ACT Announces Europe's First Human Embryonic Stem Cell Transplant in Patient with Stargardt's Disease
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"Our clinical trial program for hESC-derived RPE cells has taken another critical step as we move forward with treating patients at Moorfields Eye Hospital," said Gary Rabin, chairman and chief executive officer of ACT. "The treatment of the first patient in Europe is tangible evidence that stem cell research and development of cell therapies is making progress. It is a milestone for scientists, stem cell advocates and patients hoping for cures as well as much as it is one for ACT. Stargardt's macular dystrophy affects up to 100,000 patients in Europe and North America, and causes progressive vision loss often ending with blindness. We are honored to be working with Professor Bainbridge at Moorfields Eye Hospital, and are very pleased with the smooth progress of the trial thus far."
Professor James Bainbridge, the study's principal investigator, said, "The patient, who is severely sight-impaired, underwent transplantation of fully differentiated retinal pigment epithelial (RPE) cells derived from human embryonic stem cells. There were no complications, and the patient has tolerated the surgical procedure well. We will be regularly monitoring the safety and tolerability of the transplanted cells. While this is primarily a safety trial, we will have the opportunity to image engraftment of RPE cells non-invasively and to assess any changes in sight. We are very excited to be working with ACT on the first human embryonic stem cell trial in Europe. Stargardt's macular dystrophy is a serious and progressive disease that usually starts between the age of 10 and 20 years. It includes degeneration of RPE cells in the macula at the center of the retina, the region specialized for high acuity vision. With the loss of RPE cells in the macula comes the eventual loss of light-sensitive photoreceptor cells, leading to blindness at the prime of life. We hope that transplantation of healthy RPE cells might also help in other significant degenerative eye diseases affecting the retina for which there are no effective treatment options -- particularly dry age-related macular degeneration which is the leading cause of blindness in Europe."
The Phase 1/2 trial will involve 12 patients, with cohorts of 3 patients each in an ascending dosage format and is similar in design to the FDA-approved U.S. trial that was initiated in July 2011. The open-label study is designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation in patients with Stargardt's macular dystrophy at 12 months, the study's primary endpoint.
"Stem cells provide the possibility of providing new treatment strategies for currently incurable retinal degenerative diseases," said Robert Lanza, M.D., chief scientific officer of ACT. "Although these initial studies are designed to determine the safety and tolerability of hESC-derived RPE, we eventually hope to treat patients earlier in the course of the disease, further increasing the likelihood the new cells will rescue photoreceptors and prevent visual loss. We recently initiated two clinical trials in the U.S. and are very pleased to be starting the first clinical trial in Europe. To-date, our preclinical and clinical data relating to the safety and effectiveness of this approach is very encouraging. We look forward to working with Professor Bainbridge and Moorfields Eye Hospital to obtain additional clinical data and to enroll further patients in this trial. As a scientist, it is satisfying to see years of benchside research finally moving into the clinic. We believe RPE is just the first of many different vital differentiated cell types that may help patients suffering from a wide spectrum of eye disorders caused by conditions ranging from diabetes to vascular and autoimmune diseases."
In August 2011, the National Institute of Health Research (NIHR) announced the award of Biomedical Research Centre (BRC) for Ophthalmology at Moorfields Eye Hospital NHS Foundation Trust and UCL Institute of Ophthalmology. The award provides 26.5 million pounds Sterling over five years to translate lab-based discoveries into new cutting edge treatments, technologies, diagnostics and other interventions in clinical settings.