A glimpse into future cholesterol control: A time without daily pills perhaps?

For millions of people worldwide, the daily ritual of taking a statin or other drugs to manage cholesterol levels is a fact of life. These powerful medications have dramatically reduced the risk of heart attacks and strokes, thereby transforming cardiovascular healthcare. However, the hassle of having to take these medicines every day is a cumbersome infringement on life itself.

- DR B. J. C. PERERA

Now, imagine a future where the burden of daily pills for this purpose could be replaced by a single, or at most, a few injections per year. This is NOT science fiction; it is the audacious goal of a new frontier in medicine, with revolutionary drugs currently undergoing rigorous clinical trials. These cutting-edge therapies are designed to act directly in the liver, targeting the very genetic instructions or enzymes responsible for producing "bad" cholesterol, potentially offering a more profound and long-lasting solution than ever before.

This appears to be the dawn of a new era with medicines that employ gene-silencing and enzyme-neutralisation techniques. The excitement surrounding these new treatments stems from their innovative approaches. Unlike traditional statins, which work by blocking an enzyme involved in cholesterol production, these newer drugs go further upstream, interfering with the body's internal machinery that regulates cholesterol at a more fundamental level.

Two primary classes of these emerging therapies are making waves at present:

1. PCSK9 Inhibitors: A Deeper Dive

There are a few currently approved drugs that inhibit PCSK9 gene. But they require injections every two to four weeks. These antibody-based drugs work by binding to and neutralising a protein called PCSK9 (Proprotein Convertase Subtilisin/Kexin type 9), which acts like a "destroyer" of LDL receptors on the surface of liver cells. These LDL receptors are crucial for grabbing "bad" LDL cholesterol from the bloodstream and bringing it into the liver for processing and removal. By blocking PCSK9, these inhibitors allow more LDL receptors to remain active on liver cells, significantly boosting the liver's capacity to clear LDL cholesterol from the blood.