Favorable Results in an Early-Stage Clinical Trial of a Stem-Cell Therapy Funded by FFB

January 15, 2015
Jan. 15, 2015 – Six months after the launch of a clinical trial for a bone marrow stem-cell treatment targeting retinal disorders, researchers are reporting encouraging preliminary findings. 
The first six patients enrolled in the on-going clinical trial, taking place at the University of California Davis Eye Center, tolerated the procedure well without any serious adverse events.  Vision in most of the treated eyes also improved modestly after treatment. 
Results of the trial, funded in part by the Foundation Fighting Blindness (FFB), were reported in the journal Investigative Ophthalmology & Visual Science. The scientists plan to conduct a larger human study of the stem-cell treatment to gain a better understanding of its potential for saving and restoring vision. 
“The primary goal of early human studies like the stem-cell trial at UC Davis is to ensure that the treatment is safe and doesn’t diminish vision,” says Stephen Rose, Ph.D. chief research officer, Foundation Fighting Blindness. “With favorable results in the first few patients, the trial can be expanded to get a better sense of the treatment’s true effect on vision and long-term safety.”
The treatment involved an ocular injection of bone marrow stem cells obtained from the patient’s hip. Doctors used a needle to extract the cells from each participant’s hip bone in an outpatient procedure requiring only a local anesthetic. An average of 3.4 million cells were then injected into each patient’s vitreous, the gel-like substance in the middle of the eye. Because the patients were treated with their own stem cells, no immunosuppression was needed to prevent rejection of the therapy.
In previous studies in people with cardiac conditions, this treatment was found to have the potential to repair cardiac tissue damaged by the blockage of blood vessels. Research has shown that the stem cells migrate naturally to the site of damage and play an important role in repair. These findings inspired Susanna Park, M.D., Ph.D., at UC Davis to study the potential use of these stem cells as treatment for blinding retinal conditions.  
The UC Davis clinical-trial participants included two women with dry age-related macular degeneration (AMD), two men with Stargardt disease, one man with retinitis pigmentosa and one man with central retinal artery and vein occlusion. All participants had advanced, irreversible vision loss from the retinal condition at the time of treatment.   
Though vision improved in most of the treated eyes, Dr. Park cautions that the improvements could be attributed to placebo effects or increasing familiarity with the vision exams. She also noted that mild progression of retinal degeneration was observed in the treated eyes of patients with dry AMD after six months despite the stem-cell treatment.  
“Our findings definitely warrant further investigation of stem cell therapy,” Dr. Park says. “So, we are planning a larger study with longer follow-up to further explore the safety and potential efficacy of the treatment.”