Why Scotland is well-positioned to lead in advanced therapies for complex diseases
The global health burden of complex diseases such as severe lung, liver, and vascular disease is huge, and growing by the second. Yet, treatments, where they exist, are often invasive or unsatisfactory.
As society changes and the population ages, standard therapies will be successful even less often because patients are more likely to have multiple health issues and thus be taking multiple medicines that can have adverse interactions with each other.
Older patients are also more likely to have chronic conditions that are ongoing over a long period (including cardiovascular disease and dementia).
Advanced therapies, which involve regeneration and repair from cell to organ, offer great hope, but also face obstacles in development, not least the early expense and risk.
Scotland: Well-placed to bring advanced therapies to patients
Here in Scotland, right now, we have a number of elements in place which position us well to reduce some of the risk and make it possible for advanced therapies to take that essential step forward towards patient care.
Firstly, we have excellent biological research, carried out in a data-driven, interdisciplinary way, supported by cutting-edge facilities. At the University of Edinburgh, Professor Susan Rosser is engineering biology to understand the underlying molecular mechanism of a disease and its progression; to model diseases and screen for toxicity during drug development, and engineer cell lines to produce biologic drugs. She uses Europe’s only Berkeley Lights Beacon System in an academic setting, at the Edinburgh Genome Foundry, and CRISPR-Cas 9 gene editing technologies.
Meanwhile, FUJIFILM Diosynth Biotechnologies chose Edinburgh to build the Centre of Excellence in Bioprocessing 2.0 to manufacture biologics, because of our history and proficiency in using Chinese hamster ovary cells to produce proteins.
Edinburgh: Expertise in cutting-edge technology
Data, artificial intelligence (AI), and machine learning (ML) are being harnessed by Edinburgh neuroscientists, Eisai, Gates Ventures, medical research charity LifeArc, and Health Data Research UK in the NEURii partnership for the prediction, prevention, management, and treatment of dementia.
Stem cell diabetologist Professor Shareen Forbes leads a group that aims to deliver cell therapies to people with Type 1 diabetes, in order to manage and reduce dependence on insulin administration.
And Professor Stuart Forbes has recently had some extremely encouraging phase 2 clinical trial results using macrophage immune cells to treat liver cirrhosis, supported by his spinout company Resolution Therapeutics. Since cloning Dolly the Sheep at the Roslin Institute in 2006, Edinburgh scientists have been reprogramming cells, leading to current expertise in iPSCs (induced pluripotent stem cells) and in the creation of new cell therapies.
Edinburgh is a leader in single cell RNA sequencing - a cutting-edge technology which allows us to measure gene expression levels across thousands of individual cells in human samples.
An NHS relationship, positioning for success
Access to these samples is made possible by our well-governed relationship with the National Health Service – another key element that positions us for success. The University of Edinburgh and NHS Lothian co-design research projects, for example, and that gives us ethically approved and fully consented access to spare human tissue, which means that we can identify human mechanisms of disease and not solely rely on other routes for generating new therapies.
The NHS relationship also gives us access to patients for research in investigational clinical trials, and in Scotland, unfortunately, we have a large patient pool for these complex diseases associated with lifestyle factors.
Unlike health systems elsewhere, the Scottish National Health Service is a single provider, so it keeps and can share clinical metadata from all its health boards. This helps us build complex data sets that aid understanding of disease, down to the level of individual patient characteristics, and, along with an interdisciplinary approach, allow us to underpin clinical research with bioinformatics and mathematical prediction models. Sharing data safely also helps us assess the effectiveness of different therapies, and facilitates collaboration.
The importance of collaboration
Collaboration is supported by physical co-location and at Edinburgh’s Bioquarter, a teaching hospital, Edinburgh’s Royal Infirmary, labs, companies and the NHS share space, facilitating research and innovation at all levels. The Institute of Regeneration and Repair is a new home to over 500 scientists and clinicians studying tissue regeneration and repair, for example.
Roslin CT, which spun out of the Roslin Institute, is based here, also, and partners with pharma and biotech companies from development phase through to late-stage clinical manufacturing of advanced therapies.
We can ensure Good Manufacturing Processes (gmps) from storage to testing, partly through our relationship with the Scottish National Blood Transfusion Service, which ensures research projects featuring organ transplantation have the potential to scale up.
And this is partly why the UK Cell and Gene Therapy Catapult, which facilitates technological innovation in healthcare, recently opened at the Edinburgh Bioquarter.
Elsewhere in Scotland, we have numerous collaborative elements, including the Industrial Biotechnology Innovation Centre and the Glasgow Centre for Precision Medicine, focussing on translation of research into new products and services for a global market.
And in the University of Edinburgh’s commercialisation service, Edinburgh Innovations, we have a team of experts skilled at supporting and accompanying the journey of research from bench to clinic.
Complex diseases are a wicked problem that can only be solved when a number of elements come together at the same time. Advanced therapies require a large investment, especially in the early stages when success is not guaranteed.
But in Scotland, the above features – data-driven, excellent health research; co-location and collaboration; and our relationship, including stringently-governed data-sharing, with the NHS – de-risk the process of developing the desperately needed therapies that can help us meet the compound health challenges of today.
