Abstract
Purpose of review:
The only currently approved treatment for primary sclerosing cholangitis (PSC) is liver transplantation, with a median time to transplant of 12-18 years after diagnosis. There are a number of emerging drugs that have the potential to meet this critically unmet need that will be summarized and discussed herein.
Recent findings:
Although the cause of PSC is unknown, there are a number of novel therapeutics under development. These drugs target presumed pathogenic mechanisms largely extrapolated from ex-vivo and in-vivo preclinical models, as well as translational observations.
Summary:
Future therapeutic strategies for PSC may include a multitude of complex pathogenic mechanisms encompassing pathways of immunomodulation, the microbiome and inflammation-related fibrosis.
MeSH terms
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Anti-Bacterial Agents / therapeutic use
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Chenodeoxycholic Acid / analogs & derivatives
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Chenodeoxycholic Acid / therapeutic use
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Cholangitis, Sclerosing / drug therapy*
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Cholangitis, Sclerosing / microbiology
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Clinical Trials as Topic / methods
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Fecal Microbiota Transplantation
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Fibric Acids / therapeutic use
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Gastrointestinal Microbiome / drug effects
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Humans
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Immunologic Factors / therapeutic use
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Molecular Targeted Therapy / methods*
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Molecular Targeted Therapy / trends
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Organic Anion Transporters, Sodium-Dependent / antagonists & inhibitors
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Probiotics / therapeutic use
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Symporters / antagonists & inhibitors
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Ursodeoxycholic Acid / therapeutic use
Substances
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Anti-Bacterial Agents
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Fibric Acids
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Immunologic Factors
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Organic Anion Transporters, Sodium-Dependent
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Symporters
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obeticholic acid
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Chenodeoxycholic Acid
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sodium-bile acid cotransporter
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Ursodeoxycholic Acid