Shares in Advanced Cell Technology (OTCBB:ACTC) powered ahead again today after the company confirmed that the US Food and Drug Administration (FDA) had cleared the company`s application to initiate a Phase I/II trial to treat Dry Age-Related Macular Degeneration (AMD). Advanced Cell Technology jumped more than 15% on the news.
Dry AMD, which affects between 20 and 25 million individuals in Europe and the United States alone, is a breakdown or thinning of the layer of retinal pigment epithelial (RPE) cells in the patient's macula, the region at the center of the retina responsible for high acuity vision. Over time, the progressive loss of RPE cells and accompanying loss of photoreceptors can cause severe vision loss and even blindness. There is currently no treatment for Dry AMD.
Advanced Cell Technology has developed a technique using RPE cells derived from human embryonic stem cells to replace lost RPE cells in the patient`s eyes.
Advanced Cell Technology has a proprietary RPE cell manufacturing process protected by a number of broad patents, as is the use of hESC-derived RPE cells for treating macular degeneration.
The Phase I/II trial will be an open-label study designed to determine safety and tolerability of the RPE cells following transplantation into patients. 12 patients will be enrolled in the trial, and though the first trial is focused on safety and tolerability, Advanced Cell stated its desire to conduct additional trials to examine the ability of its RPE cells to slow or halt the progression of Dry AMD.
"ACT is now the first company to receive FDA clearance for two hESC trials, and is now a true translational leader in the field of regenerative medicine," said Gary Rabin, Interim Chairman and CEO of ACT.
"It marks a major step forward, not just within the stem cell sector, but, potentially for modern healthcare techniques. We plan to proceed into the clinic with both of our hESC-based programs as quickly as possible. ."
"Dry AMD is the leading cause of blindness in individuals over the age of 55," stated Robert Lanza, MD, ACT's Chief Scientific Officer.
"As the population ages, the incidence of AMD is expected to double over the next 20 years, further exacerbating this unmet medical need. Using our clinical-grade hESC lines, we are able to generate a virtually unlimited and reproducible supply of healthy RPE cells.”
Lanza went on to note that only a small number of cells (less than 200,000) are required to treat a patient, indicating that manufacturing and distribution of RPE would be scalable. ACT`s Chief Scientific Officer added that the company was “very excited” about the opportunity for not only Dry AMD, but a separate Phase I/II trial using RPE cells to treat Stargardt`s Disease, which received FDA approval in November.