Clinical Trials
Study of New, Non-Growth Hormone Treatment for Short Stature
By Rush University Medical Center
Sep 13, 2005, 21:05

Rush University Medical Center is participating in a clinical trial to evaluate the potential benefit of the first major innovation in 20 years for the treatment of growth failure. The drug, called Increlex, was approved by the FDA August 31 for the most severe form of short stature due to a deficiency of Insulin-like Growth Factor-1 (IGF-1). Ongoing trials will determine if the drug may be used for less severe growth disease.

Insulin-like Growth Factor-1 (IGF-1) is the proximate hormone necessary for statural growth and must be present in order for children's bones, cartilage and organs to grow normally. In healthy individuals, growth hormone is secreted into the bloodstream by the pituitary gland and binds to growth hormone receptors on liver and other cells, where it stimulates the cellular production and secretion of IGF-1 into the bloodstream.

"For decades, the only available drug treatment for short stature has been shots of growth hormone. However, patients who are deficient in IGF-1 and resistant to the effects of growth hormone do not respond well, if at all, to the shots," said Dr. Richard Levy, a pediatric endocrinologist at Rush. "Instead of using growth hormone to stimulate the production of IGF-1, the goal is to replace the IGF-1 directly."

Increlex is a genetically engineered copy of IGF-1. The purified protein has been shown to be structurally and functionally identical to natural human IGF-1. It is injected daily before a meal to provide the catalyst the body needs to grow.

The FDA's approval of Increlex is based on clinical trial data from 71 patients. Data reported at the 2004 Annual Meeting of the Endocrine Society demonstrated a statistically significant increase in growth rate over an eight-year period in response to Increlex therapy. Compared to pre-treatment growth patterns, on average, children gained an additional inch per year for each year of therapy over the course of eight years. In addition, an analysis of safety in the study concluded that long-term treatment appears to be well tolerated and has an acceptable safety profile. The most common adverse events were hypoglycemia, lipohypertrophy and tonsillar hypertrophy.

Primary IGFD afflicts an estimated 30,000 children evaluated for short stature in the United States. A child with short stature is defined as being shorter than 97.5 percent of all children the same age and gender. If untreated, Primary IGFD may lead, in children and adults, to a range of other metabolic disorders, including lipid abnormalities, decreased bone density, obesity, and insulin resistance.

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