Carefully Curing Chronic Illnesses
The key medical task facing humans in this century will be curing chronic illnesses.
Over the course of the 20th century, through a combination of sanitation and immunization, humankind greatly reduced the dangers of infectious disease. That means people are living much longer now and facing much different challenges. Instead of the threat coming from bacteria and viruses outside the body, it’s often coming from DNA inside the body.
To defeat these disorders, humans are working to develop gene therapy. The National Institutes of Health explains that, “Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.”
But the key words to note there are “experimental” and “may allow.” Gene therapy is very much in the trial stages, and there are still more risks than rewards to the treatment.
Gene therapy, the NIH says, can be carried out in several ways:
- Replacing a mutated gene that causes disease with a healthy copy of the gene.
- Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
- Introducing a new gene into the body to help fight a disease.
Any one of those approaches is very difficult, and at the edge of available science. Consider, for example, CRISPR. It seems to be a great invention. In theory, it allows scientists to splice a little DNA out here, insert a little DNA there, and cure a host of ailments. Time magazine makes it sound easy.
But the truth is more complex. When you splice DNA into a human, you change the entire genetic structure. Remember the twins in China who were genetically edited using CRISPR? The intention was to make the girls immune to the AIDS virus.
MIT Technology Review says the treatment may have done more than that. “New research shows that the same alteration introduced into the girls’ DNA, deletion of a gene called CCR5, not only makes mice smarter but also improves human brain recovery after stroke, and could be linked to greater success in school.”
And while that all sounds positive, remember that nobody knew that was a possibility when the gene splicer was used. There could very well be negative side effects as well, and they’ll only be discovered in the future. The truth is that nobody knows. A single scientist has changed the human genome. This isn’t the way that science is supposed to work.
Gene therapy may be promising, but it must be tested rigorously in the lab before it’s deployed in humans. We cannot afford the risks of tests carried out “in the wild.” And in the lab, it’s often causing problems.
Earlier this year, a gene therapy experiment at the University of Pennsylvania went badly awry. The experiment used three rhesus monkeys and three pigs and aimed to test whether a particular gene therapy would be effective against muscular disease. In less than a week, four of the six animals were dead, and the two monkeys that survived suffered toxic reactions.
The point here isn’t to condemn gene therapy. This is actually how science is supposed to work: it’s being tested on animals before humans. The point is to show that gene therapy is very experimental, and potentially dangerous.
Before any therapies are approved for use in humans, they should have to pass rigorous tests and prove they’re safe. Americans need to know that the FDA is on the job and up to the task.
There’s so much we don’t know about the human genome. But what we do know is that it’s the guidebook that contains instructions for how to build humans. It may be technically possible to erase pages in that book and write different chapters. But doing so could change our planet’s future. By all means, let’s cure illnesses such as cancer, diabetes, and muscular dystrophy. But let’s not kill ourselves while trying.
Beau Rothschild, the founder of Rothschild Policy and Politics, formerly served as the members outreach director for the Committee on House Administration.
