A bioethicist says scientists owe clinical trial volunteers support

Some people see clinical trials as a chance for a miracle cure. In reality, these experimental drug tests and medical interventions often fail. With researchers in the United States now testing the gene editor CRISPR/Cas9 for the first time in people with cancer, blood disorders or inherited blindness (SN: 8/14/19), one bioethicist says it’s important to remind scientists running these trials and others about the responsibilities researchers bear for study volunteers.

That’s not to say that scientists doing clinical trials are doing anything wrong or that such studies should stop, says Laurie Zoloth of the University of Chicago. Her role as a bioethicist, she explains, is “to make sure that human progress goes forward in a way that’s safe and ethical.”

Clinical trials in people — whether testing new drugs, devices, surgical methods or CRISPR technology — must meet higher ethical standards than work conducted in a lab, she says. “Having a human being as a subject means you have different obligations than you would to an animal or a petri dish,” Zoloth says. A scientist conducting clinical trials on humans should be “responsible for them, in my opinion, forever.”

That means that researchers should pay not just for the experimental treatment, she says, but also for any treatment needed for treating side effects — including those that show up later — or if something goes wrong during the trial. Many people’s insurance policies do not cover experimental procedures, yet some informed consent forms required for participating in clinical trials contain clauses claiming that a subject’s insurance will cover side effects, she notes. When Zoloth and others who review research applications point this out to scientists, “sometimes they’re surprised that they haven’t thought of it,” she says. But “sometimes they hope that, by putting that [clause] in there, it will release them from some burden.”

Zoloth, a professor of religion, understands the tendency to view clinical trials as godsends. Many people who sign up are struggling with untreatable conditions or poor prognoses. But sometimes experimental drugs and procedures just don’t work. For instance, stem cells for spinal cord injuries didn’t function as well in people as they did in rats, Zoloth says. “That’s very, very frustrating for scientists and certainly for patients,” she says.

And that’s exactly why researchers need to do these tests in people, whom Zoloth calls research subjects instead of patients. Research subjects play a crucial role in making sure treatments that show promise in animal tests are safe and effective in humans, but these tests should be recognized as experiments.

“It’s very brave and very noble of people with these diseases to take on the additional risk of being a subject,” Zoloth says. “Without this act of nobility, clinical research would collapse.”

Especially since in most cases, she says, “the intervention will not help them,” nor will subjects benefit financially from participating. “If you don’t benefit at all, at least you shouldn’t be harmed at all. You shouldn’t alone have to bear the burden of a severe adverse event.”

In general, researchers in the United States do follow guidelines, she says. A trial conducted by researchers at the University of Pennsylvania that tested the safety of CRISPR-edited immune cells against certain types of cancer was a good example of a careful study. It “didn’t make elaborate claims,” but made clear that it was an experimental safety trial, Zoloth says. Early results from that study showed that the gene-edited cells were safe, but didn’t stop the three volunteers’ cancer (SN:12/16/19).

Zoloth intends to continue reminding scientists of their responsibilities and of the risks subjects face. That “sobers the scientists,” she says. “It makes them understand the gravitas of their interactions with a human subject.”