RT info:eu-repo/semantics/article
T1 A proof-of-concept clinical trial using mesenchymal stem cells for the treatment of corneal epithelial stem cell deficiency
A1 Calonge, Margarita
A1 Pérez Soto, María Inmaculada
A1 Galindo de la Rosa, Sara
A1 Nieto Miguel, Teresa
A1 López Paniagua, Marina
A1 Fernández Martínez, Itziar
A1 Alberca Zaballos, Mercedes
A1 García-Sancho Martín, Francisco Javier
A1 Sánchez García, Ana María de los Ángeles
A1 Herreras Cantalapiedra, José María
K1 Clinical trial
K1 Ensayo clínico
K1 Corneal blindness
K1 Ceguera corneal
K1 In vivo confocal microscopy
K1 Microscopía confocal in vivo
K1 Mesenchymal stem cells
K1 Células madre mesenquimales
K1 Stem cell transplantation
K1 Células madre, Trasplante de
AB Ocular stem cell transplantation derived from either autologous or allogeneic donor corneoscleral junction is a functional cell therapy to manage extensive and/or severe limbal stem cell deficiencies that lead to corneal epithelial failure. Mesenchymal stem cells have been properly tested in animal models of this ophthalmic pathology, but never in human eyes despite their potential advantages. We conducted a 6- to 12-month proof-of-concept, randomized, and double-masked pilot trial to test whether allogeneic bone marrow-derived mesenchymal stem cell transplantation (MSCT], n = 17) was as safe and as equally efficient as allogeneic cultivated limbal epithelial transplantation (CLET), (n = 11) to improve corneal epithelial damage due to limbal stem cell deficiency. Primary endpoints demanded combination of symptoms, signs, and the objective improvement of the epithelial phenotype in central cornea by in vivo confocal microscopy. This proof-of-concept trial showed that MSCT was as safe and efficacious as CLET. Global success at 6–12 months was 72.7%–77.8% for CLET cases and 76.5%–85.7% for MSCT cases (not significant differences). Central corneal epithelial phenotype improved in 71.4% and 66.7% of MSCT and CLET cases, respectively at 12 months (P = 1.000). There were no adverse events related to cell products. This trial suggests first evidence that MSCT facilitated improvement of a diseased corneal epithelium due to lack of its stem cells as efficiently as CLET. Consequently, not only CLET but also MSCT deserves more preclinical investigational resources before the favorable results of this proof-of-concept trial could be transformed into the larger numbers of the multicenter trials that would provide stronger evidence. (ClinicalTrials.gov number, NCT01562002.)
PB Elsevier
SN 1931-5244
YR 2019
FD 2019
LK http://uvadoc.uva.es/handle/10324/40509
UL http://uvadoc.uva.es/handle/10324/40509
LA eng
NO Translational Research, 2019, vol. 206. p. 18-40
NO Producción Científica
DS UVaDOC
RD 20-sep-2024