Israeli Company Advancing Clinical Trial for Stem-Cell Derived-Treatment for Dry AMD

July 08, 2016

Cell Cure Neurosciences, a regenerative-medicine company in Jerusalem, Israel, reports that its OpRegen® therapy for the dry form of age-related macular degeneration (AMD) has been safe thus far in a Phase I/IIa clinical trial at Hadassah Ein Kerem Hospital. Based on these encouraging results with the first group of patients, the investigators will be administering a higher dose of the therapy to a second cohort of patients. The Foundation Fighting Blindness provided funding for pre-clinical research that helped make the trial possible.

Cell Cure’s therapy is an injection of retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESC) underneath the patient’s retina. In a healthy retina, a single layer of RPE cells provides supportive functions such as nutrition and waste management for photoreceptors, the cells that process light to provide vision. Some retinal diseases, such as dry AMD, are caused by degeneration of RPE cells. When RPE cells are lost, photoreceptors lose their support system and degenerate. Ultimately, vision is lost.

Hadassah’s Eyal Banin, M.D., Ph.D., one of the developers of the technology and a leading investigator of the clinical trial, hopes that OpRegen will replace and support the patient’s dysfunctional RPE cells, thus helping to maintain the photoreceptors. Regardless, he believes the treatment may have additional effects.

“In all probability, additional mechanisms are at play, including the secretion of growth factors and modulation of the patient’s immune system,” Dr. Banin says. “But the biggest advantage of this type of therapy may be its communication with the surrounding cells and environment. This two-way interaction may help these remaining cells to survive and function properly.”

In mouse studies, a single subretinal injection of OpRegen organized into a natural single-layer structure and survived for the lifetime of the mice. “The proper organization and long-term survival of the therapeutic cells are a major advantage of this therapeutic approach, and increases the potential for benefit to patients,” says Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness.

Dr. Banin stressed that the treatment does not allow regeneration of lost photoreceptors, and therefore, if proven safe and effective, it would need to be applied at early stages of the disease, before a significant number of photoreceptors have degenerated.

He adds that OpRegen is produced using a proprietary process that drives hESC to develop into highly pure RPE cells. Also, because no animal products were used in derivation or expansion of the hESC, the company believes they are reducing safety concerns with the therapy.