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The Arc of Innovation Protects Our Hearts

By Michael D. Shapiro, DO, MCR

What if we – as a health care community – focused on cardiovascular disease prevention with the same level of intensity as we do treatment?

Take the example of high cholesterol. Innovation continues to blossom here. Breakthrough drugs have helped millions of patients get their high cholesterol down to a healthy level.

A few years ago, PCSK9 inhibitors entered the market. They were a great addition to cholesterol lowering therapies and were transformational for individuals with inherited high cholesterol.

Despite ongoing access challenges, PCSK9s bring great benefit to many patients.

And around the corner is the approval of a potentially more patient-friendly medication, inclisiran. The new LDL cholesterol-lowering injectable has been approved in Europe and it’s only a matter of time before it will be approved in the U.S.

Inclisiran inhibits PCSK9 in a different fashion than currently available PCSK9 inhibitors. While the LDL (bad) cholesterol reduction is similar,  its effect lasts substantially longer. Patients receive an injection of the drug once every six months – as opposed to every 2-4 weeks. Find me a patient who isn’t excited about achieving nearly the same level of effectiveness with fewer injections.

But innovation in cholesterol management doesn’t stop with new medications. Researchers are now moving toward editing a person’s DNA to achieve low cholesterol levels.

Gene or genome editing gives scientists the ability to change one’s DNA. The approach being researched to address high cholesterol would allow beneficial changes to existing DNA to ultimately lower blood cholesterol levels substantially. This is groundbreaking for high cholesterol patients, who otherwise would be subjected to a lifetime of medications.

The concept isn’t lifted from a sci-fi film. It’s feasible and it’s where we’re headed.

The beauty of this editing is that it’s one and done. Patients with stubbornly high cholesterol could get an injection of DNA editor that would lower their cholesterol levels, and, in turn, lower their risk of heart attack and stroke indefinitely.

The procedure could benefit several patient populations, but people with familial hypercholesterolemia could be among the first. FH is present from birth and is associated with early aggressive cardiovascular disease – sometimes even after treatment with the best medications.

It’s likely that, over time, DNA editing would expand to address other conditions too.

One in four people in the United States dies from cardiovascular disease. But with continued innovation that won’t be the case forever. I feel confident that we are headed in the right direction.

Michael D. Shapiro, DO, MCR, is a practicing cardiologist and is the Fred M. Parrish Professor of Cardiology and Molecular Medicine at Wake Forest University where he directs the Center for Prevention of Cardiovascular Disease. He is also a member of the Alliance for Patient Access’ Cardiovascular Disease Working Group.

Breakthrough, an IfPA blog series, offers health care providers a voice in the ever-growing conversation about innovation and value.

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