First European Embryonic Stem Cell Trial Approved
September 23, 2011

First European Embryonic Stem Cell Trial Approved


The first ever European trials of human embryonic stem cell therapy will take place before the end of the year, various media outlets reported on Thursday.

According to Guardian Science Correspondent Ian Sample, the trial will take place at Moorfields Eye Hospital in London, and will involve treatment of a form of juvenile blindness known as Stargardt's macular dystrophy.

"Massachusetts-based ACT will start the trial of its treatment, which uses retinal pigment epithelium derived from human embryonic stem cells (hESCs), on 12 patients," explained Kate Kelland of Reuters. The trial was approved by the Medicines and Healthcare Products Regulatory Agency on Thursday and is currently scheduled to get underway in December.

In a statement released Thursday afternoon, ACT Chairman and CEO Gary Rabin called it "an important milestone" both for the company and "for the field of regenerative medicine."

"This is the first time an embryonic stem cell trial has ever been approved anywhere else in the world," added ACT Chief Scientific Officer Robert Lanza. "Stargardt´s disease is currently untreatable, and is one of the leading causes of juvenile blindness in the world“¦ We are excited to start these trials in Europe, and look forward to analyzing the data we continue to collect in our ongoing trials to determine the engraftment and function of the transplanted RPE cells."

The disease affects an estimated 80,000 to 100,000 individuals in the U.S. and Europe, according to ACT, and causes progressive vision loss, often starting when patients are between the ages of 10 and 20. The company notes that photoreceptor loss, combined with degeneration in a specific area of the retina (the retinal pigment epithelium), ultimately leads to blindness in most cases.

According to Kerry Sheridan of AFP, ACT was also the first company to launch a U.S. trial of embryonic stem cell treatment of Stargardt's disease. Their first trial, which launched in November 2010, has to date treated just a pair of patients, with researchers focusing on ensuring that the treatment was safe before analyzing the effectiveness of the treatments.

Lanza told Sheridan that the company was "pleased" with the results of the trials thus far, and that ACT was "in the process of scheduling the next two patients" for their American trials.

The UK trials will be led by Professor James Bainbridge, consultant surgeon at Moorfields Eye Hospital and the Chair of Retinal Studies at University College London (UCL), the company said.

"There is real potential that people with blinding disorders of the retina including Stargardt´s disease and age-related macular degeneration might benefit in the future from transplantation of retinal cells," the professor said in a statement. "The ability to generate retinal cells from stem cells in the laboratory has been a significant advance and the opportunity to help translate such technology into new treatments for patients is hugely exciting. Testing the safety of retinal cell transplantation in this clinical trial will be an important step towards achieving this aim."


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