EU approves first human embryonic stem cell trial in Europe

Advanced Cell Technology, Inc, a bitechnology company a biotechnology company applying cellular technology in regenerative medicine, yesterday said that it has received clearance from the UK Medicines and Healthcare products Regulatory Agency (MHRA) to begin treating patients as part of a Phase 1/2 clinical trial for Stargardt's Macular Dystrophy (SMD) using retinal pigment epithelium (RPE) derived from human embryonic stem cells (hESCs).

ACT has also received similar approval from the the Gene Therapy Advisory Committee (GTAC), which is responsibile for the ethical oversight of proposals to conduct clinical trials involving gene or stem cell therapies in the UK. The European Medicines Agency (EMA) had earlier granted orphan drug designation for the company's RPE cell product use for treating SMD.

''This is another important milestone for ACT and for the field of regenerative medicine,'' says Gary Rabin, chairman and CEO of ACT. ''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. We recently announced the dosing of the first patients in our Phase 1/2 clinical trials for Stargardt's macular dystrophy and dry age-related macular degeneration (dry AMD) with hESC-derived RPE cells in the US, and both patients successfully underwent the outpatient transplantation surgeries.

"Clearance from the MHRA to begin an SMD trial in the UK is the first step in our European clinical trial programme. Europe not only represents the world's second-largest pharmaceutical market, but it is also home to some of the best eye hospitals and surgeons in the world. Building international relationships around our clinical programs, such as with Professor James Bainbridge at Moorfields Eye Hospital is very important to our strategy of developing new regenerative medicine therapies.''

Stargardt's Macular Dystrophy affects an estimated 80,000 to 100,000 patients in the US and Europe, and causes progressive vision loss, usually starting in people between the ages of 10 to 20. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, the retinal pigment epithelium.

The first patient to be treated in the US with stem cell-derived RPE cells was a young woman who was already legally blind as a consequence of this disease. This newly-approved clinical trial in Europe will be a prospective, open-label study designed to determine the safety and tolerability of RPE cells derived from hESCs following sub-retinal transplantation to patients with advanced SMD, and it is similar in design to the FDA-cleared US trial initiated in July.