Stem Cell Research & Therapy
14, Article number: 261 (2023)



Intravitreal allogeneic mesenchymal stem cells: a non-randomized phase II clinical trial  for acute non-arteritic optic neuropathy


Jose C. Pastor1,2,3,4, Salvador Pastor‑Idoate1,3,4* , Marina López‑Paniagua1,2,3,5, Marta Para1, Francisco Blazquez1,3, Esther Murgui1, Verónica García3,6,7 and Rosa M. Coco‑Martín1,2,


Background An efective treatment for acute non-arteritic ischemic optic neuropathy (NA-AION) has not been
known or proven yet. Previous studies have suggested a neuroprotective efect of allogeneic bone marrow-derived
mesenchymal stem cells. This study aims to report the results of a clinical trial on patients with acute non-arteritic
optic neuropathy (NA-AION) treated with an intravitreal injection of allogeneic bone marrow-derived mesenchymal
stem cells (BM-MSCs) (MSV®).

Methods We conducted a prospective, non-randomized, clinical phase-II study (Eudra CT number 2016-00302940; Registry NCT03173638) that included 5 patients with acute unilateral NA-AION diagnosed
within 2 weeks after symptom onset and who received an intravitreal injection of allogeneic BM-MSCs (0.05 ml; cell
concentration: 1.5× 106
cells/mL). The patients underwent regular ophthalmological examinations and were followed
for one year.

Results In this trial, allogeneic BM-MSCs appeared to be safe as no patients developed signs of acute nor chronic
intraocular infammation or a signifcant change in intraocular pressure, although an epiretinal membrane
was developed in one patient. A retrolental aggregate formed shortly after the injection spontaneously disappeared
within a few weeks in another phakic patient, leaving a subcapsular cataract. Visual improvement was noted in 4
patients, and amplitudes of P100 on the visually evoked potentials recordings increased in three patients. The retinal
nerve fber layer and macular ganglion cell layer thicknesses signifcantly decreased during the follow-up.
Conclusions Besides the development of an epiretinal membrane in one patient, the intravitreal application of allo‑
geneic BM-MSCs appeared to be intraocularly well tolerated. Consequently, not only NA-AION but also BM-MSCs
deserve more clinical investigational resources and a larger randomized multicenter trial that would provide stronger
evidence both about safety and the potential therapeutic efcacy of intravitreally injected allogeneic BM-MSCs
in acute NA-AION.

Trial registration: Safety Assessment of Intravitreal Mesenchymal Stem Cells for Acute Non-Arteritic Anterior Ischemic
Optic Neuropathy (NEUROSTEM). NCT03173638. Registered June 02, 2017


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