Josephine Moulds reports in The Times 20th August 2014: Stem cell therapy will cure cancer and make blind see (with apologies to The Times for reproducing the “good news” in full)
A revolution in British healthcare can be accelerated if class-leading research gets the right funding
On the twelfth floor of Guy’s Hospital, next to the Shard, is a gleaming laboratory with panoramic views of London. To the untrained eye, it looks rather like a room full of photocopiers, microwaves and washing machines — but the equipment in here sets pulses racing for the scientists who come to stir, spin, modify, test and manufacture cells within its spotless glass walls.
This is the heart of the cell therapy string of the government’s Catapult programme, which invests in areas of leading academic research, in this case designed to drive the growth of a revolutionary form of treatment that proponents say will cure cancer and make the blind see.
Chris Denning, professor of stem cell biology at the University of Nottingham, says: “If we can achieve even a fraction of what we hope with stem cells, then it will be a revolution for healthcare. It will give us a whole new set of tools to treat diseases like cancer and repair damaged organs, such as the eye, brain or heart.”
British universities are recognised worldwide for producing some of the best research in this field, but the race is becoming more crowded. “China is an emerging area,” Professor Denning says. “It is snapping at people’s heels. [The UK is] among the best in the world, there is no question, but there is no space to rest on our laurels. We need to keep at the forefront, otherwise we will get overtaken.”
There are fears that Britain is failing to translate its research into moneymaking businesses. Michael Hunt, the chief executive of ReNeuron, one of the few cell therapy companies based in Britain (see box), says: “The UK has a strong presence and history of stem cell research and cell biology research. At the moment, it has a bit of catch-up to do to translate that research into therapeutic candidates that can be taken through clinical development to the market. We [at ReNeuron] are very, very alone as a pure cell therapy business that has the aim of doing just that.”
The problem is that academics have little incentive to undertake the daunting task of translating an idea that works well on paper into a therapy that can be administered to a patient.
Andrew Baker, president of the British Society for Gene and Cell Therapy, says: “Scientists are judged on the quality of their papers; that is often linked to fundamental research findings.
“You don’t get major brownie points or publications if you translate the finding. From a tactical point of view, scientists think: ‘Why am I going to spend five years doing this trial?’ ”
This is where the Catapult programme comes in. Keith Thompson, chief executive of the cell therapy Catapult, says: “Sometimes it’s seen as the boring slog of science, turning the idea into something that you can make routinely. But unless you do this stuff, a) you’ll never get the therapy to the clinic and b) you’ll never keep it for the UK.”
One of the biggest hurdles facing fledgeling cell therapy companies is the lack of precedent. Even the relatively simple task of developing a business model is a lot more complicated when nobody has done it before.
Mr Thompson says: “Health economics are basically built up around interventions from a drug that often will have some sort of lifelong therapy, whereas a stem cell-based cure might give a lifelong cure.
“How do you cope with something like that? If you cure somebody from blindness, they are going to have a lifelong benefit from that. How do you price that? And how do you pay for it? You may have to pay for it over a number of years.”
Delivering these therapies is far more complex than shipping containers of tablets around the world. Many cell therapies are developed from the patient’s own cells, such as the replacement windpipes developed by Videregen (see box).
Mr Thompson says: “If you’ve got a trachea, it’s got a very, very short shelf life. How do you get the cells from the patient, grow the new trachea and get it back to the patient? You are talking very short supply-chain delivery times — 24 hours is not uncommon. That brings in a whole range of complications, integrating manufacturing with clinical use.”
The lab at Guy’s can be used by SMEs and even large companies to manufacture cells. The equipment is of the highest specification, but the facility also has lower-spec machines, so that companies are able to replicate the processes in their own facilities. Mr Thompson has ambitions to provide an even fuller service to cell therapy companies that wish to come to the UK. The Catapult has put a bid in to government to fund a 5,000 sq m manufacturing hotel, where companies could lease space for final, large-scale clinical trials and initial market supply.
“As the thing takes off, we would then expect the company to build its own facility and take its people with it,” he says. “We are designing the facility in a way that will be highly replicable. The expectation is that the company would want to do it relatively near by, so that the staff it had would move with it.”
This is the beauty of pouring money into cell therapy at an early stage. Small businesses attracted to the UK by the facilities on offer are likely to stay put as they grow, because of the regulatory headaches they would face if they tried to move abroad.
Mr Thompson says: “Because these things are complex to make and there is a lot of knowhow in it, and they are regulated, it means that once you build these places and therapies get licensed in them, they don’t move very easily. So it’s really important to have a strategy which embeds these things into the UK very early on.”
Every big pharmaceuticals company has monoclonal antibodies in their portfolio. They generate revenues of $45 billion a year and the market is growing at 5 per cent to 7 per cent a year.
“These things are a roaring success,” Mr Thompson says. “The trouble is that so much of the manufacturing of that and exploitation has gone offshore. One of my aims, by putting down manufacturing and supply chain routes, is to keep the long-term value as much as possible in the UK.”
Companies that develop intellectual property with the help of the Catapult must then negotiate over how that IP is meted out. Mr Thompson says that a company might want to keep the IP specific to the disease it is seeking to cure, while the Catapult will try to hold on to generic IP.
“If we managed to get some IP around a scale-up system that reduced the cost of goods for everybody, we would want to try and disseminate that across the board non-exclusively.” The Catapult could use IP to generate revenues, which would help to support the centre, but its primary concern would be to make sure that it is used.
It is able to have this open approach to IP because of relatively secure government funding. The coalition has committed to provide the Catapult with £10 million of funding every year for the first five years of its existence, and it is believed that that will be renewed regardless of the general election result, thanks to cross-party support. The Catapult, in turn, is expected to find an additional £20 million a year from private investors and grants from the European Union and others.
Mr Thompson says that continuing government funding is important to ensure the longevity of the project. “I’ve been around in biotech for a long time. I’ve seen institutes start up with a bit of seed money and, frankly, they often lose focus on what the mission is and focus on trying to bring in the money.”
Steve Bloor, the chief executive of Videregen, won a £1.9 million grant from the government’s Technology Strategy Board and then raised £1.3 million from private investors. He says: “It is very difficult to get large amounts of investment capital or equity capital for these types of contracts, in contrast to the US, where there is much more freely available capital. We are in danger of losing out to places like the US and Korea, Japan and China. The money is over there.”
The Catapult is seen as crucial to attract academics, companies and investors to the UK to build a vibrant cell therapy industry. Professor Denning says: “The US has put vast amounts of money into this area, so has China. This is more a question of if we don’t do this we’ll end up buying back the technology from other countries in the future. Unless we do this, we won’t keep up.”
$12.2 billion estimated size of global market for stem cell technologies by 2018
£9 million spent by the cell therapy catapult on its lab at Guy’s Hospital, London
60,000 stem cell transplants performed each year on oncology and blood-based disorders
$900 million revenue generated by cell therapy products distributed by biotherapy companies in 2012
2,500 ongoing regenerative medicine clinical trials around the world
15 per cent trials sponsored by industry