Ethical Challenges in Early-Phase Pediatric Research for Life-Limiting Illness

Research with children is necessary to assure more effective treatments and potential cures of childhood illnesses. Ethical conduct of research requires minimizing the inherent risks of research, especially when it involves vulnerable populations such as children. Duchenne muscular dystrophy (DMD) is a progressive and fatal disease with no Food and Drug Administration–approved treatment. Clinical trials investigating so-called gene therapies are viewed by many in the DMD community with great promise. The goal of research is to secure generalizable knowledge and not directly benefit patients, yet many parents of boys with DMD hope, and even expect, that their sons will derive medical benefit by participating in early-phase “gene therapy” trials, raising concern for the therapeutic misconception. Physician-investigators must assist patient-subjects to distinguish realistic from unrealistic hope while maintaining reasonable expectations. In this article, I examine the therapeutic misconception and related concepts as framed within the context of “gene therapy” for DMD.