TY - JOUR
T1 - Stem cell-derived retinal pigment epithelium transplantation in age-related macular degeneration
T2 - Recent advances and challenges
AU - Kashani, Amir H.
N1 - Funding Information:
This work was supported by the Wilmer Eye Institute and the Johns Hopkins University through the T Boone Pickens Endowed Professorship in Ophthalmology.
Funding Information:
Dr. Kashani's former employer, the University of Southern California, received grant funding from Regenerative Patch Technologies to support Dr. Kashani's role as principal investigator in a clinical trial utilizing stem cell derived RPE in the treatment of NNAMD.
Publisher Copyright:
© 2022 Lippincott Williams and Wilkins. All rights reserved.
PY - 2022/5/1
Y1 - 2022/5/1
N2 - Purpose of reviewAge-related macular degeneration (AMD) is one of the leading causes of irreversible vision loss in the world with more than 80% of the prevalence accounted for by the nonneovascular (NNAMD) or 'dry' form of the disease. NNAMD does not have any definitive treatment once vision loss has ensued and presents a major unmet medical need. This review will highlight stem cell-based therapies that are a promising form of treatment for advanced NNAMD.Recent findingsIn the past decade, clinical trials utilizing both induced pluripotent stem cell-derived RPE and human embryonic stem cell-derived RPE have been aggressively pursued as potential treatments of RPE loss and prevention of overlying neurosensory atrophy. While promising preliminary results demonstrating safety and potential efficacy have been published, new challenges have also been identified. These include selecting the most appropriate cell-based therapy, identifying and managing potential immune response as well as characterizing anatomic and functional efficacy. In this review, we will discuss some of these challenges in light of the available data from several early phase clinical trials and discuss the strategies that are being considered to further advance the field.SummaryCell-based therapies demonstrate promising potential to treat advanced stages of NNAMD. Several early phase clinical trials using both induced pluripotent stem cells (iPSC) and human embryonic stem cell derived (hESC) have demonstrated safety and preliminary signs of efficacy and highlighted remaining challenges which appear surmountable. These challenges include development of selection criteria for use of cell suspensions versus RPE sheets, especially in light of immunological properties of RPE that are intrinsic to the status of RPE differentiation in each of these cell formulations.
AB - Purpose of reviewAge-related macular degeneration (AMD) is one of the leading causes of irreversible vision loss in the world with more than 80% of the prevalence accounted for by the nonneovascular (NNAMD) or 'dry' form of the disease. NNAMD does not have any definitive treatment once vision loss has ensued and presents a major unmet medical need. This review will highlight stem cell-based therapies that are a promising form of treatment for advanced NNAMD.Recent findingsIn the past decade, clinical trials utilizing both induced pluripotent stem cell-derived RPE and human embryonic stem cell-derived RPE have been aggressively pursued as potential treatments of RPE loss and prevention of overlying neurosensory atrophy. While promising preliminary results demonstrating safety and potential efficacy have been published, new challenges have also been identified. These include selecting the most appropriate cell-based therapy, identifying and managing potential immune response as well as characterizing anatomic and functional efficacy. In this review, we will discuss some of these challenges in light of the available data from several early phase clinical trials and discuss the strategies that are being considered to further advance the field.SummaryCell-based therapies demonstrate promising potential to treat advanced stages of NNAMD. Several early phase clinical trials using both induced pluripotent stem cells (iPSC) and human embryonic stem cell derived (hESC) have demonstrated safety and preliminary signs of efficacy and highlighted remaining challenges which appear surmountable. These challenges include development of selection criteria for use of cell suspensions versus RPE sheets, especially in light of immunological properties of RPE that are intrinsic to the status of RPE differentiation in each of these cell formulations.
KW - age-related macular degeneration
KW - immune response
KW - retinal pigment epithelium
KW - stem cell
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U2 - 10.1097/ICU.0000000000000838
DO - 10.1097/ICU.0000000000000838
M3 - Review article
C2 - 35200164
AN - SCOPUS:85131107349
SN - 1040-8738
VL - 33
SP - 211
EP - 218
JO - Current opinion in ophthalmology
JF - Current opinion in ophthalmology
IS - 3
ER -