TY - JOUR
T1 - Ethical challenges for a new generation of early-phase pediatric gene therapy trials
AU - Iyer, Alexander A.
AU - Saade, Dimah
AU - Bharucha-Goebel, Diana
AU - Foley, A. Reghan
AU - Averion, Gilberto ‘Mike’
AU - Paredes, Eduardo
AU - Gray, Steven
AU - Bönnemann, Carsten G.
AU - Grady, Christine
AU - Hendriks, Saskia
AU - Rid, Annette
N1 - Publisher Copyright:
© 2021, This is a U.S. government work and not under copyright protection in the U.S.; foreign copyright protection may apply.
PY - 2021/11
Y1 - 2021/11
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=85110024677&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85110024677&partnerID=8YFLogxK
U2 - 10.1038/s41436-021-01245-3
DO - 10.1038/s41436-021-01245-3
M3 - Review article
C2 - 34234300
AN - SCOPUS:85110024677
SN - 1098-3600
VL - 23
SP - 2057
EP - 2066
JO - Genetics in Medicine
JF - Genetics in Medicine
IS - 11
ER -