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New targets in and potential treatments for cholesterol gallstone disease
1Auckland University of Technology, Division of Health Practice, Akoranga Campus, Northcote, Auckland, New Zealand. s.doggrell@xtra.co.nz
Abstract:
Gallstone disease is very common among American Indians and Hispanics, and approximately 20 million patients are treated for this disease annually in the US. Bile acid receptor (nuclear farnesoid X receptor; FXR) knockout mice fed a lithogenic diet are more susceptible to gallstone disease than wild-type mice. The C57L mouse is also susceptible to gallstone formation when fed a lithogenic diet, and in this model, the small-molecule FXR agonist GW-4064 prevents the precipitation of cholesterol. Bile acids (eg, P-muricholic acid) and their derivatives are also being developed as FXR agonists. Fatty acid bile acid conjugates have the potential to prevent and reverse cholesterol crystallization. Furthermore, agents that increase the expression of selected hepatocyte bile acid transporters may also be useful in the treatment of gall bladder disease.
Insights
Gallstone disease affects millions annually. Targeting the bile acid receptor (FXR) with agonists like GW-4064 or specific bile acids shows promise in preventing cholesterol crystal formation and treating gallstone disease.
Area of Science:
- Hepatology
- Gastroenterology
- Molecular Endocrinology
Background:
- Gallstone disease is prevalent, particularly in certain ethnic groups, affecting millions of Americans.
- The nuclear farnesoid X receptor (FXR) plays a role in bile acid metabolism and gallstone formation.
- Genetic susceptibility, like in C57L mice, highlights the need for therapeutic targets.
Purpose of the Study:
- To investigate the role of FXR in gallstone susceptibility.
- To evaluate the efficacy of FXR agonists in preventing cholesterol precipitation.
- To explore novel therapeutic strategies for gallstone disease.
Main Methods:
- Utilizing FXR knockout mice and C57L mouse models fed a lithogenic diet.
- Administering small-molecule FXR agonists (e.g., GW-4064).
- Investigating the effects of bile acids and their derivatives on cholesterol crystallization.
Main Results:
- FXR knockout mice exhibit increased susceptibility to gallstone formation.
- GW-4064 treatment prevents cholesterol precipitation in susceptible mouse models.
- Certain bile acids and fatty acid conjugates demonstrate potential in managing cholesterol crystallization.
Conclusions:
- FXR activation is a viable therapeutic strategy for gallstone disease.
- Small-molecule FXR agonists and specific bile acid derivatives show promise for prevention and treatment.
- Modulating hepatocyte bile acid transporters may offer additional treatment avenues.
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