Chenodeoxycholic Acid: An Update on Its Therapeutic Applications.

Area of Science:

• Biochemistry
• Endocrinology
• Pharmacology

Background:

• Chenodeoxycholic acid (CDCA) is a primary bile acid synthesized from cholesterol in the liver.
• CDCA undergoes conjugation and intestinal metabolism, yielding compounds like ursodeoxycholic acid (UDCA) and lithocholic acid (LCA).
• CDCA acts as a ligand for the farnesoid X receptor (FXR), while LCA activates GPBAR1 (TGR5).

Purpose of the Study:

• To review the therapeutic applications and mechanisms of action of CDCA.
• To highlight the efficacy of CDCA in treating cerebrotendinous xanthomatosis (CTX).
• To explore potential roles of CDCA in metabolic regulation.

Main Methods:

• Literature review of CDCA's biochemical properties, clinical uses, and molecular interactions.
• Analysis of studies on CDCA treatment for gallstone dissolution and CTX.
• Examination of CDCA's effects on cholesterol metabolism and hormone release.

Main Results:

• CDCA was previously used for gallstone dissolution but had limited efficacy and side effects.
• CDCA is now an approved orphan indication for CTX, effectively reducing cholestanol levels and stabilizing neurological symptoms.
• CDCA may inhibit HMG CoA reductase and promote GLP-1 release in diabetic patients.

Conclusions:

• CDCA's therapeutic utility has shifted from gallstone dissolution to the treatment of rare metabolic disorders like CTX.
• CDCA is a crucial therapy for CTX, addressing the underlying deficiency in bile acid synthesis.
• Further research may uncover additional metabolic benefits of CDCA, including its role in glucose homeostasis.