TY - JOUR
T1 - Retinal stem cell transplantation
T2 - Balancing safety and potential
AU - Singh, Mandeep S.
AU - Park, Susanna S.
AU - Albini, Thomas A.
AU - Canto-Soler, M. Valeria
AU - Klassen, Henry
AU - MacLaren, Robert E.
AU - Takahashi, Masayo
AU - Nagiel, Aaron
AU - Schwartz, Steven D.
AU - Bharti, Kapil
N1 - Funding Information:
•SDS: Grant support from Astellas , Nikon , Nidek and Verily
Funding Information:
The RIKEN study ( Mandai et al., 2017b ) entailed the use of a cellular sheet without an extrinsic scaffold; however, an alternative approach is to specifically address the degenerative changes in native Bruch membrane in AMD patients by including a synthetic Bruch membrane replacement as part of a composite implant. Kashani and colleagues at the University of Southern California Roski Eye Institute recently reported the interim results of a Phase 1/II clinical trial ( clinicaltrials.gov identifier NCT02590692) evaluating a composite implant of stem cell derived RPE and such a synthetic substrate in the worse-seeing eye of subjects with advanced dAMD ( Kashani et al., 2018 ). The study was sponsored by Regenerative Patch Technologies.
Funding Information:
•TAA: Research to Prevent Blindness , National Institutes of Health (Center Core grant, P30EY014801 and P30EY001319 ), the Department of Defense ( W81XWH-09-1-0675 ), and the Klorfine Foundation (to Dr. Albini)
Funding Information:
?MSS: Research to Prevent Blindness (to the Wilmer Eye Institute), The Shulsky Foundation, The Hartwell Foundation, Foundation Fighting Blindness?SSP: National Institute of Health (UG1EY026876-01A1)?TAA: Research to Prevent Blindness, National Institutes of Health (Center Core grant, P30EY014801 and P30EY001319), the Department of Defense (W81XWH-09-1-0675), and the Klorfine Foundation (to Dr. Albini)?MVCS: National Institute of Health (R01EY022631 to Dr. Canto-Soler), BrightFocus Foundation (to Dr. Canto-Soler), The Gates Family Foundation (to Dr. Canto-Soler), The Doni Solich Family Foundation (to Dr. Canto-Soler), Research to Prevent Blindness (to the Department of Ophthalmology at the University of Colorado)?HK: California Institute for Regenerative Medicine (to Dr. Klassen via jCyte), Polly and Michael Smith Foundation (to Dr. Klassen), Geneva Matlock, MD (to Dr. Klassen), Research to Prevent Blindness (to Department of Ophthalmology)?REM: The Medical Research Council, NIHR Senior Investigator Award, Oxstem Ocular and the Royal College of Surgeons of Edinburgh?MT: Center for Biosystems Dynamics Research, RIKEN?AN: The Las Madrinas Endowment in Experimental Therapeutics for Ophthalmology and an unrestricted grant to the Department of Ophthalmology at the USC Keck School of Medicine from Research to Prevent Blindness?SDS: Research to Prevent Blindness, the Price Foundation, UCLA Broad Stem Cell Research Center, and the Stein Eye Institute Clinical Research Center, UCLA Department of Ophthalmology?KB: National Eye Institute Intramural Research Program and Common Funds Therapeutic Challenge Award?MSS: Patents on retinal cell delivery technology (Johns Hopkins University)?REM: Grant funding from Oxstem Ocular Ltd and Astellas Inc.?SDS: Grant support from Astellas, Nikon, Nidek and Verily
Funding Information:
•REM: Grant funding from Oxstem Ocular Ltd and Astellas Inc .
Publisher Copyright:
© 2019 The Authors
PY - 2020/3
Y1 - 2020/3
N2 - Stem cell transplantation holds great promise as a potential treatment for currently incurable retinal degenerative diseases that cause poor vision and blindness. Recently, safety data have emerged from several Phase I/II clinical trials of retinal stem cell transplantation. These clinical trials, usually run in partnership with academic institutions, are based on sound preclinical studies and are focused on patient safety. However, reports of serious adverse events arising from cell therapy in other poorly regulated centers have now emerged in the lay and scientific press. While progress in stem cell research for blindness has been greeted with great enthusiasm by patients, scientists, doctors and industry alike, these adverse events have raised concerns about the safety of retinal stem cell transplantation and whether patients are truly protected from undue harm. The aim of this review is to summarize and appraise the safety of human retinal stem cell transplantation in the context of its potential to be developed into an effective treatment for retinal degenerative diseases.
AB - Stem cell transplantation holds great promise as a potential treatment for currently incurable retinal degenerative diseases that cause poor vision and blindness. Recently, safety data have emerged from several Phase I/II clinical trials of retinal stem cell transplantation. These clinical trials, usually run in partnership with academic institutions, are based on sound preclinical studies and are focused on patient safety. However, reports of serious adverse events arising from cell therapy in other poorly regulated centers have now emerged in the lay and scientific press. While progress in stem cell research for blindness has been greeted with great enthusiasm by patients, scientists, doctors and industry alike, these adverse events have raised concerns about the safety of retinal stem cell transplantation and whether patients are truly protected from undue harm. The aim of this review is to summarize and appraise the safety of human retinal stem cell transplantation in the context of its potential to be developed into an effective treatment for retinal degenerative diseases.
UR - http://www.scopus.com/inward/record.url?scp=85072045185&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85072045185&partnerID=8YFLogxK
U2 - 10.1016/j.preteyeres.2019.100779
DO - 10.1016/j.preteyeres.2019.100779
M3 - Review article
C2 - 31494256
AN - SCOPUS:85072045185
VL - 75
JO - Progress in Retinal and Eye Research
JF - Progress in Retinal and Eye Research
SN - 1350-9462
M1 - 100779
ER -