PERSONAL HEALTH : Growth-Drug Debate: What Is Too Short?

At age 12, Jake is 4 feet tall. He is in the unfortunate position of having to look up at least eight inches to face the other boys in his class.

He has always been the shortest boy--on his team, in his neighborhood. He knows well what it’s like to be the butt of jokes.

But now, Jake (not his real name) and his parents are hoping for a change.

Three times a week, Jake injects himself with human growth hormone--hGH--a drug that mimics the natural hormone responsible for growth, which is produced by the pituitary gland. Both he and his parents hope fervently that, with several years of use, the drug will allow him to measure up to his peers.

About 20,000 American children are candidates to receive hGH because their bodies do not make enough of the hormone for adequate growth.

The problem is Jake is not one of them.

Like thousands of children receiving the drug, Jake’s body produces a normal amount of growth hormone. But Jake is short because his parents are short. And, according to medical experts, an increasing number of parents are seeking hGH for their short but healthy children even though the drug is not approved for this purpose and there is no evidence it works for these kids.

Even if the hGH does prove effective in these cases, its use presents a prickly medical and ethical dilemma for pediatricians, parents and the government:

Should extreme shortness be considered a disease, a disability, or neither?

To Dr. John Lantos, a pediatrician and medical ethicist at the University of Chicago, treating short but healthy children with hGH is more like performing a cosmetic procedure than treating disease.

“I suppose you could compare human growth hormone (for healthy children) to any cosmetic procedure, such as getting braces or breast implants,” says Lantos, a longtime voice of caution against overuse of hGH. “But the more invasive, the more risks, the more expense, the more controversial it becomes. And when the patients are children, some people might say that doctors shouldn’t be involved in this and that it should be illegal.”

But, says Dr. Neil Shulman, an associate professor at Emory University School of Medicine and an advocate for research on hGH for extremely short children:

“I think that (use of hGH) should be up to the person who is going to be 3 feet tall. I don’t think someone should stand up on a pedestal and say, ‘We know what is best for you.’ From a medical standpoint, we can become a little bit elitist if we get up and tell people what is their problem and what is not.” The questions on whether shortness should be classified as a disease arose from what has been widely regarded as a significant medical achievement: In 1985, a genetically engineered form of human growth hormone was approved for use to treat children with diagnosed hGH deficiency.

Before then, the hormone was extracted from the pituitary glands of cadavers and injected into growth-deficient children. The therapy had serious side effects, and there was not enough hGH for every child who needed it.

The synthetic form of hGH is costly but plentiful and is considered safe for hGH-deficient children. And parents widely praise the drug for allowing these children to achieve their full growth potential.

But with the expanded availability has come questions on whether hGH should be given to children who are just as short as those with hGH deficiency--but for the “wrong” reason.

Legally, once a drug is approved, it can be used for purposes other than those for which it was originally intended.

“While we have learned much about hGH in the past decade, we seem to know less about how best to use it,” says Dr. David B. Allen, a pediatric endocrinologist at the University of Wisconsin Children’s Hospital who hosted a symposium last year on the uses of hGH.

For reasons ranging from heredity to hGH deficiency, as many as 90,000 children born each year rank in the lowest 3% of height in comparison to their peers. This means that if you measured 99 other children the same sex and age, the chances are that 97 of them would be taller than such children.

Whether hGH can help healthy children has been the subject of a government study that was temporarily halted last September because of criticism over its ethics and design.

In the study, up to 80 children ages 9 to 15 are being monitored for eight to 10 years while receiving injections of either hGH or a harmless saline solution three times a week. None of the children has hGH deficiency, but all meet strict criteria for short stature and are in the lowest 1% for height.

Officials at the National Institutes of Health argue that the study, which began in 1990, is important because the demand for hGH for short but healthy children will probably increase, and doctors and parents should know whether the treatment works and is safe.

“Pediatricians who feel as though it works will give it to kids, and pediatricians who feel it doesn’t work won’t give it,” says Shulman, an associate professor at Emory University School of Medicine and the author of a new book, “Understanding Growth Hormone.” “Parents who want it badly enough will shop around until they get a pediatrician who is willing to do it.”

For hormone-deficient children, hGH therapy usually adds to their final adult height and has proven safe so far. But for short but healthy children, studies have suggested that the drug only accelerates growth in adolescence and may not add to final adult height.

“For a kid with no underlying problem, they grow faster. That’s clear,” Lantos says. “The question is whether they end up taller then they would have been.”

If the drug proves effective, it should be available to extremely short children whatever the cause of their stature, argues Dr. Raymond L. Hintz of Stanford University Medical Center, which also is studying hGH in short but healthy children.

“It is not for children who are a little short, just below average,” he says. “These children (in the Stanford study) are just as short as those with bona fide hGH deficiency. We would never think of dealing with people who are just a little bit shorter.”

Proponents of these studies say further research on short stature also may reveal more about its causes.

Indeed, some extremely short children may have a disorder other than hGH deficiency and could benefit from the drug, Shulman says. For example, studies on children who have Turner syndrome, a genetic abnormality in girls, and renal failure show that hGH is effective in adding to adult height. Many children with these conditions, however, have been unable to get hGH because insurers won’t pay for the therapy.

Thousands of other children will still have no obvious cause for short stature other than heredity. For these children, the debate on the use of the hormone has deeply divided experts.

Some critics, such as Jeremy Rifkin, president of the Foundation on Economic Trends, attack the use of any drugs for physical enhancement or to promote social norms. Others question whether short stature needs a cure, while some simply wonder if it’s all that bad to be extremely short.

Although clearly not everyone with short stature has adjustment difficulties, studies show that taller people have a better chance of success on the job, in relationships and achieving social status.

Besides subtle discrimination directed at them, short people--those in the lowest 3% on the growth chart--are expected to put up cheerfully with any difficulties fostered by their size, such as the inability to drive a car or reach the top shelves in stores, says Shulman.

“There is no strong advocacy for short people in the country. People laugh at it. Shortness is not considered a disability,” he says.

The disadvantages of being an extremely short adult are more widely documented than potential problems faced by very short children, however. And some experts say the central question concerning the hGH studies is whether the therapy improves the self-esteem and behavior of very short children.

One recent study suggests children who are significantly shorter may get teased a lot but do not appear to have substantially more emotional or behavioral problems than taller children, says researcher David Sandberg of the State University of New York at Buffalo.

Sandberg also questions whether a child having emotional or behavior problems would find those problems alleviated by hGH therapy.

“If a child or adolescent is having difficulty coping with their stature, that should be addressed directly” through professional counseling or parental guidance, he says.

Ethicists also argue that even if hGH was given to children in the lowest 3%, there would always be a new category of people who are the shortest.

“The problem is not so much when do you start as when do you stop?” Lantos says. “If you say children in the first percentile should get the treatment, what happens when they get to the third percentile?”

Insurers are also drawn into the debate. When is short stature considered a medical condition for which treatment is reimbursable? (The drug costs about $20,000 a year, and treatment may last for more than five years.)

Those questions are a long way from settled, and endocrinologists say they are hopeful that public debate on these issues will precede widespread use of the drug.

Meanwhile, several developments could increase the availability of hGH in the near future. Orphan-drug protection for its two U.S. manufacturers will expire soon, allowing more competitors, experts say. In addition, other uses for hGH are expected to receive formal Food and Drug Administration approval, such as for Turner syndrome patients and, possibly, for those with osteoporosis, infertility or severe burns.

Now, studies are even under way to allow hGH to be administered as a nasal spray rather than as an injection, although researchers caution that this technology is at least a decade away.

But when that day arrives, some critics fear, it may be as easy to add a few inches as take off a few pounds.