Gene-editing treatments could eventually offer lasting protection against high cholesterol and other widespread conditions, reducing the need for daily medication. But because genetic changes may be difficult to reverse, researchers say long-term safety must be established before these therapies are used broadly.
In a perspective published in JAMA Internal Medicine, University of Michigan Health Frankel Cardiovascular Center cardiologists Eric D. Smith and Venkatesh L. Murthy set out factors doctors should weigh when considering genomic therapies. These include how serious the condition is, whether existing treatments work, how durable the intervention is, the risks of delivering it, and whether patients can be monitored over many years.
Gene therapies can supply a working gene, silence harmful genetic activity or directly alter DNA. Some are designed as one-time treatments, unlike conventional medicines that usually need to be taken repeatedly. However, a single administration does not guarantee lifelong benefits. Many therapies use modified viruses to deliver genetic instructions, and an immune response can make repeat treatment difficult if the effects fade.
The researchers note that gene therapies currently generally target somatic cells, rather than reproductive cells, so they are not intended to pass genetic changes on to future generations. While these treatments have shown important results for some serious conditions, using them for common diseases presents a different balance of risks and benefits.
High cholesterol, heart disease, obesity and diabetes already have established treatments that can be adjusted or stopped if a patient develops side effects. A permanent genetic intervention may not offer that flexibility. And even a rare complication could affect many people if a treatment were given to millions.
Early studies are investigating whether a single genetic treatment can produce sustained reductions in LDL cholesterol, which contributes to fatty deposits in arteries and cardiovascular risk. The researchers say promising early findings alone would not justify widespread use: evidence is needed that benefits last and risks remain acceptably low over many years.
Long-term monitoring is therefore central to evaluating these treatments. Clinical trials are often too small and too short to detect very rare complications or problems that emerge years later. Smith and Murthy call for independent, standardized monitoring systems that can track both effectiveness and unexpected health effects at scale.
Cost is another obstacle. Some existing gene therapies have list prices exceeding $1 million per patient. The researchers say treatments intended for common conditions would need to be affordable and accessible, so their use does not deepen healthcare inequalities.
They also raise the possibility that some genetic interventions may eventually include biological “off-switches” or other ways to reverse their effects. Until such safeguards are shown to work reliably, the ability to adjust or stop conventional medication remains an important advantage.
Sources: ScienceDaily.