Advancing cell and gene therapies: Levelling up life sciences investment in the North-West
Professor Fiona Thistlethwaite and Professor Brian Bigger
The UK in general, and Manchester in particular, has been at the forefront of research into advanced cell and gene therapies for cancer and other diseases. These therapies have the potential to revolutionise outcomes for a wide range of diseases with high levels of morbidity and mortality where standard treatments have proved ineffective. The Government seeks to ‘level up’ the north, and one key aspect of that is expanding its growing specialism in life sciences. But are we investing where we need to and in the facilities that we need to in order to turn this ambition into a reality?
Complex decisions for policy-makers
In the health policy world, we are used to talking about QALYs (Quality-Adjusted Life Years), as the standard to decide whether a medication or intervention is acceptable. A QALY means extending a patient’s life by one year of good health, and the National Institute of Health and Care Excellence (NICE) class a treatment worth the money, if it can add QALYs for £20,000–30,000 or less.
Cell and gene therapies upset these calculations in that they are very expensive, but they do offer a potential cure for some cancers. For patients, whose average prognosis was around five more months of life, advanced cell therapies have led to full remission and recovery. As research into these therapies continues to develop, policy-makers will be faced with increasingly complex decisions about how to compare treatments that extend life (via a number of QALYs) and those that could extend life further or even cure a cancer, but at greater immediate expense.
One way that these costs can be reduced is by shortening supply chains and bringing crucial elements of the production processes that underpin these therapies closer to the research and treatment centres that need them. There have been promising investments made in the north by the Department for Business, Energy and Industrial Strategy (BEIS) and the Medical Research Council to advance this agenda. These include the creation of new ‘bioproduction’ and research facilities in Barnsley (cell production), Sheffield (virus production) and the north-east (Darlington – not a production facility but engaged in optimisation work on existing products).
For patients, whose average prognosis was around five more months of life, advanced cell therapies have led to full remission and recovery.
Bioproduction: The role of manufacturing
The way that some advanced cell therapies, such as ‘CAR-T’ and ‘TIL’ immunotherapies, work is that cells are taken from a patient’s body, we replicate those cells (in a bioproduction facility) and they may then be genetically modified using a virus. This virus enables the cell to produce modified proteins that can help the cell to detect and destroy cancer cells or to treat rare genetic diseases. This requires two different manufacturing processes. We need to be able to make the virus, and also to produce the human cells that the virus is inserted into.
There is a distinct lack of this manufacturing capacity in the north of England. We have one facility planned in Sheffield for virus production, and a cell production facility in Barnsley but nowhere nearby for the insertion of the virus into cells, which is its own distinct and specialised process.
Leading the world: 1% of the population, 12% of the trials
Here in Manchester, the Advanced Therapy Treatment Centre (known as iMATCH) has been working to scale up activity in both cell and gene therapy since 2018. Our work at the centre includes looking at the training needs for staff, challenges in setting up advanced cell therapy trials, and how we can translate research insights into treatments for patients. The Advanced Immunotherapy and Cell Therapy team at The Christie currently has twelve open trials, which is the largest number of solid tumour cell therapy trials taking place in a single centre in the UK.
Despite being leaders in pioneering these treatments, in Manchester and across the UK, we face the considerable expense and logistical challenge of having to purchase and often import the cells we need to work with from other countries. With less than one per cent of the world’s population, the UK is currently conducting 12% of global gene therapy trials. This is a distinctive and valuable edge for the UK’s life sciences sector, but one where we are at significant risk of falling behind unless more investment in domestic bioproduction facilities follows.
Here in Manchester, the Advanced Therapy Treatment Centre (known as iMATCH) has been working to scale up activity in both cell and gene therapy since 2018.
‘Levelling up’ through life sciences investment?
With the Government’s commitment to ‘levelling up’ the UK’s regions, and the prioritisation of the life sciences sector as part of its wider Industrial Strategy, now is the time to push for investment in new bioproduction facilities in the north of England. We welcome the investment that led to the virus production facility in Sheffield, but viruses are only one of the two necessary elements for advanced cell therapies. Cell therapy requires both virus production and production capacity for the cells that deliver the virus into the body, making a cell production facility in the north the logical next step to build on that investment.
There are a number of reasons that Greater Manchester in particular stands out as a strong candidate for the location of such a facility:
- A large population of students with experience in biomedical disciplines. This is a valuable skills base that can form a natural talent pipeline for the specialist jobs that a bioproduction plant would create;
- Greater Manchester is well placed for transport, with established links to London and across the Northern Powerhouse region via the ‘M62 Corridor’;
- The University of Manchester is home to the central hub of the national Royce Institute for Advanced Materials. Materials being produced by the Royce, such as hollow-fibre materials for advanced filtration, are already bringing down market prices for the advanced materials needed for this work, and their proximity will allow for greater collaboration and development of new materials in future;
- Manchester is an advanced therapy training centre with several MSc and PhD level programmes in the biotechnology sector, including iMATCH, and already offers specialised hands-on training for the specialised workforce required.
Regardless of its exact location, a cell production facility in the north would create highly skilled jobs, enhance our national research capacity, shorten existing supply chains, and add to the existing life sciences industrial specialisation across Cheshire, Greater Manchester and beyond.
The freedom to set our own regulatory environment for life sciences research in the UK since Brexit is a key strand of government thinking on the future of this sector in the UK economy. To attract more international investment in drug development and human trials, we need to build our own cell production infrastructure, rather than relying on importing the materials we need from the US and other countries.
The US provides one potential model for the UK government to follow. In California, the California Stem Cell Initiative at UCLA was pump-primed with significant federal investment. What followed was the organic development of a life sciences industry cluster, with pharmaceutical companies and research organisations opening laboratories and offices close by. With the Government commitment and industry partnerships underpinned by the post-Brexit Life Sciences Sector Deal, the UCLA example could be replicated here in northern England.
To attract more international investment in drug development and human trials, we need to build our own cell production infrastructure.
The Office for Life Sciences (a government body ran jointly between BEIS and the Department for Health and Social Care) is best placed to catalyse government engagement with the strategic economic and health benefits that a new bioproduction facility will deliver. We encourage the Office for Life Sciences to convene a working group with representatives from government, industry, research and clinical practice to examine the California/UCLA precedent and its potential application to a new bioproduction facility in the north-west.
With leadership from both national and cityregional government, the next expansion of the northwest’s significant life sciences sector will underwrite new breakthroughs in advanced research, new treatments for many deadly cancers, new high-skill/high-wage jobs, and secure UK leadership in international life sciences investment and exports. The benefits are simply too great for this decision to be delayed or postponed any longer.
About the authors
Professor Fiona Thistlethwaite
Fiona Thistlethwaite is a Medical Oncology Consultant within the Experimental Cancer Medicine Team (ECMT), Advanced Immunotherapy and Cell Therapy (AICT) Team Clinical Lead, Honorary Professor at The University of Manchester, and Director of iMATCH (Innovate Manchester Advanced Therapy Centre Hub). She has an active clinical trials research portfolio in early phase clinical trials (adult solid tumours) within the ECMT and AICT.
Professor Brian Bigger
Brian Bigger is Professor of Cell and Gene Therapy in the Division of Cell Matrix Biology and Regenerative Medicine at The University of Manchester. His Stem Cell and Neurotherapies lab works on the development and delivery of clinical cell and gene therapies for rare genetic diseases and brain tumours. He is also Chairman of the European Study Group on Lysosomal Diseases (ESGLD).