A Self-Perpetuating Treatment
Humatrope and the Medicalization of Social Problems
by Ross Feldberg

What is “normal”? Most of us would agree that individuals who experience constant pain, or whose physical limitations prevent them from living a full life, suffer from an abnormality. We strive to help them attain a “normal” life. But what about those human traits which show a so-called normal distribution in the population? Does lying far from the mean of such a distribution classify one as abnormal? This may sound like an abstract question, but the potential economic and social implications of our answer to this question lie at the heart of a profound change in the way we view our biology.

Consider, for example, the distribution of human height — a trait determined by multiple and interacting genes as well as by the developmental environment. As we expect in any distribution, there are individuals who lie at the end of the distribution and are thus much shorter or much taller than the mean. This is a typical human condition, although it is interesting that physicians have assigned a special name to those very short individuals who have no apparent organic defect: they are characterized as “idiopathic short stature” (ISS). The significance of this is that assigning a medical term to a normal human condition is the first step in justifying its treatment as a pathology.

The history of the medical treatment of individuals with genetic deficiencies in growth hormone is an interesting
one. As early as the 1950s, biochemists began extracting the trace amounts of human growth hormone present in hypothalamuses taken from cadavers. This scarce and very costly material was then used to treat individuals who displayed a genetic mutation which rendered them unable to produce growth hormone. The cost of this material, as well as the eventual discovery that the preparations were potentially contaminated with prions that could lead to neurodegenerative disorders, meant that its use was limited to treating individuals with a clear
genetic hormone deficiency.

However, when it was announced in the early 1980s that human growth hormone had been copied and could be
produced artificially in the lab, there was a buzz of excitement. Potentially unlimited amounts of HGH, free of prion
contamination, could now be produced at a far lower cost than before. However, these benefits also brought new complications, and it should not have been a surprise when, in the October 27, 1983 issue of the New England Journal of Medicine, it was reported that artificially produced human growth hormone had been used to treat children without any deficiency in growth hormone, but who were simply very short for their age.

I raised the implications of this in an article in the May/June 1985 issue of Science for the People magazine. What I said then holds equally true today:

So what we have is a case of apparently healthy children being treated with a powerful hormone in an attempt to push them toward the average height. Is shortness per se a sickness? Shortness can indeed be a social problem. Short individuals do suffer a variety of forms of discrimination. But is the appropriate response to that [social problem] to make those individuals change or to make the society change? Medicalizing this “problem” transfers the responsibility for the discrimination away from those doing the discrimination and to the victims. Are we to deal with discrimination by making short people taller or black people whiter? It would be laughable if it weren’t so frightening.

Now, fast forward to July 2003. The FDA makes headlines with its approval of Eli Lilly’s recombinant human growth hormone, Humatrope, for very short children without hormone deficiencies. Based on several studies, the FDA concluded that the treatment was safe and more or less effective. Achieving the full effect required six shots a week for four to five years at a cost of about $20,000 per year. With this regimen, ISS children receiving the injections grew between two and four inches more than a control set of ISS children receiving placebo
injections (making me wonder what institutional review board accepted a protocol which required healthy children to receive six injections of placebo per week for four to five years).

The FDA suggested limiting the use of this drug to otherwise normal U.S. children who fall more than 2.25 standard deviations below the average height for their age and sex (i.e., the lowest 1.2% in a normal distribution). One can easily understand why the “problem” of short stature has been an attractive challenge for the pharmaceutical industry and the medical profession for some years. It is estimated that there are about 400,000 children in the U.S. who will fall below the FDA’s suggested cut-off and will thus be candidates for this “therapy”. Because the treatment will require six injections per week for four to five years, and will cost, on average, $20,000 per year, Eli Lilly estimates that at most 40,000 children will opt for this treatment. Even if Lilly is optimistic by a factor of two, 20,000 children using $20,000 of Humatrope each means $400 million entering the medical industry annually — and all for a “problem” that is not medical to begin with.

Lilly has assured the FDA that it will maintain tight control over Humatrope’s availability, allowing it to be
prescribed only by specialists in pediatric endocrinology and to be supplied only by regulated pharmacies. But if Viagra is any guide, it will only be a matter of time before parents whose children fall below some internet standard of healthy height will be bombarded with ads imploring them to keep their children’s best interests at heart by asking their doctors for Humatrope. And consider the scenario in which significant numbers of very short children are treated with Humatrope: won’t a whole new group of individuals now fall more than 2.2 standard deviations below the chemically adjusted mean? Will these individuals now become candidates for “therapy”? If so, this would be every pharmaceutical CEO’s dream — a drug, which, when administered to a treatment group, automatically creates a fresh market of previously untreated and healthy individuals who now “need” their treatment.

Being very short is a real disadvantage in our society, and it is hard to deny a concerned parent’s wish that their child have the best chances in life. But ultimately we need to remember that discrimination is a social, not an individual, problem, and employing medical solutions to social problems may not be the wisest path. It is important that we use every venue to remind people that the short- and long-term consequences of unnecessary medical procedures are usually not as predictable as we like to think — and chemical solutions to social problems are likely to engender their own new problems.

Ross Feldberg is Assistant Professor of Biology at Tufts University.