Intravitreal autologous bone marrow cd34+ cell therapy for ischemic and degenerative retinal disorders: Preliminary phase 1 clinical trial findings

Research output: Contribution to journalArticlepeer-review

97 Scopus citations


METHODS. 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.

RESULTS. 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.

CONCLUSIONS. Intravitreal autologous BM CD34+ cell therapy appears feasible and well tolerated in eyes with ischemic or degenerative retinal conditions and merits further exploration. ( number, NCT01736059.).

PURPOSE. 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.

Original languageEnglish (US)
Pages (from-to)81-89
Number of pages9
JournalInvestigative Ophthalmology and Visual Science
Issue number1
StatePublished - Nov 14 2014


  • OCT
  • Retinal degeneration
  • Retinal imaging
  • Retinal vein occlusion
  • Stem cells

ASJC Scopus subject areas

  • Ophthalmology
  • Sensory Systems
  • Cellular and Molecular Neuroscience


Dive into the research topics of 'Intravitreal autologous bone marrow cd34+ cell therapy for ischemic and degenerative retinal disorders: Preliminary phase 1 clinical trial findings'. Together they form a unique fingerprint.

Cite this