Advanced therapies back on the table

Stem cells: back in the political arena.

But some European parliamentarians are trying for ethical reasons to exclude therapies developed using embryonic stem cell research from the scope of the law. Their attempt to limit the scope of the law has sparked a last-minute lobbying drive from the industry to try to avert the exclusion.

Scientists and businesses alike are calling for the law they say will boost Europe’s competitiveness in the most high-tech areas of medical research. For the most part they are confident that politicians have understood the need for a pan European regulation – mirroring the system for approving pharmaceuticals - covering products including gene therapies, replacement tissues such as skin, cartilage and bone, and cell replacement therapies.

A maze of regulations

Gene, tissue and cell therapies offer hope to thousands of patients with conditions that are untreatable using conventional medicines. But until now approving the advanced therapies has been complicated because they aren’t covered by standard, Europe-wide pharmaceutical rules.

The proposed regulation would harmonise the market approval procedure for these types of therapies across the EU, saving time and money for companies in the field.

With production and research facilities in 15 different EU countries Genzyme is one of the most active players on the European advanced therapies scene. Its cell therapies include Carticel, which can rebuild damaged human cartilage by growing it on a matrix using the patient’s cells, and then cutting the newly grown cartilage to the required shape.

Similarly its Epicel therapy uses cells from a burns victim to grow new skin. Both treatments are available throughout the United States, but are only available in one or two countries in the EU because of the lack of a Union-wide legal framework.

The company is researching several potential gene therapies including one for macular degeneration – a form of blindness, and another for peripheral arterial disease.

“Genzyme has several products either available or in development for which this regulation would give the first possibility of a European marketing authorization, making them potentially available to European patients,” said Wills Hughes-Wilson, Genzyme’s director for health policy in Europe.

The law will spur the development of new therapies, just as an earlier law creating a single approval system for orphan drugs boosted research in that area, Hughes-Wilson said.

It worked for orphan drugs…

Orphan drugs treat rare life-threatening conditions that affect no more than five in 10,000 people in Europe. A law passed in 2000 was designed to promote the development of orphan drugs, because without help, there isn’t the commercial incentive to develop the drugs.

“Before the law was adopted in 2000 there were only a tiny number of orphan drugs in Europe. Now 434 orphan product designations have been granted and 34 orphan medicinal products have been approved with marketing authorisation for the whole EU,” Hughes-Wilson said.

The advanced therapies regulation calls for the setting up of a committee of experts within the UK-based European Medicinal Evaluation Agency (EMEA) to handle gene, cell and tissue therapies.

“EMEA authorisation will help build confidence in these cutting edge treatments throughout the medical sector,” she said. “The therapies would be considered inside the pharma family, so doctors will know more about them and will be able to count on them applying the high standards of the EMEA,” Hughes-Wilson said.

Research into gene and cell therapies is already covered by Europe-wide laws but not the marketing approval process.  Hughes-Wilson argues that grouping them together with tissue therapies under the new law is the logical way to clearing the path for all three types of treatment.

“Including gene and cell therapies within the scope of the new law streamlines the legal framework. By creating a new law they will in effect be cutting red tape,” she said.

Concern from researchers, too

It’s not just pioneering pharmaceutical companies that are calling for the new regulation. Scientists need to know how and where their work will eventually be applied, said Jane Farrar, who researches into gene therapies at Trinity College, Dublin.

“The law will harmonise regulations for market approval right from the lab bench to the bedside, making the approval process simpler. For me it’s important that I can see an end point for the approval process – it’s all about getting the therapies into the market place,” Farrar said.

The possibility that therapies derived from embryonic stem cell research could, at this late stage in the lawmaking process, be excluded from the regulation angers scientists including Farrar.

Over the past two months lawmakers from the three main European Union institutions – the European Parliament, the Council of national government ministers and the European Commission – have been negotiating a compromise text they all hope can be agreed on when the European Parliament meets later this month.

A deal was reached that included therapies derived from stem cell research, but members negotiating for the European Parliament last week changed their position and now insist that stem cell research be excluded from the reach of the proposed law.

“This law doesn’t deal with ethical issues; it deals with organisational issues,” Farrar said, adding: “Just because it aims to harmonise the market authorization process doesn’t stop individual countries in the EU from deciding for themselves whether to permit a therapy based on embryonic stem cell research.”

By excluding this area of research, the lawmakers would be stopping advances in a wide range of medical fields. “It’s important to have a broad-based portfolio of research and not to limit research in the field of embryonic stem cells, because it is needed to stimulate research in all kinds of areas,” she said.

Harm-free extraction?

Within the field of stem cell research scientists are exploring ways of taking a stem cell from the morula, a small cluster of cells that later develop into an embryo, without harming that embryo in any way.

If they succeed then stem cell researchers will no longer need to waste human embryos in order to source early stage stem cells. it’s not her field but Farrar said it is likely this will be possible in future.

“Excluding embryonic stem cell research from the scope of the new law on ethical grounds would stop scientists overcoming the very ethical issues raised by using embryos,” she said.

Genzyme’s Hughes-Wilson is equally passionate in her defence of embryonic stem cell research. “We have to research all channels at the same time because we don’t know what will lead to medical breakthroughs,” she said.

“We are very disappointed about the latest move to exclude stem cell research from the scope of the law,” Hughes- Wilson said.

Since embryonic stem cell research can now be funded with EU money, it would be “inconsistent” if the fruits of this research couldn’t be approved for use in Europe, Hughes-Wilson said.

The parliamentarians trying to exclude embryonic stem cells have tabled two amendments to that effect through the legal affairs committee of the European Parliament.

It all hinges on the votes

With the compromise text now torn up, parliamentarians are going to have to vote on all the individual amendments instead of just voting once on the compromise text.

The outcome is far from certain. If the stem cell amendments are passed at the plenary meeting of the Parliament on 23 April, this would result in the debate being extended to a second reading in Parliament, and more three-way meetings. There is a chance that the disagreement could de-rail the proposed law altogether.

But the scientists and executives involved with advanced therapies remain hopeful. “They know this law would be a huge boost for European competitiveness, and would mean more high-tech jobs for scientists here. They see what good the law could do for society and how necessary it is,” Hughes-Wilson said.