Stem Cell Treatments for Diabetic Retinopathy

Research by a team at the University of Virginia School of Medicine provided a crucial piece to treating patients who suffer vision loss because of diabetic retinopathy, a condition that affects millions of people with diabetes. The UVA team showed that the best for adult stem cells to treat this condition are cells taken from donors who do not suffer diabetes rather than cells taken from patients’ own bodies. This work could provide a critical step toward injecting stem cells into patients’ eyes to stop or even reverse vision loss. These findings could also establish a crucial framework for evaluating stem cells to be used in potential future treatments for diabetic retinopathy.

Diabetic Retinopathy

“It answers a vital question: If we’re going to carry this therapy forward into clinical trials, where are we going to get the best bang for the buck?” said UVA researcher and ophthalmologist Paul Yates, M.D., Ph.D. “The answer seems to be, probably, taking cells from patients who aren’t diabetic. Because the diabetic stem cells don’t seem to work quite as well. And that’s not terribly surprising, because we already know that this cell type is damaged by diabetes.”

The researchers hope to use stem cells derived from fat, since they are harvested during liposuction procedures. These fat-based stem cells might be able to stop or greatly delay the vascular degeneration that eventually leads to blindness in patients with diabetic retinopathy. What are the best cells for the job in this case? UVA’s new research provides those important answers. “We now know what to look for when we harvest a patient’s cells, because we know what distinguishes good quality cells from poor quality,” said researcher Shayn M. Peirce, Ph.D., of the UVA Department of Biomedical Engineering. “We almost have a screen to determine quality control. We’re essentially establishing quality-control criteria by understanding what works and why.”

Diabetic retinopathy patients desperately need new and more effective treatments. First of all, there are a growing number of people with this condition and secondly the present treatments only show limited effectiveness. More than 100 million people are estimated to suffer from diabetic retinopathy and related conditions; current treatments use lasers to fight back invading blood vessels, but these treatments often destroy much of the retina. Alternatively, patients are required to receive injections of anti-blood vessel forming drugs such as Lucentis (ranibizumab) or Eylea (aflibercept) directly into their eyeball, sometimes every month, for the rest of their lives.

“There’s huge room for improvement on the standard of care, and the number of patients in this demographic is increasing by the day, dramatically, so the need is only going up,” Peirce said. “So I think there are three pieces working together — UVA’s strengths in this area, the FDA’s encouragement [of stem cell research in the eye] and the clinical realities — to drive this cell-based therapy toward the clinic.”

While much more work needs to be done, if all goes well, the UVA team hopes to begin clinical trials in humans within the next few years. “This is not science fiction at all,” Yates said. “The idea that you can take cells from somewhere else and inject them into the eye to treat disease is here today.”