Table 1 Overview of key ethical challenges of CGT (cellular and gene therapy) early-phase clinical trials [13,14,15,16,17]
From: A few ethical issues in translational research for gene and cell therapy
Overview of key ethical challenges of CGT early-phase clinical trials |
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Key ethical challenges of early-phase clinical trials |
Difficulty in evaluating preclinical research |
Difficulty in assessing the risk-to-benefit ratio |
Conceptualisation and estimation of patients benefits and/or social benefits |
Criteria for inclusion/exclusion of participants |
Process of information and consent |
Risk of therapeutic misconception |
Some distinctive features of CGT products that have an influence on the design of early-phase clinical trials |
Potential for prolonged biological activity after a single administration |
High potential for immunogenicity |
Need for relatively invasive procedures to administer the product |
Preclinical data not informative as for small molecule pharmaceuticals |
Unique complexities of CT products due to the dynamic nature of living cells (stem cells may undergo transformation and begin forming tumours) |
In GT products, expression of a delivered gene may be uncontrolled and may interfere with normal function of a critical enzyme, hormone or biological process in the recipient |
Genomic alteration in the recipient could cause activation or inactivation of neighbouring genes and generate benign or malignant tumours |