Understanding siRNA: A New Hope for High Cholesterol
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High cholesterol and other lipid disorders (called dyslipidemia) are a big cause of heart disease worldwide. While lifestyle changes and common medicines like statins help, not everyone responds well to them. Now, a new type of treatment called small interfering RNA (siRNA) therapy is offering fresh hope.
What Is siRNA and How Does It Work?
siRNA stands for “small interfering RNA.” It’s a kind of molecule that can “silence” or block the production of specific proteins in the body. Scientists design siRNA to target the genes responsible for making proteins that raise harmful fats in the blood—like LDL (bad cholesterol) or certain apolipoproteins. When these genes are turned off, cholesterol and fat levels drop.
How Effective Is siRNA for Cholesterol?
Several siRNA-based drugs have been tested in clinical trials, and the results look promising. For example, one of the most well-known siRNA drugs, Inclisiran, has shown to reduce LDL cholesterol by around 44%. It also helped lower other harmful lipids like apolipoproteins and triglycerides.
Other siRNA drugs like Lepodisiran, Olpasiran, and Plozasiran have been used to treat specific types of dyslipidemia. Some of these reduced certain fat levels by over 75%. One standout drug, ARO-APOC3, lowered triglyceride levels by more than 60% in some patients.
Are There Any Side Effects?
Like all medicines, siRNA therapies can have side effects. The most common ones include:
- Nasal infections (nasopharyngitis)
- Injection site pain or swelling
- New or worsening diabetes
- Back pain
- High blood pressure
In rare cases, serious side effects like stroke or heart attack were reported. Around 1.4% of patients in the studies died, although it’s unclear if all these deaths were directly linked to the treatment.
How Is siRNA Given?
Most siRNA therapies are given as an injection under the skin (subcutaneous). The good news? They are usually needed only once every few months. This is a big advantage compared to daily pills like statins.
Who Can Benefit the Most?
People who:
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Have very high cholesterol that doesn’t improve with regular treatment
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Can’t tolerate statins due to side effects
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Have rare or genetic lipid disorders
may benefit the most from siRNA therapy.
What Are the Challenges?
While siRNA therapy looks promising, there are still some hurdles:
- It’s expensive and not widely available yet
- Long-term safety is still being studied
- Delivering siRNA into the right cells in the body is tricky
Also, these therapies haven’t yet been included in standard cholesterol treatment guidelines, so doctors may not prescribe them as a first option.
The Bottom Line
siRNA therapy is an exciting new tool for lowering harmful blood fats. It’s especially helpful for people who don’t respond well to current treatments. While more research is needed to fully understand its safety and long-term benefits, it could soon become a regular part of how we treat high cholesterol and protect heart health.
If you or a loved one struggles with managing cholesterol, talk to your doctor about whether siRNA-based treatment might be right for you in the future.