Gene therapies and transplantation for sickle cell disease are increasingly moving from the bench to the bedside. In fact, while U.S. approval for the first gene therapy may come before the close of 2023, the first gene therapy for sickle cell disease has already been approved in Europe. Despite this rapid therapeutic advancement, many social and ethical questions have been left under discussed, and inquiries into the diverse perspectives about the meaning of cure by individuals living with sickle cell disease have been largely missing. This presentation draws on research from a multi-year study of individuals living with sickle cell disease who have various experiences with sickle cell disease therapeutics, including gene therapy and other curative therapies. This paper unpacks how people living with sickle cell disease understand the concept of cure for this monogenic hemoglobinopathy, especially those who require ongoing care after pursuing curative therapies. This paper finds that while biomedical endpoints are important elements in understanding the meaning of cure, social elements, which are often left undiscussed in biomedical spaces, are crucial to how individuals living with sickle cell disease understand the concept of cure. By unpacking how lived experiences drive meaning-making, expectations, and perceptions of cure and advanced therapies, this paper moves beyond the biomedical, and highlights how care and cure must be necessarily intertwined in discourse about a “cure for sickle cell disease”. These findings have direct social and ethical implications for the research, development, approval, and commercialization of emerging gene therapies for sickle cell disease.
Author: Marilyn Baffoe-Bonnie, National Institutes of Health
