November 4, 2014

A new study published in the journal The Lancet is promising for the use of stem cells in regenerative therapies. Sponsored by Advanced Cell Technology, the research showed long-term safety and success with the use of Retinal Pigment Epithelium cells in the eyes of 18 U.S. patients – half with dry age-related macular degeneration and half with Stargardt’s macular degeneration.

The study followed patients for six months following the transplant and shows the first “evidence of the mid- to long-term safety, survival, and potential biologic activity of pluripotent stem cell progeny into humans with any disease,” according to ACT. It’s also the first U.S. Food and Drug Administration-approved embryonic stem cell trial that has had success, NPR noted.

Reducing fears

In the past, stem cell therapies have raised fears about tumors or inappropriate cells developing, such as bone cells in a soft tissue area, The New York Times explained. But, ACT was able to change the stem cells into RPE cells, which can sense light and work for retinal repair, before using them on the patients. There were no adverse growths or issues observed.

In addition to being safe, this study found improvement in the vision of 10 out of the 18 patients after six months.

“Diseases affecting the eye are attractive first-in-man applications for this type of investigational therapy due to the immune-privileged nature of the eye. Despite the degenerative nature of these diseases, the vision of 10 of 18 patients showed measurable improvement at the six month follow up, after transplantation of the RPE cells,” Robert Lanza, M.D., Chief Scientific Officer of ACT explained.

Both AMD and SMD have no other treatments that are effective at this time. AMD is the leading cause of eye sight loss for millions of older adults, while SMD affects far fewer and mainly children or teenagers. SMD and AMD are major causes of blindness around the world, particularly in developing countries.

A ‘very important’ study

Surgeon and director of the Wake Forest Institute for Regenerative Medicine at Wake Forest University, Anthony Atala called this paper “very important,” telling NPR’s All Things Considered that this technology may have implications for various other ailments such as heart or lung disease.

This study not only was a success with promising implications, but as The New York Times explained, ACT pointed to a technique that can use embryonic stem cells without destroying the embryo. This could reduce a significant amount of criticism related to embryonic stem cell use. The newspaper noted that one embryo was destroyed for stem cells in this study, however.