Intravitreal Autologous Bone Marrow CD34+ Cell Therapy for Ischemic and Degenerative Retinal Disorders: Preliminary Phase 1 Clinical Trial Findings

• DOI: 10.1167/iovs.14-15415
• Publication Date: 2014-12-10T05:11:17Z
• Authors: S. S. Park; G. Bauer; M. Abedi; S. Pontow; A. Panorgias; R. Jonnal; R. J. Zawadzki; J. S. Werner; J. Nolta
• Keywords: Microperimetry; Stem Cell Therapy; Cell therapy

Purpose.: Because human bone marrow (BM) CD34+ stem cells home into damaged tissue and may play an important role in repair, this pilot clinical trial explored the safety feasibility of intravitreal autologous BM as potential therapy for ischemic or degenerative retinal conditions. Methods.: This prospective study enrolled six subjects (six eyes) with irreversible vision loss from vascular occlusion, hereditary nonexudative age-related macular degeneration, retinitis pigmentosa. were isolated under Good Manufacturing Practice conditions mononuclear cellular fraction aspirate using a CliniMACs magnetic cell sorter. After injection, serial ophthalmic examinations, microperimetry/perimetry, fluorescein angiography, electroretinography (ERG), optical coherence tomography (OCT), adaptive optics OCT performed during 6-month follow-up. Results.: A mean 3.4 million (range, 1–7 million) injected per eye. The was well tolerated no intraocular inflammation hyperproliferation. Best-corrected visual acuity full-field ERG showed worsening after 6 months. Clinical examination also follow-up except among degeneration whom mild progression geographic atrophy noted both eye contralateral at follow-up, concurrent some possible decline on multifocal microperimetry. Cellular vivo imaging changes suggestive new incorporation macula Conclusions.: Intravitreal appears feasible eyes merits further exploration. (ClinicalTrials.gov number, NCT01736059.)