World first for AMD stem cell therapy
A patient in Japan has become the first person to be treated with a stem cell treatment for age–related macular degeneration (AMD).
In a world first, researchers at the RIKEN Centre for Developmental Biology (CDB), in Kobe, Japan, used induced pluripotent stem (iPS) cells to grow a thin sheet of retinal pigment epithelial cells, which have been implanted directly into the patient’s retina to replace diseased tissue. The iPS cells were generated using skin cells taken from the patient’s arm, which were reprogrammed to revert to an embryonic-like state.
A team of three ophthalmologists from the Institute for Biomedical Research and Innovation in Kobe carried out the procedure on Friday (September 12), which reportedly lasted for two hours. The patient is reported to be a woman in her 70s from the Hyogo prefecture of Japan.
The latest development comes just days after the treatment was cleared by a Japanese health ministry committee. Led by Masayo Takahashi, a stem cell researcher and opthlamologist at the CDB, the research team demonstrated the safety and efficacy of the treatment in mice and monkeys, which allowed the team to proceed with the first trials in humans. Earlier this week, Nature News reported that the trial at the RIKEN “will be the first opportunity for the technology to prove its clinical value.”
According to RIKEN, the study will eventually include six participants, with the first three staggered by eight week intervals. The remaining three patients will receive the treatment after a safety evaluation of the first group of subjects. All patients will be monitored for one year after the procedure.
At a press conference held at RIKEN on Friday, Dr Takahashi commented: “I am very happy that the operation went smoothly. We have managed to take the first step in regenerative medicine with iPS cells.”
Commenting on the latest development, Robin Ali, Professor of Human Molecular Genetics and head of the Department of Genetics at the UCL Institute of Ophthalmology, told OT the trial is “an exciting development for both patients and for the scientific community.” Professor Ali explained that as the tissue is derived from the patient’s own cells, the risk of rejection after transplant might be reduced.
He added: “It will be a number of years until we know whether retinal pigment epithelium differentiated from iPS-cells will prove to be well tolerated and have benefits for vision. However, this is an extremely encouraging next step and could in the future help preserve or even improve vision for people with AMD.”
Ade Deane-Pratt, research communications officer for charity Fight for Sight, said: “This is a remarkable human trial and we will be watching the outcome with great interest. The success rate of previous studies to replace retinal pigment epithelium cells has been poor as it is difficult to get the cells to take hold and begin to function without the potential for cancerous growths or rejection by the immune system. However the field of regenerative medicine offers great hope.”