A US based company received permission to begin the first clinical trial in Europe involving human embryonic stem cells (hES). Advanced Cell Technology (ACT), based in Marlborough, Massachusetts, today received approval from the UK Medicines and Healthcare products Regulatory Agency (MHRA) to begin a trial that will treat 12 patients with macular dystrophy Stargardt. The disease strikes people between the ages of 10 and 20, causing a progressive loss of vision. There is currently no treatment.
Eye surgeon James Bainbridge of Moorfields Eye Hospital and University College London will lead the UK trial. He and his colleagues will inject into patients' eyes of the cells of the pigment epithelium of the retina that ACT-derived cells hES. In animal models, the cells were able to delay or even reverse the progression of the disease. Phase I / II will primarily examine the safety of the treatment, however.
Robert Lanza, scientific director of ACT, said the trial was essentially the same design as the US trial of the company with Stargardt's patients, which was approved last year. The fact that the US Food and Drug Administration approved the trial, however, was not a shoo-in with U.K. authorities said Lanza. "I thought it was going to sail through I was wrong; .. They were very strict"
The new trial is the first using hES cells to be approved outside the United States, said Lanza. the company plans to apply for approval for a second UK trial for macular degeneration, he added, would complement its US-based assay for the same condition. They then plan to wait until that the first results are in before starting any additional tests, he said. the first two US patients received cells of the company in July-a patient with Stargardt's disease and with related macular degeneration age. the first indications are positive, Lanza said. "We are very pleased with what we see today."
0 Komentar