Emerging pharmacologic therapies for primary sclerosing cholangitis

Area of Science:

• Hepatology and immunology
• Gastroenterology
• Drug discovery

Background:

• Primary sclerosing cholangitis (PSC) is a chronic liver disease with no approved treatments besides liver transplantation.
• Liver transplantation for PSC has a median waiting time of 12-18 years post-diagnosis.
• There is a critical unmet need for effective PSC therapies.

Purpose of the Study:

• To review and discuss emerging drug candidates for primary sclerosing cholangitis (PSC).
• To highlight novel therapeutics with the potential to address the unmet needs in PSC treatment.

Main Methods:

• Summary and discussion of novel therapeutics under development for PSC.
• Focus on drugs targeting presumed pathogenic mechanisms.
• Inclusion of preclinical and translational observations.

Main Results:

• Multiple novel therapeutics are in development for PSC.
• These agents target presumed pathogenic pathways.
• Drug development is informed by preclinical models and translational data.

Conclusions:

• Future PSC treatment strategies may involve targeting multiple pathogenic mechanisms.
• Key pathways include immunomodulation, microbiome alterations, and inflammation-related fibrosis.
• A multifaceted approach is anticipated for future PSC therapies.