Several New Stem Cell Clinical Trials Poised to Begin in …

One of the more exciting aspects of FFBs recent annual conference, VISIONS 2013, was the news on the clinical development of several emerging stem cell therapies. The field has picked up a lot of steam, with many new human studies of stem-cell treatments anticipated to begin in 2014 and 2015.

Whats particularly noteworthy is that some of these trials will be for people with retinitis pigmentosa (RP) a population not yet included in human stem-cell studies. Also, investigators will evaluate new types of cells and new, innovative approaches to implanting them.

Heres a review of three of the stem-cell clinical studies on the horizon:

Riken Institute: Sheets of RPE Derived from Induced Pluripotent Stem Cells

In June, the Japanese Health Ministry gave approval to the Riken Institute to launch a clinical trial of sheets of retinal pigment epithelial (RPE) cells for the treatment of wet age-related macular degeneration (AMD). The study is planned for 2014.

The sheets will replace native RPE cells damaged by leaky blood vessels, which are the hallmark of wet AMD. RPE cells play a critical role in supporting photoreceptors, the cells that make vision possible.

For the first time in a human study, the researchers will produce RPE cells from induced pluripotent stem cells (iPSC) stem cells derived by turning back the development clock on a small sample of skin taken from the patient. The stem cells are then coaxed forward to become RPE. Because the cells come from the patient, theres less chance of immune rejection.

David Gamm, M.D., Ph.D., of the University of Wisconsin-Madison, said that transplanting sheets of RPE cells rather than injecting a bolus or a clump of cells was likely to produce better results, because RPE cells naturally occur as a layer in the retina. He believes that sheets of cells have a better chance to integrate and function normally.

By the way, Dr. Gamm, in collaboration with Dennis Clegg, Ph.D., of the University of California, Santa Barbara, is receiving Foundation funding to develop a patch of cells comprised of photoreceptors and RPE cells for treating retinal diseases. While his work is still a few years away from the clinic, it is very promising, because so many retinal diseases affect both of these cell types.

UC Irvine: Retinal Progenitor Therapy for Reactivating the Retina

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Several New Stem Cell Clinical Trials Poised to Begin in ...