First Human iPSC Trial Started

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The RIKEN Center for Developmental Biology in Kobe, Japan, has had a tough year. Lauded at the start of the year for their discovery of STAP cells, the Institute later became the subject of controversy after uncertainty surrounding the papers resulted in their retraction and the suicide of one of the paper’s authors.

But now, hopeful news of a scientific move forward is coming from RIKEN. The Institute just started the first human trial using induced pluripotent stem cells, led by ophthalmologist Dr. Masayo Takahashi.

The media is quickly picking up this story, which involves using iPSCs for the treatment of age-related macular degeneration. Using iPSCs avoids the potential pitfalls associated with using embrokyonic stem cells in humans, owing to their improved immunoresponse and the absence of ethical issues.

Back in January, in an interview with New Scientist, Dr. Masayo Takahashi expressed confident optimism about the trials, but also explained that one of the disadvantages of her treatment – the extremely high cost – is because of the cells being derived from the same patient, as opposed to being allogeneic.

After the final safety green light, the first trial was performed on September 12th.

The patient was a Japanese woman in her 70s. According to RIKEN, a 1.3 x 3 mm sheet of retinal pigment epitelium cells was engrafted into the subretinal space of her eye.

The preliminary nature of this study implies an inherent uncertainty about its outcome, as well as potential risks associated with the treatment, which the scientists involved in the study are fully aware of, as was the patient.

Despite Takahashi’s initial optimism, there is no certainty that the study will reveal a successful therapeutic outcome. Nonetheless, this is a tremendous first step that the scientific community has welcomed with cautious, but hopeful, optimism.



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