US scientists report in The Lancet the first successful use of embryonic stem cells to treat eye disease (macular degeneration) in humans. The study was done on two patients with advanced eye disease, reporting some vision improvement after four months of the stem cell treatment. Below Australian and UK experts respond.

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* ‘Embryonic stem cell trials for macular degeneration: a preliminary report’ by Steven Schwartz et al. is published in The Lancet, 23^rd January 2012.

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Professor Martin Pera is Chair of Stem Cell Science at the University of Melbourne and Program Leader of Stem Cells Australia

“The report in the Lancet today from scientists at UCLA and Advanced Cell Technologies provides hope for patients suffering from dry macular degeneration, a very common cause of blindness. Although the work is preliminary, the study shows that replacement of retinal pigment epithelial cells (the cell type that is lost in this disease) by grafts derived from human embryonic stem cells appears to be safe, and may lead to an improvement in vision. This landmark study is the first published account of a human trial of embryonic stem cell based therapy. The authors described results from only two patients, and it is possible that the improvements that they observed may be related to other factors apart from the graft itself. Nonetheless the early findings are most encouraging.

Macular degeneration is a very promising target for stem cell therapies, because the required cell type can be efficiently produced from embryonic stem cell cultures, and because the eye is easily accessible for surgery and for monitoring the progress of the graft. Centres in California, the United Kingdom, and elsewhere are also progressing towards clinical trials of stem cell therapies in this disease. It will soon be clear whether these preliminary results published today are a harbinger of a new era in cell therapy for this devastating medical condition.”

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Dr Bernie Tuch is Director of the New South Wales Stem Cell Network

“This is a wonderful concept which should be strongly supported. It provides hope that something positive can be done about it for people who are losing their vision.”

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Dr Andrew Laslett is Research Group Leader, Stem Cells, CSIRO Materials Science & Engineering, Victoria

“This report allows cautious optimism that cells derived from human embryonic stem cells may be useful for the treatment of a common cause of blindness, namely macular degeneration. It is very early days and only two patients have been reported on to date but this first ever peer-reviewed report of a human embryonic stem cell-based human clinical trial demonstrated no adverse side effects and both patients showed mild improvement in their vision. The long term hope, based on these results, is that earlier intervention may lead to greater improvements in visual acuity for people with macular degeneration and that these improvements will persist and be safe.”

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Associate Professor Kuldip Sidhu is Director of the Stem Cell Lab and Chair of Stem Cell Biology at the University of New South Wales

“This is an another significant proof of principle that hESC-derived cells have potential in therapeutics if produced carefully avoiding residual hESCs that may cause teratoma - a major concern so far in the use of such cells. In this study a pure population of RPE derived from hESCs were transplanted into the patients eyes diagnosed with macula degeneration. Although only the phase I trials on two patients were carried out and the short term follow up for four months shows cell survival and apparently no adverse effects or tumours formation - a significant way forward in the clinical use of such cells in future. It is too early to conclude that clinical outcome is due to transplanted cells, it may simply be a trophic effects as also pointed out by the authors in this publication. A further long term follow up on these patients is essential to rule out adverse affects if any. Apparently the protocol developed for obtaining RPE from hESCs in Dr Robert’s lab so far involves the use of feeder cells from animals that can be avoided in the future as feeder-free systems are evolving rapidly.”

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Expert response from UK scientists

Compiled by our colleagues at the UK Science Media Centre

Expert reaction to preliminary data on stem cell trial for macular degeneration, as published in The Lancet*

Professor Daniel Brison, Co-Director of the North West Embryonic Stem Cell Centre, Manchester, said:

“This is a very exciting moment for embryonic stem cell therapies.  This is the first peer-reviewed scientific report showing that cells derived from human ES cells can be transplanted safely into a patient with no sign of complications.  Although the study is limited to safety considerations, very small in scope, and at a very early stage, this is nonetheless a ground breaking moment for embryonic stem cell therapies.  It is also very significant for the UK that the second trial of these therapies has now begun in London, only 4 months behind the US trial.  These trials have used human ES cells created at research grade and this was possible as they were transplanted into the eye which is a very localised site in the body.  In order to realise the full potential of ES cell therapies in the future, it will be very important to use the new generation of clinical grade ES cells now being produced in the UK.”

Professor Chris Mason, Chair of Regenerative Medicine Bioprocessing, University College London, said:

“The preliminary data on the two US patients treated using human embryonic stem cell-based therapies in June 2011 is highly encouraging, but is only the start of gathering the necessary safety data before it is possible to test if the therapy will have an impact on patients’ vision. Overall the process of testing for safety and efficacy is likely to take a minimum of 5-10 years before the potential therapy could enter routine clinical practice.

“It is not surprising that the first European human embryonic stem cell-based therapy was carried out in London, given that the UK is a world leader in cell therapy.

“The safety of embryonic stem cell-based therapies in patients is now slowly starting to emerge with both Geron data for spinal cord injury and now ACT for retinal disease. It is still a long way to go before we will have the answer as to whether embryonic stem cell-based therapies will be safe and efficacious, but progress continues to be made towards striving for the ultimate goal of life-changing therapies for patients and their carers.”

Dr Dusko Ilic, Senior Lecturer in Stem Cell Science, Kings College London, said:

“The most important thing is that Robert Lanza and his team at the Advanced Cell Technology get across a message to the media and the public that ongoing clinical trials for dry age-related macular degeneration and Stargardt’s disease with retinal pigment epithelium (RPE) derived from hES cells are safety trials. Even though in preclinical trials, the RPE were capable of extensive photoreceptor rescue in an animal model of retinal disease, resulting in improvement in visual performance without evidence of untoward pathology, we should keep in mind that people are not rats. The number one priority of initial clinical trial is always patient safety. If everyone expects that the blind patients will see after being treated with hES cell-derived RPE, even if the treatment ends up being safe (which is what Advanced Cell Technology are trying to determine in this trial), they risk being unnecessarily disappointed.”

Professor Peter Coffey, Director of the London Project to Cure Blindness said:

“At last seeing fruits of human embryonic stem cell research entering clinical trials. This will help determine the safety of these therapies. I am immensely happy that this has happened in the eye. And will only help those patients with, until now, blinding eye diseases. Hopefully we will be able to enter our own clinical trials using embryonic stem cell therapy soon.”

* ‘Embryonic stem cell trials for macular degeneration: a preliminary report’ by Steven Schwartz et al. is published in The Lancet today, 23^rd January 2012.