Stem cell plays have cooled their jets since the news of Geron and Advanced Cell Technology becoming the first companies to receive U.S. Food and Drug Administration clearance to proceed with clinical trials. While perhaps not in the spotlight at the moment, Advanced Cell is still plugging ahead with their research to use retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs) to treat Stargardt’s Macular Dystrophy (SMD), one of the most common forms of macular degeneration in the world that leads to blindness.
On November 22, 2010, the Marlborough, Massachusetts-based industry leader received its U.S. FDA clearance to conduct human trials with the revolutionary therapy on Stargardt patients. Today, the Advanced Cell management announced that they have filed a clinical trial application with the European Medicines and Healthcare products Regulatory Agency (MHRA) seeking clearance to initiate its Phase 1/2 clinical trial using RPE cells derived from human embryonic stem cells (hESCs) to treat patients with SMD.
The proposed clinical trial will be a prospective, open-label study that is designed to determine the safety and tolerability of the RPE cells following sub-retinal transplantation to patients with advanced SMD, similar to the FDA-cleared U.S. trial which is set to commence in the first half of this year. During the review process, which requires a minimum of 60 days, the reviewers decide if an applicant is permitted to proceed with its proposed clinical trial and determine if any additional information is required from the applicant.
The team at Advanced Cell Technology is hoping that the European process will continue to follow in the tracks of U.S. approvals. Last year, the U.S. FDA granted Orphan Drug designation for ACT’s RPE cells for treating SMD, and earlier this year the company received a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) towards designation of this product as an orphan medicinal product for the treatment of Stargardt’s disease. ACT anticipates adoption of the EMA’s recommendation by the European Commission in coming weeks.
Gary Rabin, interim CEO of Advanced Cell Technology, commented, “With this filing, our initiatives in Europe are really starting to gain momentum. Through data from this proposed trial, and the two trials we are preparing to commence in the United States, we are eagerly anticipating beginning to assess the capabilities of our RPE cells to repair and regenerate the retina. As in the US, we also intend to file in Europe for clinical trials involving Dry Age-Related Macular Degeneration (Dry AMD) and other degenerative diseases of the retina, concurrently targeting the two largest pharmaceutical markets in the world.”
Since the original FDA-clearance announcement in November, shares of ACTC have surged from 4 cents to approximately 27 cents from low to high. Not maintaining the highs, the stock’s price has slid back in the area of 18 cents. Interested investors can learn more about Advanced Cell Technology on the corporate website at www.advancedcell.com