Regenerative medicine is one of the most promising fields of medical research, offering the prospect of disease reversal. According to a special report in Science/Business, Europe currently leads the world in the therapeutic applications of a series of technologies that fall under the heading of regenerative medicines. These range from patches of decellularised pig tissue for use in repairing veins, to patient-specific cell therapies for repairing damaged knee cartilage and human embryonic stem cell-based (hESC) treatments for degenerative eye diseases.
The first European-developed therapy based on hESCs is due to enter the clinic in 2013. The product, developed in collaboration between Pfizer Inc and University College London, is a treatment for age-related macular degeneration, a cause of sight loss and blindness.
In October Science|Business convened business leaders, politicians, research funders, patients’ representatives and scientists to the British Embassy in Brussels to debate the challenges that exist for Europe to maintain its lead in the field. The goal was to translate publicly funded science into marketed products that could provide significant health benefits and deliver on the commercial potential of regenerative medicine.
Some EU member states and pro-life members of the European Parliament (MEPs) are lobbying for the existing restrictive rules on funding embryonic stem cell research to be further tightened in its successor, the proposed €80 billion Horizon 2020 program, which currently is under consideration by the European Parliament.
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. The triple-lock states that any EU-funded embryonic stem cell research must conform with the laws of the country in which it is undertaken; that the research is subject to ethical review; and that EU money cannot be used for the derivation of new human embryonic stem cell lines, or any research involving the destruction of human embryos. The European Council supports retaining the triple-lock agreement.
The European Commission’s approach to funding regenerative medicine research in Horizon 2020 is based on the assessment that it is potentially a high value technology that will deliver life-changing treatments. “It is a new paradigm for medicine, ” said Arnd Hoeveler, Head of the Unit for Advanced Therapies and Systems Medicine, DG Research and Innovation. An analysis by Science/Business of the Advanced Therapy Medicinal Products coming before the European Medicines Agency shows they are mostly sponsored by academics, charities and start-ups. Few larger and better-financed companies are involved in the field. “There is a need for public sector support if the promise of the new technology is to be realized, ” Hoeveler said.
The therapeutic power, the huge unmet medical demand, and the difficulties involved in scaling-up and commercializing regenerative medicines is highlighted by the research of Suchitra Sumitran-Holgersson, Professor of Transplant Biology at Gothenburg University, Sweden.
Sumitran-Holgersson has developed a technique for removing all the cells from a vein taken from a deceased donor and repopulating it with a patient’s own endothelial and smooth muscle cells, obtained by differentiating stem cells obtained from the bone marrow of the recipient. In the case of a 10-year-old girl who had an obstruction of the portal vein feeding blood between the intestines and the liver, the graft immediately provided a functional blood supply. And because the donor’s cells were replaced with her own, there is no need for the girl to take drugs to suppress her immune system.
When details of the case were published in The Lancet in June 2012, it drew thousands of emails and inquiries from around the world, convincing Sumitran-Holgersson there is a widespread need for this type of regenerative medicine. “How can I build the infrastructure and get the therapy out there?” she asked.
It is an unfortunate circumstance that cell therapies involve huge amounts of know-how that cannot readily be copied in a legal document such as a patent. However, intellectual property protection is important to investors, and therefore small companies and the innovative physicians they represent, are unlikely to get funded to start out on the long path to market unless they have patents.
