Carnegie Mellon University

Gene Therapy

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Ethics of Early-Stage Research

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Clinical trials on early-stage gene therapy are recruiting patients from the developing world. These trials provide medically deprived populations access to interventions that show potential promise but also may pose a great risk — their effects in humans are largely unknown.

The technologies being tested are most directly relevant to the health concerns of the wealthier countries who sponsor the research rather than those of the poorer nations whose citizens are serving as research subjects. And that has bioethicists at Carnegie Mellon and McGill universities raising a red flag.

According to commentary published in The Lancet, the practice may be inconsistent with international ethics guidelines on justice.

"There are many reasons why researchers might look to the developing world for research subjects," said Alex John London, lead author of "Justice in Translation: From Bench to Bedside in the Developing World" and director of Carnegie Mellon's Center for the Advancement of Applied Ethics and Political Philosophy.

"But serious ethical issues can arise when research relies on the deprivations experienced by people living in developing countries to advance research that is not responsive to the urgent health needs of their communities," he said.

London and his co-author, Jonathan Kimmelman, an assistant professor in McGill's Biomedical Ethics Unit, urge organizations that sponsor research in low- and middle-income countries (LMICs) to ensure that they are addressing the most pressing health needs of those nations. The article also notes that any interventions developed as a result of such research should be affordable and easily implemented in those countries' health care systems.

Other authors have explored ethical issues in later stage clinical trials, in which the interventions have already been deemed safe and effective, for the most part. But London and Kimmelman are the first to discuss the more complicated considerations surrounding the riskier early-stage research.

"Our report centers on complex agents like gene therapies that are being tested for the very first time in human beings," Kimmelman said.

Researchers have various reasons for turning to developing nations for clinical trial subjects. In some cases, patients are recruited because diseases like malaria were much more common in LMICs. In other cases, diseases are so rare as to necessitate worldwide recruitment.

But some trials also appear to have recruited patients who did not have access to treatments routinely available in developed countries. Such patients provide a pool of "treatment-naive" subjects that would not otherwise be available to researchers.

Treatment-naive subjects are particularly valuable, as they offer the opportunity for researchers to observe an intervention's behavior on a "blank canvas" of sorts.

London's and Kimmelman's article echoes requirements that have been articulated in a range of international ethics documents by groups such as the World Health Organization. Ensuring that research meets this requirement represents an important step toward unlocking the substantial promise of innovative research like gene therapy for populations that often experience staggering health needs.

"Our goal is not to curtail research in low- and middle-income countries," said London, who is also an associate professor of philosophy at Carnegie Mellon. "It is to make sure that project sponsors give careful consideration to relationship between a particular research study and the needs of the communities from which study participants are drawn."

A complete copy of London's and Kimmelman's article is available by contacting Kelli McElhinny at 412-268-6094 or kellim@andrew.cmu.edu or Mark Shainblum at 514-398-2189 or mark.shainblum@mcgill.ca

Related Links: CAAEPP  |  Dept of Philosophy