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This morning’s announcement that the Novartis drug Kymriah will be funded by the NHS is excellent news for a particular group of UK patients. The treatment is a type of CAR-T (chimeric antigen receptor T-cell) therapy and has the potential to dramatically change outcomes for children and adolescents with relapsed/refractory acute lymphoblastic leukaemia (r/r ALL). Currently these patients have only a 30% chance of 5-year survival and although Kymriah is too recent a drug to have the final figures, the early outcomes look excellent with 83% of patients responding in the short term.

This news follows on the recent approval of Kymriah by the EC and last year’s FDA authorisation. It’s been particularly surprising to see how quickly these approvals have been granted – the first clinical trial was started in 2012, and this really is a testament to how companies, academic medical centres and regulators have worked together to bring a safe and effective new treatment onto the market in a timely manner. The success of the treatment for the very first patient, Emily Whitehead has been replicated with a significant cohort of patients, and it is great to hear that UK patients will be able to join them.

One of the big challenges of CAR-T is the complexity of manufacturing the product. In a multistage process, T-cells (a type of white blood cell) are taken from the patient, genetically manipulated to express a new receptor that recognises tumour cells, and then reinfused. At the moment the process is largely done in a cleanroom with significant amounts of manual operator involvement. This is just about manageable for paediatric r/r ALL, where in the UK there are around 15 cases per year, but as CAR-T broadens into other types of cancers, such as non-Hodgkin’s lymphoma (such as Yescarta from Gilead) and solid tumours, the numbers of patients mean that this manual process won’t be able to cope with demand. As a result, companies developing cell therapies are having to look at how the process can be closed (meaning they could be made outside of the expensive cleanroom environment), automated and most importantly made cheaper, in order to bring the very high price of the treatment down to a level which is more acceptable to payers. Another interesting challenge is deciding on where to manufacture the product – in a central lab, which causes lots of logistical difficulties in moving living cells across countries or even continents, or locally in hospitals which in turn has issues around staff training and ensuring comparability from site to site and maintaining quality across the distributed manufacturing network.

Cambridge Consultants has been working with a number of leaders in the field, using our long track record in developing and introducing automation, in-process controls, analytics and novel sensing and measuring techniques to see how the cell therapy manufacturing process can be made to work at scale. It’s been eye-opening how many of the areas that need to be improved are analogous to projects we’ve done in other sectors – from the process engineering of single-batch personalised medical implants, through to high-throughput rapid cell characterisation in diagnostic instruments and the sterility and in-process controls in food and drink production. We’re excited about this rapidly evolving field, and looking forward to playing our part in bringing CAR-T and other cell therapies down to a price point that means that these highly effective products will be available to a wider group of patients in desperate need of better treatments.

Author
James Hallinan
Business Development Manager, Synthetic Biology

James is a business developer at Cambridge Consultants and specialises in synthetic biology and early-stage technology commercialisation across life sciences and healthcare