Big guns back stem cell therapy for AMD

The pioneering stem cell therapy, developed by British researchers to tackle AMD, has received the financial backing of pharma giant, Pfizer Inc., which is expected to pump in £50 million over the next three to five years.

The treatment, led by scientists and surgeons from the Institute of Ophthalmology at University College London and Moorfields Eye Hospital involves replacing a layer of degenerated cells with new ones created from embryonic stem cells. The collaboration will examine how human embryonic stem (hES) cells differentiate into retinal pigment epithelium (RPE) with the goal of developing stem cell-based therapies primarily for wet and dry macular degeneration (AMD).

The project is led by Professor Pete Coffey who is working alongside Lyndon da Cruz, a surgeon at Moorfields. The team used embryonic stem-cells to grow replicas of the bad cells on a thin artificial membrane, inserting it behind the retina of pigs and rats, restoring normal sight. The team believes human trials are just two-years away, which will be only the second human trial using embryonic stem-cells, in the world behind the US.

Professor Coffey said: “We have nearly 14 million people in Europe with AMD. This will ensure that the therapy gets through to clinical trials in a safe and effective manner. The introduction of new technology needs confidence. We have not only the benefit of Pfizer's experience of the regulatory process and their expertise in stem cell technology but the ability, if this works, to produce on a much larger scale. It has huge implications, not only for our project, but for the field of regenerative medicine as a whole. And it is great that Britain is at the forefront of this research.Ѽ

Professor Peng Khaw, director of the Biomedical Research Centre at Moorfields and the UCL Institute of Ophthalmology, added: “This shows that stem cell therapy is coming of age. It offers great hope for many sufferers around the world who cannot be treated with conventional treatment.”

“We are excited to be working with pioneers in the field of stem cell ophthalmology from UCL,” said Ruth McKernan, PhD, Chief Scientific Officer of Pfizer Regenerative Medicine. “While we have much to learn about how stem cells can be used therapeutically, we are confident that this relationship will increase that understanding and help us advance to a time when our work may benefit patients worldwide.”

Under the terms of the agreement, Pfizer will provide funding to UCL to enable research into the development of stem cell-based therapies for AMD as well as other retinal diseases. Pfizer is granted exclusive worldwide rights to develop and commercialize an RPE stem cell-based therapeutic in the ophthalmology field. After the completion of preclinical safety studies, Pfizer will have the option to conduct clinical trials to determine efficacy of treatment and commercialize any resulting product.