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Published on: February 15, 2019
Novel treatment approaches to fibrosis in scleroderma
1Department of Internal Medicine III and Institute for Clinical Immunology, University of Erlangen-Nuremberg, Krankenhausstrasse 12, 91054 Erlangen, Germany.
Abstract:
The molecular mechanisms leading to tissue fibrosis were only poorly understood in the past, and even today the cause or trigger of systemic sclerosis is still unknown. Remarkable breakthrough findings have been obtained regarding the identification of key molecules, key cellular mechanisms, and key intracellular signaling cascades, which mediate the perpetuation of fibrosis rather than trigger it. These findings have true translational implications, because modifiers of these key mediators and key mechanisms are often in clinical use in other disease indications, such as cancer. This article summarizes the clinical and preclinical evidence of examples of these novel antifibrotic treatment approaches in systemic sclerosis, including stem cell transplantation, modifiers of transforming growth factor-beta1 signaling, intravenous immunoglobulins, tyrosine kinase inhibitors, and histone deacetylase inhibitors.
Insights
Researchers are uncovering new antifibrotic treatments for systemic sclerosis by targeting key molecular pathways. These novel approaches, including stem cell therapy and targeted inhibitors, show promise for managing fibrosis progression.
Area of Science:
- Fibrosis research
- Systemic sclerosis pathogenesis
- Translational medicine
Background:
- Molecular mechanisms of tissue fibrosis were poorly understood.
- The exact trigger for systemic sclerosis remains unknown.
- Recent advances identified key mediators of fibrosis perpetuation.
Purpose of the Study:
- To summarize novel antifibrotic treatment approaches for systemic sclerosis.
- To review clinical and preclinical evidence for these therapies.
- To highlight the translational potential of targeting fibrosis mediators.
Main Methods:
- Review of clinical and preclinical studies.
- Identification of key molecular mediators and signaling pathways in fibrosis.
- Analysis of existing drugs (e.g., cancer therapies) for antifibrotic potential.
Main Results:
- Key molecules, cellular mechanisms, and signaling cascades mediating fibrosis perpetuation have been identified.
- Several novel antifibrotic strategies show promise.
- Existing drugs targeting these pathways have translational implications.
Conclusions:
- Novel antifibrotic treatments for systemic sclerosis are emerging.
- Targeting identified mediators offers therapeutic potential.
- Approaches include stem cell transplantation, TGF-β1 pathway modifiers, IVIg, TKIs, and HDAC inhibitors.
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