From artisan to industrial: getting stem cells ready for the clinic
The potential to use stem cells as therapies is one of the most exciting areas of biomedical research. But how do we turn the promise of the lab into reality in the clinic? Prof Peter Andrews, director of a new regenerative medicine research ‘hub’ announced today, explains what lies ahead.
The idea of replacing worn out or diseased tissue with new healthy cells grown from stem cells is an attractive one.
And it’s certainly an idea that captures the imagination. Although we’re never going to transplant brains, the announcement last month that researchers had grown tiny brain-like ‘organoids’ in the lab gained a huge amount of press attention.
But how close are we to a point where having a cell transplant is the same as any other treatment? It’s fair to say that we’re a long way off. I don’t expect cell therapies to be commonplace for at least another 20 years, and probably a lot longer.
But one thing I can see more clearly is what we need to do to get to that point — and it’s a lot of careful, painstaking work in the lab.
Today our new research hub — a collaboration between my institution, the University of Sheffield; the University of Cambridge; Loughborough University; the National Institute for Biological Standards and Control (NIBSC); the Wellcome Trust Sanger Institute; and the Babraham Institute — has been announced.
We’re joining forces to focus on some of the important obstacles we need to overcome so that we can turn growing stem cells from the work of a craftsman in the lab to a near-automated industrial process.
At the moment it’s relatively easy to grow a small group of stem cells in the lab and get them do what you want them to: become a particular type of brain or eye cell, for example. But being able to do this reliably, 100 per cent of the time, in lots of different cell ‘factories’ and to standards that regulators will approve, is a different matter.
A crucial aspect here is ‘differentiation’. Stem cells, whether they are embryonic stem cells taken from early embryos or reprogrammed adult cells (induced pluripotent stem cells), can become almost any type of cell. They take cues from the environment they’re growing in to develop into one cell type or another, and researchers at the University of Cambridge will be looking at the exact combinations of cues needed to grow particular types of cell reliably.
Another aspect is working out the processes involved in producing millions of cells to treat potentially hundreds of thousands of patients. The University of Loughborough will be using their expertise in manufacturing to investigate ways of producing large amounts of cells to high standards.
We also need to know that the cells that are produced are safe. For example, a team from the NIBSC will develop ways to check that the cells do not acquire infectious agents that might compromise safety. Also, we know that embryonic stem cells can gain extra chromosomes, or changes in their DNA, when they are grown in the laboratory. We’re working at the University of Sheffield to find out whether these changes affect the chances of them becoming successful treatments — will they cause cancer, or be difficult to grow into the type of cell we want? Do they have particular genetic changes that are associated with disease? Our partners at the Wellcome Trust Sanger Institute and at the Babraham Institute will be contributing their specialist expertise to these studies.
Overarching all this is making sure we involve pharmaceutical and biotechnology companies. Producing stem cell therapies is vastly different to making pills or vaccines, and the business model is one that companies haven’t encountered before. We’ll be talking to companies about how we can make commercially viable products.
Without such companies, we’re unlikely to have cell therapies, at least on the scale needed for transplants to be just as commonplace as popping a pill.
The £4.5m hub is part of the UK Regenerative Medicine Platform (UKRMP), funded by the Biotechnology and Biological Sciences Research Council (BBSRC), Engineering and Physical Sciences Research Council (EPSRC) and the MRC. Also announced today was a further £20m of capital funding from the MRC to provide state-of-the-art facilities and equipment to support the work of the UKRMP and the wider regenerative medicine research community.