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Recombinant insulin-like growth factor-1 as a therapy for IGF-1 deficiency in renal failure

Ross G Clark1

  • 1Tercica, 651 Gateway Boulevard, Suite 950, South San Francisco, CA 94080, USA. ross.clark@tercica.com

Pediatric Nephrology (Berlin, Germany)
|February 1, 2005
PubMed

Insights

Children with short stature due to renal disease may benefit from insulin-like growth factor-1 (IGF-1) therapy. IGF-1, not growth hormone (GH), appears to be the primary driver of growth, suggesting new treatment strategies.

Area of Science:

  • Pediatric Endocrinology
  • Growth Hormone Axis
  • Metabolic Disorders

Background:

  • Renal disease in children impairs growth by disrupting the growth hormone (GH) and insulin-like growth factor (IGF) axis.
  • Short stature in these children is often linked to IGF-1 deficiency (IGFD) rather than GH deficiency.
  • Recent research highlights the critical role of IGF-1 in human growth.

Purpose of the Study:

  • To re-evaluate the significance of IGF-1 and IGFD in pediatric growth.
  • To explore the therapeutic potential of IGF-1 in children with renal disease-related short stature.
  • To compare the efficacy of different growth-promoting therapies.

Main Methods:

  • Review of experimental data from rodent pharmacology studies.
  • Analysis of studies on patients with GH receptor dysfunction and primary IGFD.
  • Examination of gene knockout studies in mice.
  • Consideration of combined GH and IGF-1 effects.

Main Results:

  • IGF-1 has demonstrated effectiveness as a growth-promoting therapy in various models and patient groups.
  • IGF-1 is identified as the major hormone regulating growth, surpassing GH in importance.
  • Combined GH and IGF-1 actions are necessary for certain growth effects.
  • Recombinant human (rh)IGF-1 yields significant growth responses in primary IGFD.
  • Recombinant human GH (rhGH) is optimal for combined GH and IGF-1 deficiency.

Conclusions:

  • Children with renal failure-induced short stature are GH sufficient with some GH receptor signaling.
  • rhIGF-1 or a combination of rhIGF-1 and rhGH are logical therapeutic options for these children.
  • Clinical trials are warranted to test rhIGF-1-based therapies in pediatric renal disease-related short stature.

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