Gene Editing Could Replace Lifelong Medication, Experts Warn of Risks
Researchers are exploring one-time genetic treatments for common conditions such as heart disease and diabetes, but experts say permanent changes raise serious questions.
Key points
- Gene therapies may work after a single dose, unlike daily medicines
- Experts Eric D. Smith and Venkatesh L. Murthy outline five questions before use
- Long-term monitoring is needed because rare side effects may appear later
- Some gene therapies cost over $1 million per patient
- Early studies are testing lasting reductions in LDL cholesterol
Gene therapy is already used to treat certain serious illnesses, and researchers are now asking whether similar approaches could help prevent far more common health problems. According to ScienceDaily, a perspective published in JAMA Internal Medicine argues that scientists must weigh the benefits of these treatments against their risks before they are offered widely.
The article was written by two cardiologists at the University of Michigan Health Frankel Cardiovascular Center, Eric D. Smith and Venkatesh L. Murthy. They say the central question is not only whether such treatments can change a person’s genetic activity, but whether they should.
Why one-time treatments are different
Traditional medicines usually work only while a patient keeps taking them. Genetic treatments act on genetic material itself. They may supply a healthy version of a gene, suppress a harmful gene, or modify a specific section of DNA.
That design makes single-dose treatments possible. However, a single dose does not guarantee lasting effects. Many treatments of this type rely on altered viruses as couriers that carry instructions into cells, and the immune system may respond to those carriers. This could make a repeat treatment difficult or impossible.
Current gene therapies generally target body cells rather than reproductive cells, so the changes are not intended to be passed on to future generations.
Concerns about common diseases
For rare and severe genetic conditions, the authors suggest the benefits may clearly outweigh the dangers, especially when few other options exist. The picture becomes more complicated for problems like raised cholesterol, cardiac illness, excess body weight and diabetes, which are already managed with proven medicines.
Many of those medicines can be adjusted or stopped if a patient’s condition changes or side effects appear. A permanent intervention may not offer the same flexibility. Murthy also noted that a side effect that is rare could still affect many people if a treatment is given to millions.
Five questions and the monitoring challenge
Smith and Murthy propose five factors to guide decisions. They ask how serious the disease is, whether effective treatments already exist, whether the target tissue suits a lasting one-dose approach, whether delivery risks are reasonable, and whether researchers can monitor safety over many years.
The final point is particularly difficult. Clinical trials usually involve small groups followed for limited periods, so very rare complications may only appear later. The authors say monitoring systems will need to be independent, standardised and able to operate on a much larger scale.
Cost and early research
According to the article, certain current gene treatments carry sticker prices exceeding $1 million per person, and a number of others are far pricier. Such figures may be defensible for rare, life-threatening conditions, but the authors warn that similar pricing for widespread diseases would place heavy strain on health systems.
Researchers are already running early studies to test whether a single treatment can lower LDL, often called bad cholesterol, for long periods. Lower LDL over time is an important way to reduce cardiovascular risk. Even so, the authors say promising early results will not be enough. Evidence of lasting benefit, low long-term risk and manageable cost will be needed before these treatments become routine.
Frequently asked questions
Can gene therapy replace daily medication?
Some gene therapies are designed to work after a single dose, unlike conventional medicines that must be taken repeatedly. The source notes, however, that a single dose does not necessarily mean the effects last a lifetime.
Why are experts cautious about gene editing for common diseases?
Existing treatments for conditions like heart disease and diabetes can be adjusted or stopped, while permanent gene changes may not offer that flexibility. Rare side effects could also affect many people if treatments are used widely.
How much do gene therapies cost?
According to the source, certain current gene treatments carry sticker prices above $1 million per person, and a number of others are priced far higher.
Sources
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