Experimental Stem Cell-Based Therapy in Pediatrics: A Fictional Case Study

Abstract

This chapter analyzes the complex process of decision making when applying highly experimental stem cell-based therapy to children. We focus particularly on the special case of life-threatening conditions and with no available alternative treatment, when the experimental therapy is completely untested, or only tried on a few adults. The reader finds himself/herself in the shoes of a surgeon who is torn by a dilemma: Does he have morally justifiable reasons to treat a 4-year old child with an experimental therapy, and if no, why? The reader considers, together with the surgeon, the issues that have to be raised in the process of decision making. When are experimental stem cell-based therapies in pediatrics justified? What rules governing pharmaceutical research on children should be applied in the case of stem cell-based therapies? How to weigh the risks and benefits in the case of this particular child when the stakes are high, and a balance has to be found between life and unknown risks of unknown certitude and magnitude. What if the known risks and burdens of treatment may almost amount to torture in the perception of a 4-year-old patient? The child is not capable of making an informed choice on whether to accept this treatment or not. Which values should be given priority in such a case? The surgeon searches for the answer in different ethical theories, and considers arguments drawn from consequentialist, human rights, dignitarian and other deontological theories. The surgeon also questions whether free and informed consent in the context of experimental stem cell-based therapies is possible, when the seriousness of the child's condition may influence the parental decision making regarding the experimental treatment of the child, and when the risk of therapeutic misconception is high.