The European authorities have given the go ahead for trials to treat patients with Stargardt’s Macular Dystrophy (SMD) using retinal pigment epithelium (RPE) derived from human embryonic stem cells (hESCs). If successful, the trial may pave the way to an effective treatment not only for SMD, but also for other degenerative diseases such as dry age-related macular degeneration (AMD). The trial will be the first in Europe to use hESCs.

The European Medicines and Healthcare Products Regulatory Agency (MHRA) announced today, Thursday, that it had given approval for the trials, which will begin in the next few months, to be conducted at Moorfields Eye Hospital in London, UK. They will be led by Professor James Bainbridge, who is consultant surgeon at the hospital and also Chair of Retinal Studies at University College London (UCL).

Bainbridge, a retinal surgeon, told the press:

“The ability to regenerate 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.”

Stargardt’s Macular Dystrophy (SDM) is a currently untreatable inherited eye condition that affects around 80,000 to 100,000 mostly young people in Europe and the US. It is a form of macular degeneration that results in progressive vision loss, and usually starts when people are in their teens. It is one of the leading causes of juvenile blindness in the world.

The patient eventually becomes blind due to degeneration of the the retinal pigment epithelium, the pigmented layer of the retina, resulting in loss of photoreceptors, the cones and rods that help us see.

The first person to be treated for the condition using RPE cells derived from stem cells was a young female patient who had already gone blind with SMD. The US Food and Drug Administration (FDA) gave the go-ahead for the US trials to start in July this year.

The European trial will be of a similar design to the US one: it will be a prospective, open-label study designed to determine the safety and tolerability of the treatment, which uses RPE cells derived from hESCs following sub-retinal transplantation to patients with advanced SMD.

The patients will receive the RPE cells via an injection into the retina. The operation will last up to an hour.

Bainbridge will be conducting the trials at the National Institute for Health Research (NIHR) Biomedical Research Centre based at Moorfields and the UCL Institute of Ophthalmology. He said:

“There is real potential that people with blinding disorders of the retina, including Stargardt disease and age-related macular degeneration, might benefit in the future from transplantation of retinal cells.”

Professor Peng Khaw, director of the biomedical research centre at Moorfields, said:

“We are delighted to be the site for these very exciting new clinical trials in stem cell therapy, which have the potential to give hope and make such a difference to the lives of people with currently untreatable blinding retinal conditions.”

The biotechnology of the trial has been developed by US company Advanced Cell Technology (ACT). They have received a similar approval from the Gene Therapy Advisory Committee (GTAC), which has responsibility for the ethical oversight of proposals to conduct clinical trials involving gene or stem cell therapies in the UK.

Gary Rabin, chairman and CEO of ACT, said the European go ahead represents:

“… another important milestone for ACT and for the field of regenerative medicine.”

“We are pleased that the Moorfields Eye Hospital in London has agreed to participate as a site for this study as we continue to assess the capabilities of hESC-derived RPE cells to repair the retina and reduce the impact of these devastating eye diseases,” he added.

ACT has recently announced the dosing of the first patients in the US trial, both of whom have successfully undergone the transplantation surgery as outpatients.

ACT’s chief scientific officer, Dr Robert Lanza, said this is the first embryonic stem cell trial that has ever been approved anywhere in the world outside the US.

“We believe that transplanting new, healthy RPE cells may provide an effective treatment for SMD and perhaps other macular degenerative diseases such as dry AMD,” said Lanza.

“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,” he added.

Written by Catharine Paddock PhD