The independent group of medical experts: the Data Safety Monitoring Board (DSMB) which is monitoring biotech BioTime’s Phase I/IIa OpRegen® clinical trial, has authorized the Company to move forward with enrollment for cohort 3. This follows data from the first two cohorts that supported the treatment of Dry Age-Related Macular Degeneration (Dry-AMD) with the cell therapy.
Dry-AMD is the leading cause of blindness in people over 60. Caused by loss or dynfunction of the layer of retinal pigment epithelial (RPE) cells generally in the macula – the region of the eye responsible for the sharp, central vision that is utilized in facial recognition and activities such as reading and driving. RPE cells support light detecting photoreceptor cells that are vital for vision. To combat this degradation of the RPE layer, OpRegen® injects a suspension of RPE cells subretinally. A proprietary process that drives the differentiation of human pluripotent stem cells is used to generate high purity OpRegen® RPE cells; an allogeneic, off-the-shelf product that is hoped to require just a single procedure.
Cohort 1 and 2 studies of the Fast Track designated therapy demonstrated that the transplanted OpRegen cells remained in place at the one year follow up, and uniquely in an area of the scar that was completely depleted of RPE. There was also possible evidence of a biological response with some areas appearing to show structural improvement. Cohort 3 will focus on optimizing the cell concentration and volume prior to the treatment of an earlier stage of the disease in cohort 4. Patients will be enrolled at the company’s existing sites in Israel and a further two sites in the US with treatment aiming for completion by the end of 2017.
One of the trial’s lead ophthalmologists, Richard McDonald commented, ‘Our team is enthusiastic about the early data showing a positive response from the implanted cells. The DSMB’s approval is an important milestone. We look forward to enrolling our first patient in the trial as this is a debilitating disease.’
Source: BioTime Receives DSMB Approval to Start Third Patient Cohort in Clinical Trial for Dry-AMD; Commences Patient Enrollment in the US Press Release