From discovery to approval of an advanced therapy medicinal product-containing stem cells, in the EU.
In 1997, the human corneal epithelium was reconstructed in vitro and transplanted on patients. Later, it became a routine treatment, before regulations considered advanced therapy medicinal products and drugs on the same lines. Manufacturing, before and after good manufacturing practice setting, was established in different facilities and the clinical application in several hospitals. Advanced therapy medicinal products, including stem cells, are unique products with different challenges than other drugs: some uncertainties, in addition to benefit, cannot be avoided. This review will focus on all recent developments in the stem cell-based corneal therapy.
Centre for Regenerative Medicine "Stefano Ferrari", University of Modena and Reggio Emilia, via G.Gottardi 100, Modena, 41125, Italy; Holostem Terapie Avanzate, Modena, Italy.
Graziella
G
Pellegrini
Department of Sense Organs, University of Rome "Sapienza", viale Regina Elena, Rome, Italy.
Alessandro
A
Lambiase
Unit of Ophthalmology, University of Parma, Via Gramsci 14, 43126 Parma, Italy; IMEM - CNR (Italian National Reserach Council), Parco Area delle Scienze 37/A - 43124 Parma, Italy.
Claudio
C
Macaluso
Ophthalmology Unit-Eye Bank, S. Giovanni Addolorata Hospital, via S. Stefano Rotondo 9, Rome, Italy.
Augusto
A
Pocobelli
Cornea Division Stein Eye Institute, UCLA 100 Stein Plaza Los Angeles, CA 90095, USA.
Sophie
S
Deng
Ophthalmology Unit, Policlinico University Hospital, University of Modena & Reggio Emilia, via Del Pozzo 71, Modena, 41125, Italy.
Gian Maria
GM
Cavallini
Centre for Regenerative Medicine "Stefano Ferrari", University of Modena and Reggio Emilia, via G.Gottardi 100, Modena, 41125, Italy; Holostem Terapie Avanzate, Modena, Italy.
Roza
R
Esteki
Cornea & Ocular Surface Unit San Raffaele Scientific Institute Via Olgettina, 60-20132 Milano, Italy.
Paolo
P
Rama
D000818:Animals; D003316:Corneal Diseases / Q000628:therapy*; D019573:Epithelium, Corneal / Q000166:cytology*; D005062:European Union; D006801:Humans; D010946:Plants, Medicinal; D033581:Stem Cell Transplantation; D035703:Therapies, Investigational; D023822:Tissue Engineering / Q000379:methods*
ATMP commercialization; adult corneal stem cells; artificial organs; cell therapy; culture/differentiation of stem cells (niche); ophthalmology; regenerative medicine; tissue engineering; translational studies; transplantation