CHICAGO -- Rush University Medical Center is participating ina clinical trial to evaluate the potential benefit of the first majorinnovation in 20 years for the treatment of growth failure. The drug,called Increlex, was approved by the FDA August 31 for the most severeform of short stature due to a deficiency of Insulin-like GrowthFactor-1 (IGF-1). Ongoing trials will determine if the drug may be usedfor less severe growth disease.
Insulin-like Growth Factor-1(IGF-1) is the proximate hormone necessary for statural growth and mustbe present in order for children's bones, cartilage and organs to grownormally. In healthy individuals, growth hormone is secreted into thebloodstream by the pituitary gland and binds to growth hormonereceptors on liver and other cells, where it stimulates the cellularproduction and secretion of IGF-1 into the bloodstream.
"Fordecades, the only available drug treatment for short stature has beenshots of growth hormone. However, patients who are deficient in IGF-1and resistant to the effects of growth hormone do not respond well, ifat all, to the shots," said Dr. Richard Levy, a pediatricendocrinologist at Rush. "Instead of using growth hormone to stimulatethe production of IGF-1, the goal is to replace the IGF-1 directly."
Increlexis a genetically engineered copy of IGF-1. The purified protein hasbeen shown to be structurally and functionally identical to naturalhuman IGF-1. It is injected daily before a meal to provide the catalystthe body needs to grow.
The FDA's approval of Increlex is basedon clinical trial data from 71 patients. Data reported at the 2004Annual Meeting of the Endocrine Society demonstrated a statisticallysignificant increase in growth rate over an eight-year period inresponse to Increlex therapy. Compared to pre-treatment growthpatterns, on average, children gained an additional inch per year foreach year of therapy over the course of eight years. In addition, ananalysis of safety in the study concluded that long-term treatmentappears to be well tolerated and has an acceptable safety profile. Themost common adverse events were hypoglycemia, lipohypertrophy andtonsillar hypertrophy.
Primary IGFD afflicts an estimated 30,000children evaluated for short stature in the United States. A child withshort stature is defined as being shorter than 97.5 percent of allchildren 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, andinsulin resistance.
Increlex is manufactured by Tercica, Inc., abiopharmaceutical company focused on the development andcommercialization of products to improve endocrine health. For furtherinformation on Tercica please visit www.tercica.com.
For more information about Pediatric Endocrinology services at Rush, please call Dr. Levy's office at 312-942-8989.
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