Hastings Center News
Individualized Genetic Therapies Raise Hopes and Ethical, Regulatory Questions
Until recently, medicine had little to offer most of the millions of people with rare genetic conditions. That changed in 2019, when the first genetic intervention was developed for a single patient, a 6-year-old child. Individualized genetic therapies give hope to patients and families, but also raise scientific, ethical, and regulatory questions, discussed in a recent article in BMJ coauthored by Hastings Center scholars and colleagues.
Genetic therapies customized for individual patients diverge from the traditional process of biomedical research in which experimental treatments are tested on research participants to determine if they are safe and effective—before they are approved for use on patients. In contrast, individualized genetic therapies are designed for just one patient. Can they serve as models for subsequent patients? How can we know?
The ultimate question is, do these individualized interventions fall into the category of research or treatment? “When discussing these interventions with patients,” the article asks, “should clinicians wear their researcher ‘hat,’ emphasizing risks and working to address any therapeutic misconception on the part of the research subject or should they work as clinicians pursuing innovation with patients who they genuinely believe might benefit from the interventions?”
The authors argue that at least some of these novel therapies should be seen as research-treatment hybrids, a category that has repercussions for funding, ethics oversight, and regulation.
The authors are Josephine Johnston, a senior research scholar at The Hastings Center and an associate professor at the University of Otago in New Zealand; Kathryn Tabb, an assistant professor of philosophy at Bard College; Danielle Pacia, a research associate at The Hastings Center; Sandra Soo-Jin Lee, chief of the Division of Medical Ethics at Columbia University and a Hastings Center fellow; Wendy K. Chung, chief of pediatrics at Boston Children’s Hospital; and Paul S. Appelbaum, the Elizabeth K. Dollard Professor of Psychiatry, Medicine and Law, and director of the Division of Law, Ethics and Psychiatry at Columbia University and a Hastings Center fellow.