World’s First Experimental Therapy Involving Reprogrammed Donor Stem Cells Aims to Treat Macular Degeneration

April 4th, 2017 by FFB Canada

Stem cells are frequently in the news. At the FFB, we love reading about stem cell discoveries, but high-frequency reporting can make it challenging to identify truly significant breakthroughs, such as the one from last week: “Japanese man is the first to receive ‘reprogrammed’ stem cells from another person.” The story, published in a prestigious scientific journal, Nature, describes the first stem cell transplantation that used induced pluripotent stem cells (iPSCs) from another donor as an experimental treatment for a blinding eye disease.

This is a genuine breakthrough because it opens the doors to accessible stem cell therapies—not just for blindness, but for a variety of diseases. Indeed, it is a critical step forward for the entire field of regenerative medicine. It is particularly meaningful to all of us at the FFB because this pioneering trial is focused on treating the most common blinding eye disease affecting people over the age of 55: age-related macular degeneration (AMD).

The trial involves utilizing iPSCs, which are created by taking mature cells from an adult, then reprogramming them to become stem cells. The stem cells can then be coaxed into becoming a variety of different cell types, including eye cells. The approach provides an appealing alternative to developing treatments that rely on embryonic stem cells (ESCs).

The most recent procedure is part of a larger initiative led by Dr. Masayo Takahashi from the RIKEN research institute in Japan. Earlier this year we reported on Dr. Takahashi’s success transplanting retinal tissue to improve vision in blind animals. In 2014, we wrote about RIKEN’s first pioneering iPSC trial, which also focused on AMD, but relied on the patient’s own cells rather than donor-derived cells. RIKEN recently shared preliminary results from this first trial in The New England Journal of Medicine, disclosing that the patient’s vision has not declined since the surgery.

If the most recent transplant is successful, it will represent a big step forward for iPSCs as a viable approach to combatting vision loss. Whereas a patient’s own cells run the risk of carrying genetic defects, iPSCs from donors can be tested for genetic health and stockpiled according to type.

Though the recent surgery went well, its ultimate success rests on how it impacts the development of the patient’s AMD. Ideally, the vision loss that leads to blindness in many of those with AMD will be halted as a result of the introduction of these new cells. If the patient’s vision loss slows or ceases, we will know that the reprogrammed cells were effective.

RIKEN’s long-term goal is to develop an iPSC cell bank that is genetically diverse enough to treat a significant percentage of Japan’s population. Such a bank would have both social and scientific value: since the cells are taken from living donors, the bank would sidestep many of the ethical debates surrounding embryonic stem cells.

Here at the FFB we are closely monitoring RIKEN’s work: the scientific developments occurring at RIKEN and elsewhere fuel the fight against blindness. These findings also provide new insights for FFB-funded stem cell researchers, such as Dr. Gilbert Bernier and Dr. Michel Cayouette, who are both working on projects that aim to develop stem cell therapies for blindness. In the Race to Restore Sight, we still don’t know who is going to win, which is why we are so thankful for all of the FFB donors who ensure that essential stem cell research continues to progress along many fronts. We are excited by the promising clinical results emerging from this trailblazing research.

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Sources

“Japanese man is the first to receive ‘reprogrammed’ stem cells from another person” in Nature on March 28, 2017.

“Autologous Induced Stem-Cell–Derived Retinal Cells for Macular Degeneration” in The New England Journal of Medicine on March 16, 2017. DOI: 10.1056/NEJMoa1608368.

More reading about stem cells

The FFB’s guide to stem cell therapy