Treatment of macular degeneration using embryonic stem cell-derived retinal pigment epithelium: preliminary results in Asian patients.
Embryonic stem cells hold great promise for various diseases because of their unlimited capacity for self-renewal and ability to differentiate into any cell type in the body. However, despite over 3 decades of research, there have been no reports on the safety and potential efficacy of pluripotent stem cell progeny in Asian patients with any disease. Here, we report the safety and tolerability of subretinal transplantation of human embryonic-stem-cell (hESC)-derived retinal pigment epithelium in four Asian patients: two with dry age-related macular degeneration and two with Stargardt macular dystrophy. They were followed for 1 year. There was no evidence of adverse proliferation, tumorigenicity, ectopic tissue formation, or other serious safety issues related to the transplanted cells. Visual acuity improved 9-19 letters in three patients and remained stable (+1 letter) in one patient. The results confirmed that hESC-derived cells could serve as a potentially safe new source for regenerative medicine.
Department of Ophthalmology, CHA Bundang Medical Center, CHA University, Seongnam-si, Gyeonggi-do 463-712, Republic of Korea. Electronic address: songwkmd@daum.net.
Won Kyung
WK
Song
Development Division, CHA Biotech Co., Ltd., Seoul 135-907, Republic of Korea.
Kyung-Mi
KM
Park
Development Division, CHA Biotech Co., Ltd., Seoul 135-907, Republic of Korea.
Hyun-Ju
HJ
Kim
CHA Stem Cell Institute, CHA Biotech Co., Ltd., Seoul 135-907, Republic of Korea.
Jae Ho
JH
Lee
Division of Rheumatology, Department of Internal Medicine, CHA Bundang Medical Center, CHA University, Seongnam-si, Gyeonggi-do 463-712, Republic of Korea.
Jinjung
J
Choi
Division of Hematology-Oncology, Department of Internal Medicine, CHA Bundang Medical Center, CHA University, Seongnam-si, Gyeonggi-do 463-712, Republic of Korea.
So Young
SY
Chong
Department of Biomedical Science, CHA University, Seoul 135-081, Republic of Korea.
Sung Han
SH
Shim
Albert Florens Storm Eye Institute, Medical University of South Carolina, Charleston, SC 29425, USA.
Lucian V
LV
Del Priore
Ocata Therapeutics, Marlborough, MA 01752, USA. Electronic address: rlanza@ocata.com.
Robert
R
Lanza
D000368:Aged; D044466:Asian People; D002454:Cell Differentiation; D004596:Electroretinography; D053595:Embryonic Stem Cells / Q000166:cytology*; D005260:Female; D006801:Humans; D008268:Macular Degeneration / Q000151:congenital / Q000628:therapy*; D008297:Male; D008875:Middle Aged; D055213:Retinal Pigment Epithelium / Q000166:cytology / Q000378:metabolism / Q000637:transplantation*; D000080362:Stargardt Disease; D014792:Visual Acuity