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Investigational therapies for hypercholesterolemia
Gerald H Tomkin1, Daphne Owens1
1a Diabetes Institute of Ireland , Beacon Clinic and Trinity College , Dublin 2 , Ireland.
Insights
New therapeutic targets and drugs are emerging to combat dyslipidemia, a major risk factor for cardiovascular disease. This review explores novel pathways and treatments, including antisense RNA, microRNA, and monoclonal antibodies, offering hope for improved clinical outcomes.
Area of Science:
- Biochemistry
- Pharmacology
- Cardiovascular Medicine
Background:
- Cardiovascular diseases (CVD) pose a significant global health burden.
- Dyslipidemia is a key modifiable risk factor for CVD, necessitating improved therapeutic strategies.
- Current treatments for dyslipidemia have limitations, highlighting the need for novel approaches.
Purpose of the Study:
- To review recent advancements in understanding cholesterol and triglyceride metabolism.
- To examine emerging therapeutic targets and novel drug candidates for dyslipidemia.
- To assess the clinical trial data and potential of new treatments for cardiovascular risk reduction.
Main Methods:
- Literature review of recent scientific publications and clinical trial results.
- Analysis of new pathways in lipid metabolism.
- Examination of novel therapeutic agents, including small molecules, antisense RNA, microRNA, and monoclonal antibodies.
Main Results:
- Identification of novel molecular targets in lipid metabolism.
- Evaluation of new drugs demonstrating efficacy in preclinical and early clinical studies.
- Review of approved and investigational therapies showing promise in reducing cardiovascular endpoints.
Conclusions:
- Emerging targets and therapies offer significant potential for improving dyslipidemia management.
- Antisense RNA, microRNA, and monoclonal antibodies represent promising new modalities.
- The field of dyslipidemia therapeutics is rapidly evolving, with a focus on novel mechanisms to combat atherosclerosis.
Introduction:
Cardiovascular morbidity and mortality are of increasing concern, not only to patients but also to the health care profession and service providers. The preventative benefit of treatment of dyslipidaemia is unquestioned but there is a large, so far unmet need to improve clinical outcome. There are exciting new discoveries of targets that may translate into improved clinical outcome. Areas covered: This review highlights some new pathways in cholesterol and triglyceride metabolism and examines new targets, new drugs and new molecules. The review includes the results of recent trials of relatively new drugs that have shown benefit in cardiovascular endpoint outcomes, drugs that have been licenced without endpoint trials yet available and new drugs that have not yet been licenced but have produced exciting results in animal studies and some in early phase 2 human studies. Expert opinion: The new areas that have been discovered as the cause of dyslipidaemia have opened up a host of new targets for new drugs including antisense RNA's, microRNA's and human monoclonal antibodies. The plethora of new targets and new drugs has made it an extraordinarily exciting time in the development of therapeutics to combat atherosclerosis.