New Treatments for Healthy Cholesterol Levels

Quincy AdamHigh Cholesterol Treatments

Cholesterol Gene Therapy
Lifestyle changes and medication help most people lower their cholesterol. But some people still have cholesterol high, even when they’ve taken steps to lower it.

Fortunately, new therapies show great promise in the ability to lower LDL cholesterol, and may be available within the next few years.

Some Individuals Have a Rare Genetic Mutation

Many pharmaceutical companies are in a race to develop a new type of cholesterol treatment that could be more effective than the medicines currently available.

This new drug class based on the finding that some people have a rare genetic mutation that keeps their LDL cholesterol levels very low, regardless of their lifestyle choices. Studies have shown that people with this particular genetic mutation develop heart disease as much as 88 percent less frequently than members of the average population. The gene responsible for this amazing ability of the body to remove LDL cholesterol is known as PCSK9. PCSK9 has become the target of a number of clinical studies focusing on healthy cholesterol levels.

Cholesterol Gene Therapy Awaits FDA Approval

PCSK9 drugs could have a significant benefits for patients who continue to struggle with high cholesterol. As of now, PCSK9 drugs under development must be delivered by injection. However, hope for a pill which can carry the “good” genetic sequence to its target in the liver remains high for some researchers.

An article published in Nature points to more studies of people with PCSK9 mutations, and notes some exciting possibilities if this new drug class passes the final safety test in humans. For example, their studies of people born with the gene indicate that patients may be advised to begin treatment at a much earlier age than with traditional statin medications. Individuals who carry the mutation for very low LDL levels have these healthy cholesterol levels their entire lives, likely adding to the reasons why their risk of heart disease is so low.

While promising, harnessing the PCSK9 mutation gene for to lower cholesterol is still in the early phases of testing, and complications are present with other gene therapies that require transplantation. However, these offer hope to thousands who battle high cholesterol and are at risk of developing heart disease.


1Ding Q, Strong A, Patel KM, et al. Permanent alteration of PCSK9 with in vivo CRISPR-Cas9 genome editing. Circ Res. 2014;115(5):488-92. http://www.ncbi.nlm.nih.gov/pubmed/24916110?dopt=Abstract. Accessed March 11, 2015.2Van craeyveld E, Jacobs F, Gordts SC, De geest B. Gene therapy for familial hypercholesterolemia. Curr Pharm Des. 2011;17(24):2575-91. http://www.ncbi.nlm.nih.gov/pubmed/21774774. Accessed March 13, 2015