Targeting the TGFβ signalling pathway in disease

Many drugs that target transforming growth factor-beta (TGF beta) 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 TGF beta signalling pathway is a drug target, the potential clinical applications of TGF beta inhibition, the issues arising with anti-TGF beta 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.