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Crystal ball gazing: human embryonic stem cells in 2012

31 January 2012
Appeared in BioNews 642
In the last few months of 2011, a couple of stories on human embryonic stem cells (hES cells) hit the headlines. Both were bad news for stem cell researchers.

First, in October, the Court of Justice of the European Union issued its decision in BrĂ¼stle v Greenpeace, declaring inventions related to the use of hES cells as not patentable (reported in BioNews 630). The second blow came a month later; the discontinuation of Geron's clinical trial in spinal cord injury (reported in BioNews 634), just under a year after it began, left many in the science community in a state of shock.

And yet this year began with pair of rather more encouraging stories: a clinical trial for dry age-related macular degeneration (a leading cause of blindness and visual impairment) and Stargardt disease (a form of inherited macular degeneration affecting younger people) at University of California Los Angeles (reported in BioNews 642) and the first publicly available fully human clinical grade hES cell lines derived by the team at King's College London (reported in BioNews 637).

There is no doubt that 2012 will bring a similar mixed bag of both disappointing and encouraging news. Which stories will stay in the memory will depend on whether you see a glass of water as half empty or half full. Either way, we should be a step closer to the truth about the power of hES cells.

In today's other BioNews comment article I talk about the implications of the Los Angeles macular degeneration stem cell trial but will add this here: in early clinical trials the priority is always patient safety - in fact that's what the trials set out to establish. Efficacy and economics come later. If anyone expects that the blind patients will regain vision completely after treatment they risk being unnecessarily disappointed. Just remember, because prospects of 'success' were unreasonable from the outset in Geron's trial, a similar safety study, the value of the company's shares dropped by 60 percent in the first nine months of 2011 (the first patient was treated in October 2010).

And other trials are gearing up in macular degeneration. Last year, Advanced Cell Technology received approval for a trial also using hES cell-derived retinal pigment epithelium cells from the Gene Therapy Advisory Committee, the body responsible for ethical oversight of proposals for clinical trials involving gene or stem cell therapies in the UK. The first patient was treated at Moorfields Eye Hospital on 20 January 2012 by a team of surgeons led by Professor James Bainbridge. The same hospital is also a site where, in a collaborative effort with Pfizer, The London Project to Cure Blindness is likely to commence clinical trials for the same disease - age-related macular degeneration - in 2012.

Moving on, there will many more clinical grade hES cell lines surfacing in the UK, not only from King's, but also from other derivation centers at the universities of Manchester, Newcastle, Sheffield as well as at Roslin Cells, a private company. Together with the clinical trials at the Moorfields Eye Hospital, the efforts of researchers at these centres should bring the UK back to the forefront of hES cell-based research, particularly at that all-important 'translational' bridge between lab bench and bedside.

However, we should not forget that in August 2011, the USA National Institutes of Health approved funding for clinical grade hES cell lines, originally made in Singapore from embryos imported from Australia (1). The lines are now distributed through a California Institute of Regenerative Medicine-funded company BioTime for minimal costs to California-based researchers. But it is probably too early for 2012 to see these lines in clinical trials in California. How about Singapore or Australia?

There are other intriguing developments outside the US and the UK. Last year it emerged that the South Korean and Indian authorities are promoting regulated translational stem cell research in their countries. I would not be surprised if stem cell giants such as RNL in South Korea or Reliance Life Sciences and Stempeutics in India, venture to a hES cell-based clinical trial.

All this sounds pretty optimistic and is promising a rosy future for hES cell-based translational research, especially in UK and at least in the near future. Shouldn't we temper this with a healthy dose of cynicism? Yes, we should: we still do not have proof of the concept that hES cell-based therapy can successfully mend damaged tissue and restore rather than just improve its function. Taking the wrong route may hamper cell-based therapy much more than a decision by the Court of Justice of the European Union or discontinuation of a promising clinical trial. What if our approach is wrong and in order to get the best out of hES cells in regenerative medicine, we have to combine cell therapy with gene therapy and biomaterials (encapsulation)? It is about time we think about taking alternative routes.

1) The Generation of Six Clinical-Grade Human Embryonic Stem Cell Lines
Cell Stem Cell |  15 September 2007
15 October 2012 - by Dr Dusko Ilic 
It took 50 years for the Nobel committee to acknowledge one of the key developments in biology...
25 June 2012 - by Antony Starza-Allen 
Six major UK research funding bodies have called for the continued funding of human embryonic stem cell (hESC) research in the EU's programme for research and development...
8 May 2012 - by Dr Greg Ball 
Human embryonic stem cells (hESCs) are primed to self destruct in response to DNA damage in the developing embryo, scientists have reported. The research found that hESCs are highly responsive to DNA damage in the early stages of development, and can commit suicide for the greater good...
13 February 2012 - by Dr Maria Botcharova 
Three women have reported a significant improvement in sight following gene therapy in both their eyes. Initially, they received the therapy in just one eye, but this latest study demonstrates the treatment was also successful in the other...
6 February 2012 - by Ruth Retassie 
US company StemCells Inc have received Food and Drug Administration (FDA) authorisation to carry out clinical trials of their treatment for one of the leading causes of blindness in over 55-year-olds...
31 January 2012 - by Dr Dusko Ilic and Dr Emma Stephenson 
Last week, Advanced Cell Technology (ACT) of Massachusetts, USA, made two important announcements regarding human embryonic stem (hES) cell-based therapies for the potential treatment of Stargardt's dystrophy and age-related macular degeneration, two devastating degenerative disease leading to blindness....
16 January 2012 - by Professor Allan Pacey 
Crystal ball gazing has never been my strong suit but I found myself rising to the challenge now that I have taken over the mantle of Chairman of the British Fertility Society. What is clear is that 2012 should turn out to be an interesting year...
3 October 2011 - by MacKenna Roberts 
On 8 September 2011 the Anscombe Bioethics Centre (a Roman Catholic organisation named after the philosopher Elizabeth Anscombe) organised the conference 'Human Embryo Research: Law, Ethics and Public Policy'. This was topical, in a month when it was announced that Europe's first clinical trials using human embryonic stem cells to treat Stargardt's macular degeneration (an incurable disease causing blindness in youth) would take place in the UK...
26 September 2011 - by MacKenna Roberts 
The short film 'Stem Cell Revolutions: A Vision of the Future' uses interviews to document how stem cells have 'vitally changed our understanding of the human body'. The film opens with a voiceover by the film's celebrity commentator novelist Margaret Atwood: 'Sometimes it seems stem cells are proposed as the answer to everything... What can't they do?'...
19 August 2011 - by MacKenna Roberts 
Should human embryonic stem cell research be deemed unethical for its embryo destruction? The US court decision in Sherley v Sebelius on 27 July 2011 to allow federal funding of this research set a global precedent. The meaning of research was divided into two categories: that which directly involves embryo destruction and that which does not...
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