Global Report: Ready ... Set ...

The European Parliament has launched a formal process for assessing therapies developed through stem-cell research. Ethical issues that have wrangled US regulators, though, still need attention.
Mar 01, 2007


Sarah Houlton
AS THE US GOVERNMENT continues to argue over whether stem-cell research should be funded—or even permitted—the European regulatory environment for advanced therapies is becoming ever more appealing to researchers. Advanced therapies—those involving cell therapy, gene therapy, and tissue engineering—are still under development. But for a raft of incurable conditions, many of them genetic and degenerative, such treatments offer the only real hope of a cure.

And now, Europe has moved another step toward exploiting modern biology techniques in medicine. The European Parliament's Environment Committee voted at the end of January to allow these advanced therapies, but notably left ethical decisions to individual member states.

If all goes according to the committee's recommendations, a single, centralized procedure would be instituted—under the European Medicines Agency (EMEA)—to assess the safety, efficacy, and quality of such therapies. A handful of treatments already are being developed, and clarity on the regulatory front would grow the field significantly.

A centralized procedure for assessing advanced biologics is much needed. While traditional medicines—small molecules and more traditional biologics—are addressed by EMEA in a fairly streamlined manner, the process for advanced therapies remains extremely fragmented. Some EU member states have no regulatory framework for these therapies at all, while guidelines vary wildly among others.

The decision has been welcomed by the European pharma industry, which is seeing some of its research into advanced therapies for diseases such as Parkinson's and cancer beginning to bear fruit. "Regulators must find a way to encourage the emergence of these new treatments while safeguarding the public interest," said Wills Hughes-Wilson, Genzyme's chair, health policy Europe, and chair of the working group on advanced therapies for the European bioindustries trade association EuropaBio. The committee," said Hughes-Wilson, "has taken a huge positive step."

Meanwhile, the controversial field of embryonic stem cells continues to raise debate. Regulators in the United Kingdom have put off making a decision about human–animal hybrid embryo research until there is more consultation on the subject. But, the delay actually came as a great relief for researchers, who were prepared for an outright ban.

Embryonic stem cells have enormous potential in the field of regenerative medicine, as they are capable of developing into any form of tissue, from bone to brain cell. But their use remains controversial, with polarized opinions on whether embryos represent human life or just the potential for it. Hybrid embryos, combining human DNA with animal eggs to produce a cloned embryo, are intended to overcome the shortage of human eggs; genetically, they would be almost entirely human.

In the wake of the continuing arguments about stem cells in the United States, the United Kingdom has benefited—several US scientists have relocated to Europe because of the more favorable regulatory environment. But a recent controversy looked like it would lead to a setback for researchers. In a clause buried within the UK government's white paper on reforming the regulatory system for fertility treatment and embryo research, there was a proposal to ban human–animal hybrid embryos. But the independent regulator, the Human Fertilization and Embryology Authority (HFEA), deemed the decision one that should fall under its jurisdiction.

"After weighing the scientific, legal, and ethical issues, the Authority decided that there needs to be a full and proper public debate and consultation as to whether, in principle, licenses for these sorts of research could be granted," said HFEA's chief executive Angela McNab.

She added that there is still no clear agreement even within the scientific community about the need for this type of research, and that it is important for HFEA to consider the science thoroughly, and test the claims about the research's potential benefits. There already has been a limited consultation within the auspices of the white paper, but this was only a small part of a much bigger debate. HFEA feels a more widespread debate is necessary.

Industry welcomed the delay, with the Bioindustry Association claiming that there is widespread public and scientific support for this type of research, which could lead to treatments for incurable diseases such as motor-neuron disease, multiple sclerosis, and Parkinson's.

"Preventing the research would dash the hopes of millions of patients," said BIA's chief executive Aisling Burnand. "It would also completely undermine the government's support for stem-cell research and its commitment to establishing the UK as a world-leading location for innovative science research."

Sarah Houlton is Pharmaceutical Executive's global correspondent. She can be reached at