Intravitreal autologous bone marrow CD34+ cell therapy for ischemic and degenerative retinal disorders: preliminary phase 1 clinical trial findings

Because human bone marrow (BM) CD34+ stem cells home into damaged tissue and may play an important role in tissue repair, this pilot clinical trial explored the safety and feasibility of intravitreal autologous CD34+ BM cells as potential therapy for ischemic or degenerative retinal conditions. This...

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Bibliographic Details
Published in:Investigative ophthalmology & visual science 2014-12, Vol.56 (1), p.81-89
Main Authors: Park, Susanna S, Bauer, Gerhard, Abedi, Mehrdad, Pontow, Suzanne, Panorgias, Athanasios, Jonnal, Ravi, Zawadzki, Robert J, Werner, John S, Nolta, Jan
Format: Article
Language:English
Subjects:
Adult
Aged
Aged, 80 and over
Antigens, CD34 - immunology
Bone Marrow Cells - cytology
Bone Marrow Cells - immunology
Cell Transplantation - methods
Clinical Trials
Female
Fluorescein Angiography
Follow-Up Studies
Fundus Oculi
Humans
Intravitreal Injections
Ischemia - diagnosis
Ischemia - immunology
Ischemia - therapy
Male
Prospective Studies
Retinal Degeneration - diagnosis
Retinal Degeneration - immunology
Retinal Degeneration - therapy
Retinal Diseases - diagnosis
Retinal Diseases - immunology
Retinal Diseases - therapy
Treatment Outcome
Visual Acuity
Young Adult
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Summary:Because human bone marrow (BM) CD34+ stem cells home into damaged tissue and may play an important role in tissue repair, this pilot clinical trial explored the safety and feasibility of intravitreal autologous CD34+ BM cells as potential therapy for ischemic or degenerative retinal conditions. This prospective study enrolled six subjects (six eyes) with irreversible vision loss from retinal vascular occlusion, hereditary or nonexudative age-related macular degeneration, or retinitis pigmentosa. CD34+ cells were isolated under Good Manufacturing Practice conditions from the mononuclear cellular fraction of the BM aspirate using a CliniMACs magnetic cell sorter. After intravitreal CD34+ cell injection, serial ophthalmic examinations, microperimetry/perimetry, fluorescein angiography, electroretinography (ERG), optical coherence tomography (OCT), and adaptive optics OCT were performed during the 6-month follow-up. A mean of 3.4 million (range, 1-7 million) CD34+ cells were isolated and injected per eye. The therapy was well tolerated with no intraocular inflammation or hyperproliferation. Best-corrected visual acuity and full-field ERG showed no worsening after 6 months. Clinical examination also showed no worsening during follow-up except among age-related macular degeneration subjects in whom mild progression of geographic atrophy was noted in both the study eye and contralateral eye at 6-month follow-up, concurrent with some possible decline on multifocal ERG and microperimetry. Cellular in vivo imaging using adaptive optics OCT showed changes suggestive of new cellular incorporation into the macula of the hereditary macular degeneration study eye. Intravitreal autologous BM CD34+ cell therapy appears feasible and well tolerated in eyes with ischemic or degenerative retinal conditions and merits further exploration. (ClinicalTrials.gov number, NCT01736059.).
ISSN:0146-0404
1552-5783
DOI:10.1167/iovs.14-15415