First iPSC Transplant Patient Doing Well

The first patient to receive an iPS cell transplant has shown no tumor formation since her surgery in 2014.

AsianScientist (Oct. 16, 2015) – On October 2, the RIKEN Center for Developmental Biology (CDB), together with the Foundation for Biomedical Research and Innovation (FBRI) and Kobe City Medical Center General Hospital, gave an update on the first patient to undergo a transplant of iPS cells for age-related macular degeneration (AMD).

The transplantation, which was performed on September 12, 2014, was part of the first study in the world using iPS cells in the clinic, aimed at testing the safety of the therapy in patients. The progress made by the patient was described as good in a report released by the Kobe City Medical Center General Hospital, RIKEN CDB and FBRI.

Dr. Yasuo Kurimoto of FBRI, an ophthalmologist and the head of the surgical team, reported that after one year of follow-up the patient has not experienced any major side effects, that there have not been any signs of tumor formation, and that the transplanted cells remain viable.

The patient’s vision, which was deteriorating before the procedure, has remained stable since the operation. However, Dr. Kurimoto emphasized that this might be due to the removal of neovascular tissue rather than the effect of the transplanted retinal pigment epithelium (RPE).

The team has done examinations including tests of visual acuity, intraocular pressure, ocular fundus and imaging periodically on the patient during and post hospitalization. The overall results of examinations from the monitoring period are good, with the RPE sheet remaining viable and grafted in the initial transplanted site without the need for immunosuppression.

No signs of tumorigenesis or other major abnormalities were observed as a result of the transplantation. Comprehensive cancer screening (whole-body) conducted one year post-transplantation did not detect signs of tumor formation or abnormalities. Using this treatment protocol (removal of neovascular tissue followed by transplantation of an iPSC-derived RPE cell sheet into affected site), there were no signs of recurring neovascularization and the morphology of the macular showed signs of improvement.

Masayo Takahashi of RIKEN CDB said, “We are very happy at the outcome so far as they are as we hoped. I am eager to continue moving forward until we can make this treatment available to many AMD patients.”


Source: RIKEN.
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