17 Jul 2013   |   Viewpoint

Collaboration is the critical ingredient in cell therapy

There’s a one word answer to removing bottlenecks identified in the recent House of Lords report on translating stem cell science into effective therapies, and that’s Collaboration. “It’s a no-brainer,” says leading researcher, Fiona Watt

After years of working on the basic science of stem cells, Fiona Watt is now on a mission to translate the promise of research into the clinic and help drive the development of new regenerative medicines. 

Last year Watt left Cambridge University, where she was Deputy Director of the Wellcome Trust Centre for Stem Cell Research, to become the inaugural head of the Centre for Stem Cells and Regenerative Medicine at King’s College London.

A belief in collaboration is at the core of what it happening at King’s, and is something Watt speaks passionately about – it is, in her eyes, “a no-brainer”. The vision behind the setting up of the King’s centre – to nucleate stem cell research taking place in various departments at the university and King’s Health Partners – chimes with Watt’s view that collaboration lies at the heart of translating stem cell research into real patient benefit.

Watt is pleased that the Centre for Stem Cells and Regenerative Medicine is located in the same building at Guy’s Hospital as the UK’s Cell Therapy Catapult, and says there are excellent interactions between the Catapult and her Centre. 

The Cell Therapy Catapult is one of a chain of Catapults – modelled on Germany’s Fraunhofer Centres – established within the past two years to overcome barriers to commercialising research. It focuses on translating regenerative medicine into marketed products, in particular, assisting companies to develop manufacturing processes.

In May this year the Catapult signed an agreement with GlaxoSmithKline plc under which it will work with the pharmaceutical company on a range of research projects and technical and regulatory strategies for getting products to market. Before that the Cell Therapy Catapult signed a deal with ReNeuron plc to work on manufacturing technologies and assays to support the development of ReNeuron’s fetal neural stem cell product, which is in clinical development as a treatment for the after-effects of stroke.

Also in May, the Catapult published a survey of all the 71 cell therapy products in preclinical and clinical development in the UK. This showed that most are early-stage programmes run by academics, giving a sense of the potential – but also indicating that far more translational work remains.
For Watt, one of the most exciting aspects of working in the stem cell field is that it is truly multidisciplinary, bringing together biologists, clinicians and researchers across the physical sciences and mathematics, and fostering partnerships between academics and biotech and pharmaceutical companies.

In setting up stem cell Centre, King’s College aims to build on its rich heritage in the field. In August 2003, a group at the university led by Stephen Minger became the first in the UK to derive a human embryonic stem cell line, after being granted a license to carry out the controversial research under ground-breaking legislation agreed in 2002. 

Meanwhile, the science of adult stem cells has also made huge advances, and a whole new branch of stem cell science, induced pluripotent stem cells, has emerged. 

Building collaborations 

In the year or so since joining the Centre, Watt and her colleagues have made headway with a number of collaborations, perhaps most notably the Human Induced Pluripotent Stem Cell Initiative, or HipSci, which has funding of £12.75 million from the research charity the Wellcome Trust and the UK Medical Research Council.

The science of induced pluripotent stem cells makes it possible to take an adult cell, for example, a skin cell, and add a cocktail of DNA that re-programmes the cell back to a pluripotent, stem cell-like state. While the use of DNA to do this re-programming means that at present induced pluripotent stem cells are seen as unsuitable for use in cell therapy, they do provide important tools for studying disease and for drug discovery. The aim of HipSci, which King’s is running in collaboration with the leading genomics researchers at the Sanger Centre in Cambridge and other partners, is to generate induced pluripotent stem cells from healthy volunteers and from patients, and compare and contrast their DNA to determine the genetic basis of various diseases.

On the back of HipSci, Watt is involved in another collaborative project, the Stem Cell Hotel. Clinical collaborators will be invited to work in the ‘hotel’ for periods of time, allowing them to home in on disease mechanisms in great detail. Watt says the people who understand disease best are clinicians. By hosting them in the Centre, she hopes to speed up research, compared to the usual method of working in disciplinary or functional silos.

Barriers to translation

All of this reflects some encouraging progress, but there remain barriers to translation, as highlighted in a report published at the start of July by the UK House of Lords Science and Technology Committee. In particular, there is a need to streamline regulation. A researcher or company trying to develop a regenerative medicine in the UK could have to deal with up to eleven different regulators and ethics bodies, with significant overlap between their functions. 

As specialist advisor to the House of Lords Committee, Watt does not want to comment on the individual recommendations – or indeed, say whether she believes there are any gaps. But, she says, “The report is very timely. It highlights strengths, but also points at the bottlenecks to collaboration.”

While the Committee’s report focuses exclusively on the UK, it is clear that the same problems are replicated elsewhere in Europe. 

Watt believes collaboration is needed on an international scale. In particular she is keen to see a harmonised regulatory pathway for the translation and approval of regenerative medicine products. In her view, things “need to be far more streamlined – at the moment there is huge variation in regulation, and it’s often the case that people don’t realise what the requirements are in different countries.”

While she insists that the different cultural, ethical and religious views across Europe must be respected, Watt says if the promise of regenerative medicine is to be delivered, “It is critical not to stifle excellent research with impenetrable legislation.”

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