A fistula is defined as an abnormal communication between two epithelium-lined surfaces,
and fistula-in-ano consists of an abnormal tract connecting the anal canal or rectum
with the perianal skin or vagina [
[1]
]. When developing in Crohn disease, fistulas—considered an independent phenotype and
referred to as perianal disease [
[2]
]—represent a grievous and disabling condition, resulting in significant human and
health care costs because of a high relapse rate despite the use of biologics combined
with surgery as standard therapy [
[3]
]. The advent of novel therapeutic strategies based on minimally invasive surgery and
mesenchymal stem/stromal cells (MSCs) offers an unprecedented opportunity. Following
the results of a phase 3 trial in which 212 patients were randomly assigned to receive
a single local injection of 120 millions of allogeneic adipose tissue-derived MSCs
(darvadstrocel, formerly Cx601) or placebo, which showed that MSCs performed better
to achieve combined remission (51.5% versus 35.6% at week 24) within a shorter time
[
[4]
], the European Medicines Agency granted marketing authorization (Alofisel; Takeda,
Tokyo, Japan) for use in refractory cases [
[5]
]. Since that time, several issues have been raised that hamper its use and jeopardize
the knowledge regarding its therapeutic value. This prompted the authors to highlight
in this editorial the misconceptions and hurdles that, based on the authors’ own experience,
hinder an appropriate application of this new treatment and to establish a list of
recommendations whose implementation may help this therapy find its right positioning
in the management of this dismal condition.EMA/1380/2018 EMEA/H/C/004258. https://www.ema.europa.eu/en/documents/overview/alofisel-epar-summary-public_en.pdf.
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Article info
Publication history
Published online: December 20, 2022
Accepted:
November 26,
2022
Received:
April 17,
2022
Publication stage
In Press Corrected ProofIdentification
Copyright
© 2022 International Society for Cell & Gene Therapy. Published by Elsevier Inc. All rights reserved.
