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Updated: Apr 15, 2026

A Familial Hypercholesterolemia Human Liver Chimeric Mouse Model Using Induced Pluripotent Stem Cell-derived Hepatocytes
Published on: September 15, 2018
[New agents for hypercholesterolemia]
Xavier Pintó1, María Carmen García Gómez2
1Unidad de Lípidos y Riesgo Vascular, Servicio de Medicina Interna, Hospital Universitario de Bellvitge, L'Hospitalet de Llobregat, Barcelona, España; Centro de Investigación Biomédica en Red de Fisiopatología de la Obesidad y Nutrición (CIBERobn), Universidad de Barcelona, Barcelona, España.
Insights
Many patients with high cardiovascular risk struggle to meet cholesterol goals due to medication issues. New therapies like PCSK9 inhibitors offer improved cholesterol lowering and hypercholesterolemia management.
Area of Science:
- Cardiovascular Medicine
- Pharmacology
- Metabolic Diseases
Background:
- A significant number of high cardiovascular risk patients fail to achieve therapeutic low-density lipoprotein cholesterol (LDL-C) goals.
- Suboptimal medication use, poor patient tolerance, and limited efficacy of current drugs like statins and ezetimibe contribute to this challenge.
- Novel therapeutic strategies are needed to effectively manage hypercholesterolemia and reduce cardiovascular risk.
Purpose of the Study:
- To provide an overview of recently approved and investigational therapeutic agents for hypercholesterolemia.
- To discuss the characteristics, efficacy, and safety profiles of emerging cholesterol-lowering medications.
- To highlight advancements in managing patients with elevated cardiovascular risk and lipid disorders.
Main Methods:
- Review of current literature on hypercholesterolemia treatments.
- Analysis of clinical trial data for novel lipid-lowering agents.
- Synthesis of information on the mechanisms of action and therapeutic potential of new drug classes.
Main Results:
- Proprotein convertase subtilisin kexin type 9 (PCSK9) inhibitors demonstrate significant LDL-C reduction and are a promising option for hypercholesterolemia management.
- Agents targeting apolipoprotein B synthesis and microsomal transfer protein offer new avenues for severe hypercholesterolemia, including homozygous familial hypercholesterolemia.
- Cholesteryl ester transfer protein (CETP) inhibitors show potent effects on HDL-C and LDL-C, but their cardiovascular benefits and safety require further investigation.
Conclusions:
- Emerging therapies, particularly PCSK9 inhibitors, hold significant promise for optimizing cholesterol management in high-risk patients.
- New drug classes are expanding treatment options for severe and refractory hypercholesterolemia.
- Continued research is essential to fully establish the cardiovascular benefits and safety of novel agents like CETP inhibitors.
Abstract:
An elevated proportion of high cardiovascular risk patients do not achieve the therapeutic c-LDL goals. This owes to physicians' inappropriate or insufficient use of cholesterol lowering medications or to patients' bad tolerance or therapeutic compliance. Another cause is an insufficient efficacy of current cholesterol lowering drugs including statins and ezetimibe. In addition, proprotein convertase subtilisin kexin type 9 inhibitors are a new cholesterol lowering medications showing safety and high efficacy to reduce c-LDL in numerous already performed or underway clinical trials, potentially allowing an optimal control of hypercholesterolemia in most patients. Agents inhibiting apolipoprotein B synthesis and microsomal transfer protein are also providing a new potential to decrease cholesterol in patients with severe hypercholesterolemia and in particular in homozygote familial hypercholesterolemia. Last, cholesteryl ester transfer protein inhibitors have shown powerful effects on c-HDL and c-LDL, although their efficacy in cardiovascular prevention and safety has not been demonstrated yet. We provide in this article an overview of the main characteristics of therapeutic agents for hypercholesterolemia, which have been recently approved or in an advanced research stage.
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