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Nat Rev Drug Discov. 2012 Oct;11(10):790-811. doi: 10.1038/nrd3810. Epub 2012 Sep 24.

Targeting the TGFβ signalling pathway in disease.

Author information

1
Helen Diller Family Comprehensive Cancer Center, University of California at San Francisco, San Francisco, California 94158, USA. RAkhurst@cc.ucsf.edu

Abstract

Many drugs that target transforming growth factor-β (TGFβ) signalling have been developed, some of which have reached Phase III clinical trials for a number of disease applications. Preclinical and clinical studies indicate the utility of these agents in fibrosis and oncology, particularly in augmentation of existing cancer therapies, such as radiation and chemotherapy, as well as in tumour vaccines. There are also reports of specialized applications, such as the reduction of vascular symptoms of Marfan syndrome. Here, we consider why the TGFβ signalling pathway is a drug target, the potential clinical applications of TGFβ inhibition, the issues arising with anti-TGFβ therapy and how these might be tackled using personalized approaches to dosing, monitoring of biomarkers as well as brief and/or localized drug-dosing regimens.

PMID:
23000686
PMCID:
PMC3520610
DOI:
10.1038/nrd3810
[Indexed for MEDLINE]
Free PMC Article
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