First in-human study of human umbilical tissue-derivedcaeels as a therapy for retinal degenerative diseases shows promise.
In the first in-human study of human umbilical tissue-derived cells as cell-based therapy for retinal degenerative diseases, all seven patients tolerated the subretinal injection well and had no postoperative visual loss.
"One out of seven isn't very much, but if you're looking for any possible signal, that might be it," said Dr Francis, whose lab was the lead institution in the study. "Otherwise, patients didn't lose vision.
All of the participants had advanced retinitis pigmentosa (RP), no better than hand-motion vision. They were enrolled from three institutions in the US and underwent vitrectomy and single unilateral extramacular subretinal injection of an allogenic cellular product known as CNTO2476.
The cells were produced by the Stem Cell Organization, which is part of the J&J Biotechnology Center of Excellence, Centocor R&D Inc., which is now planning to move forward with studies of additional indications and additional trials.
Dr Francis reported the latest findings at the 2010 annual meeting of the Association for Research in Vision and Ophthalmology. In this preliminary study, the three men and four women, with a median age of 62 years, received cell dosages ranging from 47500 to 470000 cells delivered transvitreally to the subretinal space through a retinotomy. Follow-up evaluations were conducted for more than 1 year.
Analysis of the results showed no evidence of immune rejection. Two patients developed postsurgical retinal detachments, which may have been related to non-closure of the retinotomy site.
Although the participants in a phase I study are typically normal, healthy individuals, the nature of this therapy, which required a vitrectomy, necessitated choosing subjects with a retinal degenerative condition, Dr Francis said.
"We thought that the chances of seeing efficacy were very limited, but because this was the first in-human use of cells in this manner, and certainly in the eye, we thought that they would be the target group," he continued. "If we were going to see efficacy, it would be in one of these retinal degenerations such as RP and possibly age-related macular degeneration (AMD)."
Although it was encouraging that there was no evidence of an immune rejection, the broader implication of the phase I study for the field of stem cell research was that these cells could be successfully implanted in the retina without a deleterious effect or immune response, Dr Francis said.
"This was the first and tentative, careful step into transplantation and use of stem cells for retinal disease, and the results were encouraging," he added. "It was a small and carefully planned step forward for the field."