Saturday, April 27, 2013

Advanced Cell Technology initiates higher-dosage patient treatment in European clinical trial for Stargardt's Disease

Advanced Cell Technology, Inc., a pioneer in the field of stem cells for treating conditions such as macular degeneration. has announced treatment of the first patient in the third dosage cohort, and seventh patient overall, in its European Phase I clinical trial for Stargardt’s macular dystrophy (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). 

The patient was injected with 150,000 hESC-derived RPE cells, as compared with the 100,000-cell dose used in patients of the second cohort. The surgery was performed on Friday, April 19, without any complications, and the patient is recovering uneventfully.

The company declared that they are past the halfway point in all three of their clinical trials on both continents. They have also announced that the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP) has officially granted their hESC-derived RPE cells orphan medicinal product designation for the treatment of SMD. This will likely provide a number of benefits to them, as in protection from competition, as well as for the patients through reduced fees.
The Phase 1/2 trial is designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation in patients with SMD at 12 months, the study’s primary endpoint. It will involve a total of 12 patients, with cohorts of three patients each in an ascending dosage format.
Stargardt’s disease or Stargardt’s Macular Dystrophy is a genetic disease that causes progressive vision loss, usually starting in children between 10 to 20 years of age. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, called the retinal pigment epithelium, which is the site of damage that the company believes the hESC-derived RPE may be able to target for repair after administration. Clink here to read more, 

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