Stem Cell Therapy For Eye Disease To Be Tested In Japan

A pilot clinical study to assess the use of stem cell therapy in wet-type age-related macular degeneration has been launched in Japan.

Asian Scientist (Aug. 6, 2013) – Japanese researchers have launched a pilot clinical study to assess the feasibility and safety of using induced pluripotent stem cell (iPSC) therapy in age-related macular degeneration (AMD), the most common cause of blindness and visual impairment in people over 50 years old.

In the pilot study, which started recruiting patients on 1 August 2013, the researchers from RIKEN will treat patients with wet-type AMD by transplanting autologous iPSC-derived retinal pigment epithelium (RPE) cell sheets into the eye.

Wet-type AMD is characterized by progressive damage to the RPE, a protective layer of cells located at the back of the eye. Current drug treatments for this disease focus on preventing further loss of the protective layer, but are unable to repair damage that have already occurred.

Replacement of the RPE cells by transplantation is a potential means of repairing the damage in the patient’s eye. However, the challenge lies in obtaining RPE cells that are not rejected by the patient’s immune system and can be harvested without using invasive procedures.

In this study, the researchers will establish autologous iPSCs from each of the research participants. The iPSCs will then be differentiated into RPE cells using technology that allows these cells to be transplanted in monolayer cell sheets without using synthetic scaffolds or matrices.

The RPE cell sheets will then be shaped into 1.3 x 3 mm grafts to be transplanted into the affected site of a single eye after the damaged RPE is removed.

Transplant sites will be monitored closely for functional integration and potential adverse reactions for an initial intensive observation period of one year. The patients will subsequently be put on follow-up observation for three years.


Source: RIKEN.
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