all signs point to a near future where genetic diseases become entirely curable.
Mon, Aug 1 at 1:37 PM
What if we had a permanent fix for one of the leading contributors to heart disease, the world’s single biggest killer?
Heart disease accounts for roughly 16% of total deaths globally each year. It also costs the US economy roughly $219 billion a year.
One of the main factors for developing heart disease is high levels of cholesterol in the bloodstream. That’s why more than 200 million people worldwide regularly take statins, the leading medication for lowering cholesterol levels.
These drugs have proved effective, but they require consistent use and can result in side-effects.
We’re now closer than ever to a solution.
Scientists have long known a gene that codes for a protein called PCSK9 plays a key role in regulating cholesterol levels in the bloodstream. Earlier this month, a revolutionary new CRISPR-based therapy that can switch off the PCSK9 gene started its first human trials.
In today’s blog, I’ll explain what this exciting new treatment could mean for human health and longevity, and examine the broader potential for this new approach to gene-editing in humans.
Let’s dive in…
PCSK9 AND HEART HEALTH
The role of PCSK9 in heart health was first established in the mid-2000s by pioneering work from researchers at the University of Texas Southwestern Medical Center. They discovered that people with mutations in the PCSK9 gene that inhibit the protein’s production had remarkably low levels of low-density lipoprotein (LDL), otherwise known as “bad” cholesterol.
It turned out that levels of PCSK9 significantly impact cholesterol levels. That’s because the protein prevents the recycling of LDL receptors in the liver that are responsible for filtering bad cholesterol from the bloodstream. High levels of PCSK9 cause the overall number of receptors to drop and cholesterol levels to rise.
And the impact of this single protein is massive. In a subsequent study, the UT Southwestern researchers discovered that mutations in the PCSK9 gene were associated with 15%-to-28% reductions in LDL levels and a 47%-to-88% lower risk of heart disease.
This set off a scramble to find drugs that could target this troublesome protein. However, that has proven challenging due to a scarcity of binding sites on its surface that small molecules could latch onto to disrupt its function.
One approach that has shown promise is the use of monoclonal antibodies: laboratory-grown mimics of natural antibodies that can be targeted at a specific molecule. There are two FDA-approved monoclonal antibodies that target PCSK9 called alirocumab and evolocumab, and both cut patients’ risk of cardiovascular events in half.
However, these drugs are much more expensive than statins due to their complicated production process. During their first year on the market, only one third of patients with a prescription actually received the therapies due to insurance rejections or high copays.
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