September 23, 2011

expert reaction to announcement of human embryonic stem cell trial for macular degeneration

American biotechnology company Advanced Cell Technology (ACT) announced the launch of a clinical trial for a stem cell treatment for Stargard’s Disease, a condition that causes progressive blindness.

Professor Roger Pederson, Professor of Regenerative Medicine, University of Cambridge, said:

“The recent announcement [by ACT of their impending clinical trial in collaboration with Moorfields Eye Hospital] is an important first international cooperation in examining the safety and effectiveness of a therapy involving pluripotent stem cells. These stem cells are capable of forming all body tissues, and it is possible that the retinal cells formed from them will be useful in preventing age-related blindness as well as in treating certain genetic eye diseases. I believe we will now see increasing numbers of such trials in the US and UK, as pluripotent stem cells continue to reach the threshold of this and other clinical applications.”

Daniel Brison, Professor of Clinical Embryology and Stem Cell biology at The University of Manchester, said:

“The news of this trial is very exciting as it is the first to be approved in the UK and to benefit UK patients. Human embryonic stem cells are capable of giving rise to a range of cells and tissues which could provide treatments for a wide range of diseases. Now that this first trial has set a precedent, we hope to see many other clinical trials using human embryonic stem cells in the near future.”

Dr Dusko Ilic, Senior Lecturer in Stem Cell Science at King’s College London, said:

“There are no other alternatives to treat Stargard’s Disease and any improvement in daily life of patients will be of significant value.

“I am delighted to hear that a clinical trial using human embryonic stem cells derived cell therapy has been approved outside of the USA. European clinical trials would follow a similar one carried out in California, which is one of the most anticipated clinical trials in the history of regenerative medicine. Demonstrating safety and tolerability of hESC-derived cell therapy in a trial like this would open a new age in regenerative medicine.”

Professor Chris Mason, Chair of Regenerative Medicine Bioprocessing, Advanced Centre for Biochemical Engineering, University College London, said:

“ACT, a leading US company, by undertaking this important clinical trial in London endorses the UK’s position as world leaders in stem cell therapies. This is the first application of an embryonic stem cell-based therapy into the eye. Whilst principally a safety study, it will undoubted significantly add to the growing core of knowledge on cell therapies, thus helping advance the entire field.”

Ben Sykes, Executive Director, UK National Stem Cell Network , said:

“Following on from the Geron phase one trial last year this is the second safety trial of a cell therapy derived from human embryonic stem cells, and is another step along the way to understanding the potential of stem cell based therapies. It is only a safety trial in a low number of patients but the stem cell research community will await the results with interest.”

Science Media Centre Timeline Embryonic stem cell research November 1998 – a group led by Dr James Thomson at the University of Wisconsin isolate and grow the first human embryonic stem cells June 1999 – members of a White House ethics panel decide to recommend that federally financed scientists be allowed to derive stem cells from human embryos as well as to conduct research on stem cells derived by others August 2001 – President George W. Bush permits federal funding of embryonic stem cell research, but only on the 64 existing stem cell lines, and bans federal funding for research into lines created after this date February 2004 – researchers in South Korea led by Dr Hwang Woo-suk claim to have been the first to have cloned a human embryo and obtained stem cells for research – the work is later discredited December 2005 – investigation exposes serious fraud and ethical lapses in Hwang Woo-Suk’s landmark stem cell paper, which claimed to have produced a viable embryonic stem cell line from a cloned human embryo January 2006 – Science retracts Hwang’s key stem cell papers November 2007 – Shinya Yamanaka and colleagues publish a major paper on induced pluripotent stem (iPS) cells, involving reprogramming human skin cells so that they function as stem cells, without the need for using embryos. James Thomson publishes a separate paper at the same time drawing similar conclusions January 2008 – California based company Stemagen announce that they have created the first mature cloned human embryos from single skin cells taken from adults. These embryos can be harvested for patient-matching embryonic stem cells November 2008 – UK Human Fertilisation and Embryology Bill receives Royal Assent and becomes Human Fertilisation and Embryology Act, updating UK law on embryology research and assisted reproduction, and bringing it into line with scientific developments January 2009 – US corporation Geron receives clearance from the FDA to begin the first Phase 1 human clinical trial of a treatment involving embryonic stem cells, in this case a treatment for spinal injuries based on injecting spinal cells grown from embryonic stem cells into patients paralysed from chest down June 2009 – Barack Obama reverses George W. Bush’s policy restricting federal funding for stem cell research August 2009 – Geron’s clinical trial on spinal cord injury is halted by the FDA following the discovery of cysts found in the animal models of the trial February 2010 – Guildford-based company ReNeuron and scientists at the University of Glasgow receive approval from the Gene Therapy Advisory Committee (the national ethics advisory committee for gene therapy research) for the first Phase 1 human clinical trial for stem cell treatment of stroke August 2010 – FDA lifts halt on Geron’s clinical trial October 2010 – an American patient becomes the first person to be treated with embryonic stem cells as part of the Geron clinical trial into treatments for spinal injuries June 2011 – another US company, Advanced Cell Technology (ACT), begins Phase 1/2 clinical trials of stem cell treatments for two conditions that cause blindness, Stargardt’s Macular Dystrophy and Age-related Macular Degeneration (AMD) September 2011 – ACT receives approval to begin first trials in Europe of stem cell treatments for Stargardt’s Macular Dystrophy