Moves by some Members of the European Parliament to ban research on human embryonic stem cell (hESCs) in the Horizon 2020 R&D programme would undermine the huge progress Europe has made in developing cell therapies, delaying the registration and availability of products that are far more effective than existing treatments - and constraining Europe’s competitiveness in the global push to commercialise regenerative medicines.
Special Report: Experts debate the future of stem cells (PDF)Europe is currently a world leader in the fundamental science underpinning regenerative medicine and cell therapy, and in its therapeutic use and regulation. But how can Europe build on its leading position?
View from Europe: Delivering on the vision of regenerative medicine and stem cells
Talking to the experts: Why the EU should support human embryonic stem cell research in Horizon 2020
This is the view of experts from patients’ groups, research charities, academe, industry, science and economic policy and regulators, who were brought together by Science|Business to discuss the implications of an end to EU support for hESC research, and scope what needs to be done now to build the regenerative medicines market in Europe, to the benefit of patients and the economy.
A full report of the meeting appears here. In-depth views of some of the experts are here.
Among the conclusions reached at the meeting:
1. Cutting hESC research would damage regenerative medicine and cell therapy as a whole, holding back critical basic science, undermining collaborations that are central to this multidisciplinary field, sending the wrong message to investors – and damaging innovation in Europe.
2. Induced pluripotent stem cells derived by reprogramming adult cells are an important breakthrough and a potent new tool for drug discovery, disease research and studying pluripotency, but they are not suitable currently for use in cell therapies.
3. As the ‘gold standard’ of pluripotency, hESC cells are needed as the reference for what is the normal pluripotent state, and for understanding the mechanisms that drive stem cells to become specialised cells.
4. The ruling of the Court of Justice of European Union in November 2011 that hESC-derived products are not patentable will not block their commercialisation. Products will be protected by the significant know-how that is involved in their development and by patents on surrounding technologies such as delivery systems and manufacturing processes. As a result there is no justification on the grounds of commercial potential for saying that hESC research should not be funded under Horizon 2020.
5. The moral high ground does not automatically belong to opponents of stem cell research, who are putting greater importance on embryos up to the age of 14 days than on doing something to benefit patients with life-threatening diseases.
Disproportionate effect
Although hESC research – which to date has received €107 million to fund 18 projects - represents a small proportion of the current EU R&D programme, Framework Programme 7, axing this funding would have a disproportionate effect, signalling that the EU was moving to a more restrictive regime just as the first clinical trial of an European-developed, hESC-based therapy gets underway.
That product, developed in a collaboration between Pfizer Inc and scientists at University College London, consists of retina cells derived from hESCs, for treating age-related macular degeneration, an eye disease that causes sight loss and blindness. It is a pointer to the therapeutic potential of cell therapy as a whole.
“If there’s a negative funding environment it will send out a negative message about the field, it will be more difficult to get funding for any type of stem cell research, and more difficult to form collaborations,” Katherine Littler of the Wellcome Trust, Europe’s largest medical research charity, told the meeting.
Magda Chelbus, Director of Science Policy at the industry body, the European Federation of Pharmaceutical Industries and Associations agreed, noting that member states which favour a more restrictive approach have a disproportionate influence on the environment for R&D as a whole, “killing off the tools” that are required to support the international, multi-disciplinary work that is central to commercialising regenerative medicine and cell therapies.
Moral asymmetry
This disproportionate influence also feeds into the moral sphere, believes Alastair Kent, Director of Genetic Alliance UK, an umbrella group representing rare diseases patients’ groups. The moral high ground does not belong to opponents of hESC research. “They are putting greater importance on embryos of up to 14 days, than on doing something to benefit patients with life-threatening diseases,” he said.
Cell therapies are advancing in the clinic, but there remain many unknowns in terms of the basic biology of stem cells, what makes them remain stem-like and what prompts them to differentiate into specialised cell types. hESCs, as the ‘gold standard’ stem cells are central to investigating these issues. Translating this understanding into therapies is not a one-way street – clinicians need access to basic research as they see how - and if - treatments work, allowing therapies to be rationally improved.
The approach the EU and member states have taken to date to regulating and funding regenerative medicine research, and in fostering its translation through to robustly manufactured products, has given Europe a clear lead. Researchers have been attracted to come and work in Europe; start-ups have spun-out of universities; companies are investing in running clinical trials and building supply chains and manufacturing capabilities.
Uncertain legal climate
Europe can be seen to have benefitted from the uncertain legal climate that has hobbled hESC research in the US. That led several scientists to leave for Europe, and was a factor in Pfizer Inc’s decision – as the first big pharma to announce it was to invest in stem cell therapies – to set up its research unit in Cambridge, UK.
While President Obama lifted the ban on federal funding of hESC research on coming into office in January 2009, legal challenges meant the path was not cleared until August 2012. Now with Obama’s re-election hESC research should be free of constraints. At the same time, countries including China, Brazil, South Korea and Singapore are making considerable investments in regenerative medicine and cell therapies.
In line with the strategic view elsewhere, the European Commission sees regenerative medicine is a high value technology that will deliver life-changing treatments, Arnd Hoeveler, Head of Unit for Advanced Therapies and Systems Medicines told the meeting. An analysis of cell therapy products coming before the European Medicines Agency shows they are mostly sponsored by academics and small companies that find it hard to attract the capital needed to withstand the high costs and long development timelines involved. “There is a need for public sector support if the promise of the new technology is to be realised,” Hoeveler said.
Triple lock
The EU Research Commissioner Máire Geoghegan-Quinn has made it clear she intends to resist any change, noting that a great deal of time and effort was spent sorting out the existing “triple-lock” agreement negotiated for FP7. The triple-lock states any EU-funded hESC research must conform with the laws of the country where it takes place; that the research is subject to ethical review; and that EU money cannot be used to derive new hESC lines, or for any research involving the destruction of human embryos.
The European Council also supports retaining the triple-lock agreement in Horizon 2012.
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