Pediatric Growth Hormone Sparks Controversy

In a society where most kids view towering NBA stars as heroes, the craving to be bigger and taller is only natural. Now, their dreams of gaining a few extra inches may soon come true.

A recent controversial FDA decision will allow doctors to prescribe a supplemental growth hormone to short patients with completely normal hormone levels.

The medication in question is Eli Lilly’s (LLYHumatrope (search), an injected protein that stimulates growth, specifically in children. Once used only by boys and girls with serious hormone deficiencies, the drug has been approved for use in extremely short but otherwise healthy children.

The FDA’s decision to allow the sale of the drug to patients with short stature of unknown origin is of “enormous significance,” according to Dr. Arthur Caplan, Chair of the Department of Medical Ethics and Director of the Center for Bioethics (search) at the University of Pennsylvania.

“The reason this is big news is that this is one of the first times the [FDA] has allowed a drug to go on the market to treat something that is completely normal,” he said.

Humatrope, administered five to six times per week, also has its share of safety issues, according to Caplan.

Although the risk is small, “headaches, muscle pain, and even some rare forms of cancer have been linked to the drug,” he said.

Additionally, some critics point to the results of the drug’s two trials — in which patients displayed an average growth of just a few inches more than placebo users — as evidence that the drug is not worth its $10,000 to $20,000 average annual price tag.

Supporters of administering Humatrope to patients with normal hormone levels emphasized that very short children are often subjected to social problems.

"Children with short stature have a number of disadvantages compared to their peers, such as juvenilization, teasing and bullying, exclusion from peer activities, and loss of independence or overprotection,” said an Eli Lilly and Company representative.  “Height gain, as a result of treatment, can make a huge difference in a child’s life.” 

According to Dr. Abby Hollander, an associate professor of pediatrics at the Washington University School of Medicine in St. Louis, the drug is generally offered only on a case-by-case basis to the shortest 1.2 percent of boys and girls, who are expected to reach adult heights of less than 5 feet 3 inches and 4 feet 11 inches, respectively.

These intended recipients likely suffer from undetectable or unknown growth disorders, she added.

Dr. Pinchas Cohen, professor and chief of endocrinology at UCLA, agreed that such short stature is an abnormality stemming from an unidentified disorder.

“I believe that this is an important step for the medical community, but that it will require physicians to maintain a high standard of care in terms of evaluating children with short stature,” he said.

“In terms of safety,” he added, growth hormone “is a naturally circulating hormone that we all have. As a drug, it is one of the safest we prescribe.”

Libby Kershner, 20, a sociology major at Brandeis University, couldn’t agree more.

She received daily Humatrope injections from age 9 to 14, and at 5-feet-2-½ inches tall, is now nearly 6 inches taller than doctors had predicted.

Once the victim of relentless ridicule, Libby said she is now considered an equal among her peers.

“Not only was I physically taller, but I was emotionally taller as well,” she said, referring to her renewed self-esteem after completing the Humatrope regimen.

“Taking and benefiting from the [growth hormone] gave me confidence that I couldn't have gotten any other way.”

In a statement from Eli Lilly, the company said Humatrope will “make an important difference in the growth rate and final height of children with short stature whose growth failure has not yet been determined.”

Nonetheless, Dr. Caplan and others who oppose Humatrope’s new indication argued that preaching tolerance can prevent the social problems faced by Libby and children like her. He predicted, however, that there will likely be strong demand for growth hormone in the future, as society continues to embrace tall basketball players and other athletes as idols.

“Bigger may not be better, but the pursuit of better is likely to get a whole lot bigger in the years to come,” he said.