The future of CAR T cell immunotherapy in the UK

Image:Oral squamous cancer cell (white) being attacked by two cytotoxic T cells (red). Source: NIH image gallery on Flickr. Licensed under a CC BY NC 2.0 license. 

Cancer therapy is at the edge of a revolution.

A dramatic statement, and one many will have heard before… but the one-shot ‘cures’ delivered by CAR T cells – a completely new kind of cancer treatment – in early clinical trials were unprecedented and sent shock waves through the healthcare sector.

CAR T cell therapy involves taking out a person’s own immune cells, ‘training’ them to spot cancer cells by altering their DNA, and then putting them back into the patient to seek out and destroy tumours.

Much as it sounds, the technique is highly skilled, personalised, requires hospital admission and a whole different kind of infrastructure… all reflected in its hefty price tag.

Despite the challenges, NHS England found a way to facilitate and pay for the treatment via the Cancer Drugs Fund for the small number of children and young adults with acute lymphoblastic leukaemia (ALL) for whom chemotherapy doesn’t work.

But as we begin to see the output from hundreds of millions of dollars invested in CAR T cell therapy by the pharmaceutical and biotech industries, what needs to happen to ensure our cash-strapped, resource-limited NHS can deliver this revolution in healthcare to the ever-increasing numbers of cancer patients in the UK?

This question was discussed at length at an event run jointly by the Financial Times and Gilead Sciences. Here’s my take on some of the solutions prosed by the panellists.*

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Introducing infrastructure

NHS England set up three centres of excellence for CAR T cell therapy – in Newcastle, Manchester and London – that have the infrastructure and expertise required to deliver CAR T cell therapy.

The initial cost of this is high, so it will be crucial to make sure the infrastructure is future-proof and set up to delivery similar new types of treatment that are emerging such as gene therapies for other chronic disease.

In future, the expertise will have to be expanded so there are additional centres able to deliver the treatment to facilitate increasing demand, and it’s important that hospitals are ready to embrace these new kinds of treatments.

Picking out the patients most likely to benefit

As with all cancer treatments, if you can use a test to identify which patients the treatment is likely to work for, you can save the NHS a lot of money. Although most patients who have received CAR T cell therapy responded well, not everyone did and some experienced severe, unmanageable side effects.

It makes a huge difference to the impact of the drug if it’s only given to those likely to respond, so it should be in the manufacturer’s interest to produce companion tests for their CAR T cell product.

More data required

It’s still very early days for CAR T cell therapy. Although there have been some apparent one-shot cures, small numbers of patients have received the treatment so far and these patients have received it only recently – so long-term effectiveness and late side effects haven’t been properly assessed.

More data will allow regulators to assess how beneficial it is to patients, and if costs can be saved elsewhere.

Changing regulatory processes

In 2019, the NHS regulatory body the National Institute of Health and Care Excellence (known as NICE) will review its processes for approving new treatments for effectiveness and affordability. The Institute of Cancer Research has been heavily involved in these reviews in the past and has proposed new ways for NICE to bring innovative cancer medicines into the NHS.

One of the ICR’s suggestions is that we need a system that provides patients with stable access to cancer drugs over the long term - and this requires radical reform of how NICE reviews drugs and rewards innovation.

The current CAR T cell approval makes them available through the Cancer Drugs Fund – a source of funding specifically for cancer drugs in England and Wales.

It's likely that there isn't enough in the pot for all of the future cancer therapies coming through – particularly with the high volume of CAR T cell therapies in development, which require high costs upfront – so it’s important the NICE review takes this into account.

There are also hidden costs in many cancer treatments, such as the increased risk of getting a second cancer from chemo and radiotherapy, or complications from surgery. These treatments may not be necessary if one-shot CAR T cell therapy is successful.

We also need to make sure that the new system takes into account future healthcare benefits from CAR T cells. This is tricky without long-term data, but waiting 20 years to gather the data would mean patients miss out.

Increasing political will

It can take a long time for developments in the lab to make it through to patients.

Panellists at the event called upon government and the British public to get behind new developments in life sciences, such as CAR T cells, and highlighted the role the academic, charity and industry sectors could play in communicating this message.

We will need political incentive to push innovative treatments forward because, ultimately, healthcare advances will need more government spending.

What about patients?

People diagnosed with cancer want hope and options. They want information about new treatments – what is it, how does it work, is it safe, what are the side effects, even what does it cost the NHS? Currently, there isn’t enough information about CAR T cells out there.

At the end of the day, if you’ve been diagnosed with cancer your main focus is how to get your hands on something that’s going to make you better. Delays accessing new treatments are frustrating for patients, so there is a strong will to make innovative new treatments, such as CAR T cells, available on the NHS.

The future looks bright

This isn’t the first medical revolution the NHS has encountered. Around 70 years ago when the NHS came into being, innovative procedures such as IVF, keyhole surgery and organ transplantation didn’t exist. Now, the NHS carries out 5,000 transplants a year.

Money is tight; resources are scarce. But with the potential of a one-shot cure with possible long-term benefits, these new cell therapies have to be embraced. It will take a huge effort, but the potential is huge. We owe it to patients to give it our best shot.

*Panellists at FT/Gilead Sciences event on The Future of CAR T Cell Therapy were: Dr Claire Roddie, Consultant Haematologist and Senior Lecturer at University College London; Matthew Durdy, Chief Business Officer, Cell and Gene Therapy Catapult; Anne Marie Morris MP, Chair of the APPG on Access to Medicines and Medical Devices; Steven Scowcroft, Head of Business Development at Lymphoma Action; Steve Bates, CEO of BioIndustry Association.

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