syndrome. Recently on the news show Day One, a drug called Piracetarn was shown to help learning ability in a child with Down syndrome. Our son is six years old and has Down syndrome. We would be interested in anything that may help him reach his full potential. What do you thin about the use of these new drugs?

A The use of the so-called "smart drugs"--as well as vitamin, mineral and amino acid supplements--is a very controversial area in the treatment of children with certain developmental disorders. Recently, there has been much publicity regarding "smart drugs," specifically Piracetam, as a supplement for children with Down syndrome. Piracetam, often used in conjunction with other supplements (typically vitamins, minerals, amino acids and, sometimes, digestive aids or enzymes), is one member of a new class of drugs that are thought to enhance learning and memory. No commonly accepted mechanism of action has been established; that is, if it works, we are not exactly sure how or why.

Piracetam (trade name Nootropil) is considered an orphan drug. An orphan drug is a drug still in clinical testing and not yet approved by the Food and Drug Administration (FDA), or a drug designated for use in rare disorders affecting 200,000 people or less. Piracetam's only indicated use is for a rare condition called myoclonus (a type of seizure disorder); its use is not indicated for learning disabilities, mental retardation or Down syndrome. In fact, at this time, I do not believe Piracetam can be legally imported into the U.S. Also, because of a lack of appropriate scientific research on the use of the drug in individuals with Down syndrome, the National Down Syndrome Society does not recommend the use of Piracetam for children with Down syndrome.

I wholeheartedly agree that any child--regardless of disability--should be allowed to reach his or her full potential. Certain less conventional therapies--such as drugs or other nutritional or vitamin supplements--may have a role if used in a cautious manner. And any such supplements should be used only in conjunction with more conventional medications, helpful therapies, an appropriate educational program, a nutritious diet and appropriate exercise.

If you wish to consider the use of any of these "smart drugs" or other supplements, I encourage you to check with your son's pediatrician to make sure no other medical, therapeutic or educational options need to be considered first. Your son's pediatrician can also assess the possibility of any drug interactions. Under no circumstances should one assume that if a certain amount of a vitamin or supplement has been shown to be helpful, then twice as much (or more) of that substance will be even better.

Q Our eight-year-old daughter has global developmental delays (fine and gross motor and cognitive delays). Over the years, we have taken her to many neurologists, specialists in developmental disabilities, occupational and physical therapists, psychologists and pediatricians. None of these specialist has been able to give us a definitive diagnosis for our daughter's problems. My wife and I are uncomfortable with this situation. We are also worried about any future children we may have. Can you think of anything else we might do?

A You have done a significant amount for your daughter already. Lack of a specific diagnosis is one of the most frustrating situations for patients, parents and physicians. Patients without a specific diagnosis often tend to be "placed" into broad descriptive-based categories that describe symptoms such as hypotonia (low muscle tone), developmental delay, non-specific learning disability, encephalopathy (a disease of the brain, either unchanging or progressive) or cerebral palsy (when used in its broadest sense to mean muscular impairment or poor coordination).

Such general classifications can make it difficult to design and obtain insurance coverage for appropriate therapies and treatments. It can also make it more difficult to qualify for certain state and federal health programs that allow coverage only for certain specific "diagnoses." And parents of a child without a diagnosis are less likely to find appropriate support groups or other resources.

Family and friends may be surprised that you have not found an "answer" to your daughter's problems. You are probably getting a number of unsolicited "answers" and opinions from well-meaning family members and friends.

If you have not already done so, you should consider a genetics consultation. This would include a detailed look at your family history, chromosome studies and an evaluation of your daughter's symptoms and appearance to see if they fit any particular syndrome. This type of consultation might also address your anxieties with regard to any future children.

Medical knowledge changes rapidly, so more information about your daughter's condition may become available in the next few years. In the meantime, even without a diagnosis, you need to continue to deal with your daughter's needs for occupational, speech and physical therapy, and to make sure she is in a school program that best meets her educational needs.

Over the years, I have seen many patients who were undiagnosed; some remain undiagnosed. But their parents, like you, have accepted these children just as they are. Parents have taught me that whether a child has a specific diagnosis or not, that is the most important thing you can do.

In this column, David Hirsch, M.D., a pediatrician and member of the Exceptional Parent's Editorial Advisory Board, answers questions from readers. Dr. Hirsch is a partner in Phoenix Pediatrics, Ltd. in Phoenix, Arizona. He specializes in treating children with developmental disabilities and chronic illnesses.

Since Dr. Hirsch is responding to letters and has not examined the child in question, parents need to review his suggestions with appropriate professionals. Dr. Hirsch mentions specific products or medications only to illustrate suggestions; he is not endorsing any specific products.

Send questions to: Ask the Doctor, Exceptional Parent, 209 Harvard Street, Suite 303, Brookline, MA 02146-5005, [phone] (Fax).