In Practice

Ethical considerations in the application of cell and gene therapies in children

M S Pepper, A Pope, S Kling, M Alessandrini, W van Staden, R J Green


Rapidly evolving fields such as cell and gene therapies that involve state-of-the-art technology hold out possibilities that may be ahead of what ethics, guidelines and the law have considered. This results in a regulatory lag. Furthermore, ethical and legal considerations are often debated in real time as issues pertaining to these technologies that were previously not considered begin to come to the fore. Finding the appropriate balance between facilitating potential therapeutic gains and ensuring the safety interests of recipients of the new treatments requires close attention, especially for minors. This vulnerable population frequently has off-label treatment prescribed on the basis of extrapolation of clinical trial data derived from adults, which is ethically and scientifically questionable. In this article we discuss how best to maintain ethical integrity while introducing innovative cell and gene therapies to minors. We advocate that clinical trials of promising innovative therapies should be designed so that testing in adults is followed as soon as possible by testing in minors, given the impressive gains that have recently been reported.

Authors' affiliations

M S Pepper, South African Medical Research Council Extramural Unit for Stem Cell Research and Therapy, Institute for Cellular and Molecular Medicine, Department of Immunology, School of Medicine, Faculty of Health Sciences, University of Pretoria, South Africa

A Pope, Emeritus Associate Professor, Department of Private Law, Faculty of Law, University of Cape Town, South Africa

S Kling, Department of Paediatrics and Child Health, Faculty of Medicine and Health Sciences, Stellenbosch University, Cape Town, South Africa

M Alessandrini, Department of Pathology and Immunology, Faculty of Medicine, University of Geneva, Switzerland

W van Staden, Centre for Ethics and Philosophy of Health Sciences, Faculty of Health Sciences, University of Pretoria, South Africa

R J Green, Department of Paediatrics and Child Health, School of Medicine, Faculty of Health Sciences, University of Pretoria and Steve Biko Academic Hospital, Pretoria, South Africa

Full Text

PDF (99KB)


Minors; Clinical trials; Innovation; Gene therapy; Immunotherapy; Cancer

Cite this article

South African Medical Journal 2018;108(12):1027-1029. DOI:10.7196/SAMJ.2018.v108i12.13585

Article History

Date submitted: 2018-11-26
Date published: 2018-11-26

Article Views

Abstract views: 1896
Full text views: 941

Comments on this article

*Read our policy for posting comments here