Scotland first grabbed headlines in the stem cell world fifteen years ago with the cloning of Dolly the sheep. But Scotland’s stem cell successes didn’t end there. In 2003, scientific highlanders at the University of Edinburgh discovered Nanog, a critical pluripotency gene expressed in embryonic stem cells. And last year, doctors at Glasgow’s Southern General Hospital treated a patient in the first-ever regulated human trial for a stem cell stroke treatment.
Yet stem cell medicine in Scotland has also faced some quagmires in the moors. Five years ago, for example, embryonic stem cell pioneer Austin Smith moved to the University of Cambridge; then, the biotech company Stem Cell Sciences followed suit. But with the development of potential new stem cell therapies for multiple sclerosis and the prospect of a stem cell-based approach to make blood for transfusions, scientists in Scotland are starting to turn things around. And those efforts will be emboldened by the Scottish Centre for Regenerative Medicine, a new ₤59 million ($97 million) research and commercialization facility in Edinburgh slated to open this summer.
Earlier this year, organizers announced that Charles ffrench-Constant, a multiple sclerosis researcher at the University of Edinburgh, would replace Dolly cloner Sir Ian Wilmut as the center’s director. Ahead of the building’s launch, Nature Medicine spoke to ffrench-Constant to learn how he plans to advance stem cell medicine in the land of lochs and glens.
What milestones do you hope to meet at the new center over the next five to ten years?
I would like us to have dramatically improved our understanding of basic stem cell science, and use this knowledge to be able to take human embryonic stem cells or human induced pluripotent stem cells and convert them into very large numbers of specific cell types in the brain, liver or in the blood. We would also like to be able to do the same in skin and heart and then use those [cells] either for disease modeling purposes, drug discovery programs or put them directly into the patient. I would like to be able to tell you that these goals weren’t just feasible but also something that is practical.
How do you hope to meet those goals?
To achieve them, we need to bring together three different groups of scientists. We need the basic stem cell biologists who are trying to understand pluripotency and stem cell differentiation. We need translational scientists who are working on issues like scale up and protocol optimization. And finally we need the clinicians who are actually going to deliver these cells or therapies. Here in this center, we have brought all these groups together into a single building.
What else can be found in the new center?
We have a GMP [good manufacturing practice] facility in our new building that will be run jointly by a not-for-profit company called Roslin Cells and the Scottish National Blood Transfusion Service. They will provide us with the key ability to make sure that protocols developed in the research laboratory can actually be optimized and scaled up for use in the clinic.
So, is the idea to develop a one-stop shop to bring therapies from the bench to the clinic?
That’s correct. We certainly expect to have the capabilities within the building to generate enough cells for phase 1 or phase 2 clinical trials — ten to 20 patients.
What are the current challenges of developing stem-cell based medicines in the UK?
I think the challenges in the UK are the same as everywhere else: the difficulties of taking a discovery from the laboratory bench to the clinic. But we are helped enormously in the UK by the amount of thought that has been given to this process both by the government and the Medical Research Council. They have established a stem cell bank, for example. And, the legislative and regulatory bodies that we have here in the UK have thought long and hard about the hurdles that need to be addressed in order to accelerate these trials into the clinic, such that a number are moving forward.
What do you think the UK needs to do to be successful in regenerative medicine?
I would like to see a stronger small biotech and venture capital base that is trying to invest in this area. There are other companies in Europe and in the US that have embraced stem cell medicine more enthusiastically.
What is Scotland doing to turn this situation around?
The Scottish Centre for Regenerative Medicine is on an area of land called the BioQuarter. The vision is basically to have the academics working alongside smaller and larger companies in a site right next to a hospital with clinical research and imaging facilities. To have on a single site everything from a huge teaching hospital right through to cutting edge GMP facilities is certainly very unusual if not unique, and I think it is a huge opportunity. There is already a building going up on the BioQuarter site which will provide incubator space for spinout companies from the university.
Do you think all these efforts will put Scotland at the forefront of stem-cell therapies?
I’m confident that it will.
Image of Charles ffrench-Constant by Matt Davis for the MRC Centre for Regenerative Medicine