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The Role of Antisense Oligonucleotide Therapy in Patients with Familial Hypercholesterolemia: Risks, Benefits, and Management Recommendations

  • New Drugs Approved for Homozygous FH (SS Virani, Section Editor)
  • Published:
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Abstract

Antisense oligonucleotide therapy is a promising approach for the treatment of a broad variety of medical conditions. It functions at the cellular level by interfering with RNA function, often leading to degradation of specifically targeted abnormal gene products implicated in the disease process. Mipomersen is a novel antisense oligonucleotide directed at apolipoprotein (apoB)-100, the primary apolipoprotein associated with low-density lipoprotein cholesterol (LDL-C), which has recently been approved for the treatment of familial hypercholesterolemia. A number of clinical studies have demonstrated its efficacy in lowering LDL-C and apoB levels in patients with elevated LDL-C despite maximal medical therapy using conventional lipid-lowering agents. This review outlines the risks and benefits of therapy and provides recommendations on the use of mipomersen.

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Conflict of Interest

Anandita Agarwala declares no conflict of interest.

Peter Jones received personal fees for serving on the scientific advisory board for Merck and Amgen and the medical advisory board for Atherotech. He is the Chief Science Officer for the National Lipid Association.

Vijay Nambi has a provisional patent on the use of biomarkers in heart failure with Roche; research collaboration with GE; received personal fees from Sanofi for serving on the regional advisory board; received grants from Gillson Longenbaugh Foundation, Gulf Coast Regional Foundation, Methodist Hospital Research institute, and NIH/ NHLBI K23; national monitor for Anthera and research collaboration with Tomtec.

Human and Animal Rights and Informed Consent

This article does not contain any studies with human or animal subjects performed by any of the.

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Authors and Affiliations

  1. Department of Medicine, Baylor College of Medicine, Michael E. DeBakey Veterans Affairs Medical Center, One Baylor Plaza, Houston, TX, 77030, USA

    Anandita Agarwala

  2. Department of Medicine, Center for Cardiovascular Disease Prevention, Baylor College of Medicine, Methodist DeBakey Heart and Vascular Center, 6565 Fannin St, STE B 160, Houston, TX, 77030, USA

    Peter Jones & Vijay Nambi

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  1. Anandita Agarwala
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  2. Peter Jones
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Correspondence to Vijay Nambi.

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This article is part of the Topical Collection on New Drugs Approved for Homozygous FH

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Agarwala, A., Jones, P. & Nambi, V. The Role of Antisense Oligonucleotide Therapy in Patients with Familial Hypercholesterolemia: Risks, Benefits, and Management Recommendations. Curr Atheroscler Rep 17, 467 (2015). https://doi.org/10.1007/s11883-014-0467-4

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  • DOI: https://doi.org/10.1007/s11883-014-0467-4

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