Stem cell research is ethical balancing act
Balancing the therapeutic needs of very sick patients with the demands of rigorous scientific research is a major ethical challenge in stem cell research, according to Mary Devereaux, PhD, director of the biomedical ethics seminars and assistant director of the Research Ethics Program at the University of California, San Diego (UCSD).
“As we develop greater expertise in stem cell research and begin moving into early clinical trials, patients and families naturally want access to experimental therapies for degenerative diseases, such as ALS [amyotrophic lateral sclerosis],” she says.
Patient groups have generated a lot of discussion about ways to move the research forward more quickly, including giving patients a larger role in determining what risks they are willing to take. Duane Roth, of The California Institute for Regenerative Medicine, argues that patients and patient advocates should, through carefully selected representatives, have “a seat at the regulatory approval table.”1
“How you would involve patient representatives in the [Food and Drug Administration] process, or decide which patient groups would get a seat at the table, raises complicated issues,” Devereaux says. “Certainly in California, patients and their families do have a key role to play in the stem cell research initiative.” At UCSD, the local community is updated with an annual presentation of stem cell research by area investigators. “A recent national conference on ALS held in La Jolla included patient advocacy groups, as well as a presentation by a patient and his family,” adds Devereaux.
Proceed with caution
On the other hand, researchers and clinicians involved in stem cell trials have voiced concerns about the need to proceed with enough caution, including adequately informing and protecting human subjects. “There isn’t a whole lot that many of these patients can do, in terms of standard medicine, for some of these diseases. The pressure to do something experimental is very, very strong,” says Devereaux. “If you are cutting corners on the science, oftentimes you lose time anyway, because others can’t replicate it. The only ethical or responsible path is to do the absolute best science that you can.”
A related issue is the challenge of creating effective informed consent procedures in highly experimental areas like stem cells. “One of the things we talk about continually is that stem cell biology is a new and complex field,” says Devereaux. “You want people who are deciding to enroll in a clinical trial to have a really clear picture of what they are taking on.”
If an intervention is offered in the earliest stage, in a Phase I trial, it’s extremely unlikely that an individual patient will gain personal benefit, even if the patient is in the active wing of the trial, for example. “One of the things we always talk about as ethicists is the therapeutic misconception; that is, the false belief that the primary aim of a clinical trial is the benefit of the individual subject rather than generalized knowledge,” says Devereaux. Whatever patients are told about the risks of experimentation, what they tend to hear is that this is one thing that they can do that offers some hope, she explains.
“Our challenge consists in explaining these complicated scientific protocols so that patients know enough about what could happen that they can give thoughtful consideration to the risks and benefits for them,” says Devereaux.
The possibility of hope makes it difficult for most people to stop and think about what a clinical trial means in their own particular case. “One thing I tell trainees and students all the time is that they should not assume that because somebody has a degenerative disease or is dying of metastatic cancer, that they can’t make them worse,” says Devereaux. “If somebody has three months to live and we make them sicker, or we turn three months into two months, that is harm — a real loss for the patient.”
Patients often also don’t understand the time required to translate research into something that can actually help individual patients. “Regenerative medicine offers enormous possibilities, but it’s not going to be realized in the next three years,” says Devereaux. “The normal scientific trajectory from ‘bench to bedside,’ to get things to market that are safe and effective, is a pretty slow process. It’s tragically too slow if you’ve got a neurodegenerative disease and need a treatment today.”
At one point in time, stem cell research had to fight against opponents simply in order to continue. “Back in the Bush era, when some people wanted to make this whole field just go away, the emphasis then was either ‘yea’ or ‘nay,’” Devereaux says. “There was, at that time, a lot of hype — on the part of scientists and funders — and hope, on the part of patients and patient advocates.”
In California, there was a citizen mandate to go forward with stem cell research, reflecting acceptance and financial commitment from the general public. “Once we decided we were going to go down this road in California, then other states followed,” she says. “Now we have a different administration, and polls show that a majority in the U.S. do support stem cell research, embryonic as well as non-embryonic strategies.”
More public education is needed on how clinical trials are run, and more generally on the process of scientific discovery. “Only then will patients and providers have a better understanding of what we can realistically expect of stem cell research,” argues Devereaux. “The field is moving very fast, but we’ve got a lot to do in terms of science education. We’re talking about a very steep learning curve here.”
1. Roth D. A third seat at the table: An insider’s perspective on patient representatives. Hastings Center Report 2011;41(1):29-31.
• Mary Devereaux, PhD, Director, Biomedical Ethics Seminars/Assistant Director, Research Ethics Program, University of California, San Diego. Phone: (858) 822-5764. E-mail: firstname.lastname@example.org.