Circulating indian hedgehog is a marker of the hepatocyte-TAZ pathway in experimental NASH and is elevated in humans with NASH
Mary Patricia Moore,
Xiaobo Wang,
Hongxue Shi,
Marica Meroni,
Alessandro Cherubini,
Luisa Ronzoni,
Elizabeth J. Parks,
Jamal A. Ibdah,
R. Scott Rector,
Luca Valenti,
Paola Dongiovanni,
Ira Tabas
Affiliations
Mary Patricia Moore
Department of Medicine, Columbia University Irving Medical Center, New York, NY, USA
Xiaobo Wang
Department of Medicine, Columbia University Irving Medical Center, New York, NY, USA; Corresponding authors. Addresses: Department of Medicine, Columbia University Irving Medical Center, New York, NY 10032, USA. Tel: 212-305-5669; Fax: 212-305-4834 (X. Wang); Department of Medicine, Columbia University Irving Medical Center, New York, NY 10032, USA. Tel: 212-305-9430; Fax: 212-305-4834 (Ira Tabas).
Hongxue Shi
Department of Medicine, Columbia University Irving Medical Center, New York, NY, USA
Marica Meroni
General Medicine and Metabolic Diseases, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy
Alessandro Cherubini
Precision Medicine Lab, Biological Resource Center, Department of Transfusion Medicine, Fondazione Ca’ Granda Ospedale Maggiore Policlinico Milano, Milan, Italy
Luisa Ronzoni
Precision Medicine Lab, Biological Resource Center, Department of Transfusion Medicine, Fondazione Ca’ Granda Ospedale Maggiore Policlinico Milano, Milan, Italy
Elizabeth J. Parks
Division of Gastroenterology and Hepatology, University of Missouri, Columbia, MO, USA; Department of Nutrition and Exercise Physiology, School of Medicine, University of Missouri, Columbia, MO, USA
Jamal A. Ibdah
Division of Gastroenterology and Hepatology, University of Missouri, Columbia, MO, USA; Research Service, Harry S Truman Memorial Veterans Medical Center, Columbia, MO, USA; Department of Medical Pharmacology and Physiology, School of Medicine, University of Missouri, Columbia, MO, USA
R. Scott Rector
Division of Gastroenterology and Hepatology, University of Missouri, Columbia, MO, USA; Department of Nutrition and Exercise Physiology, School of Medicine, University of Missouri, Columbia, MO, USA; Research Service, Harry S Truman Memorial Veterans Medical Center, Columbia, MO, USA
Luca Valenti
Precision Medicine Lab, Biological Resource Center, Department of Transfusion Medicine, Fondazione Ca’ Granda Ospedale Maggiore Policlinico Milano, Milan, Italy; Department of Pathophysiology and Transplantation, Universitádegli Studi di Milano, Milan, Italy
Paola Dongiovanni
General Medicine and Metabolic Diseases, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy; General Medicine and Metabolic Diseases, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan 20122, Italy. Tel: 02.5503.3467; Fax: 02.5503.4229 (P. Dongiovanni).
Ira Tabas
Department of Medicine, Columbia University Irving Medical Center, New York, NY, USA; Department of Pathology and Cell Biology, Columbia University Irving Medical Center, New York, NY, USA; Department of Physiology and Cellular Biophysics, Columbia University Irving Medical Center, New York, NY, USA; Corresponding authors. Addresses: Department of Medicine, Columbia University Irving Medical Center, New York, NY 10032, USA. Tel: 212-305-5669; Fax: 212-305-4834 (X. Wang); Department of Medicine, Columbia University Irving Medical Center, New York, NY 10032, USA. Tel: 212-305-9430; Fax: 212-305-4834 (Ira Tabas).
Background & Aims: Non-alcoholic steatohepatitis (NASH)-induced liver fibrosis is emerging as the most common cause of liver disease. For evaluation of therapies, there is a pressing need to identify non-invasive, mechanism-based biomarkers. A pro-fibrotic process relevant to human NASH involves a pathway in which a transcriptional regulator called TAZ (WWTR1) in hepatocytes induces the secretion of pro-fibrotic Indian hedgehog (IHH). We therefore reasoned that circulating IHH may be a useful mechanism-based marker to assess changes in NASH fibrosis. Methods: Circulating IHH was assessed in wild-type and hepatocyte-TAZ-silenced NASH mice and in three separate cohorts of patients with mild–moderate NASH. Results: Circulating IHH was elevated in mice with diet-induced NASH compared with chow-fed mice or with NASH mice in which hepatocyte TAZ was silenced, which is an effective means to decrease NASH fibrosis. In patients with fatty liver disease with or without NASH, NASH fibrosis was associated with increased concentrations of circulating IHH. Conclusions: The results of these analyses support further investigation to determine whether circulating IHH may be useful as a mechanism-based indicator of target engagement in anticipated future clinical trials testing NASH fibrosis therapies that block the IHH pathway. Impact and implications: Non-alcoholic steatohepatitis (NASH)-induced liver fibrosis is a common cause of liver disease. Circulating biomarkers that reflect liver fibrosis in NASH would be very useful to evaluate therapies. One mechanism of NASH fibrosis with potential as a therapeutic target involves a liver-secreted protein called Indian hedgehog (IHH). We report that circulating levels of IHH in experimental and human NASH associates with NASH and NASH-associated liver fibrosis, providing the premise for further investigation into using circulating IHH to evaluate anticipated future NASH therapies that block the IHH pathway in liver.