Authors:
Dr. Eline Bunnik | Erasmus MC | Netherlands
Sietske van Till | Erasmus MC | Netherlands
Introduction: Induced pluripotent stem cell (IPSC) technology can be used to develop patient-derived two-dimensional neuronal cell models for drug screening and treatment selection. This technology is especially promising for patients with monogenic neurodevelopmental disorders, given the severity and heterogeneity of these disorders, the hitherto very low therapeutic success, and limited access to brain tissue of affected children. However, the clinical use of IPSC technology poses ethical questions, e.g. in relation to patient selection, informed consent, assessment of benefits and risks, governance of samples, commercialisation of cell-based models, and equitable access. It cuts across various disciplines, each associated with its own set of ethical issues: genetics, (neuronal) stem cell technology, the inclusion of children (with cognitive impairments) in research, and the blurring boundaries between research and care in n-of-1 clinical trials and personalized medicine.
Methods: I will review three sets of ethical literature: that on personalised medicine and n-of-1 clinical trials, the use of (neuronal) IPSC, and the inclusion of children with neurodevelopmental disorders in research. I will carve out a place for personalised medicine using neuronal IPSC models for children with monogenic neurodevelopmental disorders across these literatures, and determine the framework (research, care or otherwise) within which it should be evaluated. Then, I will collate and synthesize current ethics guidance for this application of IPSC technology.
Outcome: Drawing on several sets of ethical literature, I will set out some conditions for responsible clinical implementation of IPSC technology in personalised medicine for children with monogenic neurodevelopmental disorders.