Ethical challenges for a new generation of early-phase pediatric gene therapy trials

Alexander A Iyer; Dimah Saade; Diana Bharucha-Goebel; A Reghan Foley; Gilberto 'Mike' Averion; Eduardo Paredes; Steven Gray; Carsten G Bönnemann; Christine Grady; Saskia Hendriks; Annette Rid

doi:10.1038/s41436-021-01245-3

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Ethical challenges for a new generation of early-phase pediatric gene therapy trials

Journal article

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Ethical challenges for a new generation of early-phase pediatric gene therapy trials

Alexander A Iyer, Dimah Saade, Diana Bharucha-Goebel, A Reghan Foley, Gilberto 'Mike' Averion, Eduardo Paredes, Steven Gray, Carsten G Bönnemann, Christine Grady, Saskia Hendriks, …

Genetics in medicine, Vol.23(11), pp.2057-2066

11/2021

DOI: 10.1038/s41436-021-01245-3

PMID: 34234300

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Abstract

After decades of setbacks, gene therapy (GT) is experiencing major breakthroughs. Five GTs have received US regulatory approval since 2017, and over 900 others are currently in development. Many of these GTs target rare pediatric diseases that are severely life-limiting, given a lack of effective treatments. As these GTs enter early-phase clinical trials, specific ethical challenges remain unresolved in three domains: evaluating risks and potential benefits, selecting participants fairly, and engaging with patient communities. Drawing on our experience as clinical investigators, basic scientists, and bioethicists involved in a first-in-human GT trial for an ultrarare pediatric disease, we analyze these ethical challenges and offer points to consider for future GT trials.

Child

Clinical Trials as Topic - ethics

Genetic Therapy - ethics

Humans

Treatment Outcome

Details

Title: Subtitle: Ethical challenges for a new generation of early-phase pediatric gene therapy trials
Creators: Alexander A Iyer - National Institutes of Health Clinical Center
Dimah Saade - National Institute of Neurological Disorders and Stroke
Diana Bharucha-Goebel - National Institute of Neurological Disorders and Stroke
A Reghan Foley - National Institute of Neurological Disorders and Stroke
Gilberto 'Mike' Averion - National Institute of Neurological Disorders and Stroke
Eduardo Paredes - National Institute of Neurological Disorders and Stroke
Steven Gray - The University of Texas Southwestern Medical Center
Carsten G Bönnemann - National Institute of Neurological Disorders and Stroke
Christine Grady - National Institutes of Health Clinical Center
Saskia Hendriks - National Institutes of Health Clinical Center
Annette Rid - National Institutes of Health Clinical Center
Resource Type: Journal article
Publication Details: Genetics in medicine, Vol.23(11), pp.2057-2066
DOI: 10.1038/s41436-021-01245-3
PMID: 34234300
NLM abbreviation: Genet Med
ISSN: 1098-3600
eISSN: 1530-0366
Language: English
Date published: 11/2021
Academic Unit: Stead Family Department of Pediatrics; Iowa Neuroscience Institute; Neurology (Pediatrics)
Record Identifier: 9984354151802771

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