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THROMBOPOIETIN: FROM CLONING TO CLINIC

Kaushansky, Kenneth

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Pathology - Journal of the RCPA: 2010 - Volume 42 - Issue - p S39
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Thrombopoietin was cloned by a functional expression strategy in 1994. Since then many of the biological characteristics of the molecule have been elucidated, the signal transduction pathways employed by the thrombopoietin receptor identified, and the wider than anticipated range of activities, including effects on haematopoietic stem cells, determined. Translation of these finding into clinical utility, however, was delayed because of the propensity of an altered form of the molecule to induce an immune reaction that cross reacted with the native hormone. Because of this unanticipated untoward reaction, attempts to identify small molecules that bind to and stimulate the thrombopoietin receptor (TPO mimics) were undertaken by a number of groups. These efforts have now been productive, and the clinical experience with two such molecules, AMG531 and Eltrombopag, will be reviewed. Thus, it took 14 years but we are now poised to rationally intervene in patients with severe, life‐threatening thrombocytopenia.

© 2010 Royal College of Pathologists of Australasia