I just learned some promising news from the stem cell research front. RIKEN, an innovative research group in Japan, is hoping to launch a clinical trial of an age-related macular degeneration (AMD) treatment derived from induced pluripotent stem cells (iPSC). Based on my current knowledge, this would be the first-ever iPSC-based treatment for the retina to move into a human study.
As Dr. David Gamm reported recently on this blog, iPSC therapy is a treatment approach in which the donor serves as his or her own source of stem cells. Researchers take cells from the patient’s skin or blood and genetically tweak them to an embryonic-like state. Next, the cells are coaxed forward to become retinal cells, which can be transplanted into the donor’s eyes. Depending on the type of retinal degeneration, researchers may also use gene therapy to correct the underlying genetic defect in the transplanted cells.
One of the key benefits of iPSC is that, like embryonic stem cells, they can become any type of cell in the body, and be easily replicated to make large quantities of cells for therapeutic purposes. However, because the donor is the source of the transplanted cells, there is less chance of rejection.
Though I don’t know all the details of the study, the RIKEN treatment sounds similar, in some respects, to the treatment developed by Advanced Cell Technology (ACT) now in clinical studies for dry AMD and Stargardt disease. While each group’s approach involves transplanting retinal pigment epithelial (RPE) cells into patients’ retinas, RIKEN will derive its treatment from iPSC. ACT, on the other hand, develops RPE cells from embryonic stem cells.
Though we currently have more research experience with embryonic stem cells, iPSC offers the important advantages I just mentioned. And there’s nothing like a clinical trial to see how well they might really work.
The Foundation funds both iPSC and embryonic stem cell research because we don’t know which option will be best. It may be the case that both alternatives will have their own unique advantages in different diseases or stages of degeneration.
Pictured above: Rod cells derived from iPSC at the RIKEN labs.