Precision paediatrics: Treating patients with CAR-T

Guest Blogger|9th July 2019

Dr Stuart Adams is among the scientists leading the quest to develop precision medicines for seriously ill paediatric patients. This includes working with an expert team at the Great Ormond Street Institute of Child Health to help embed precision medicine throughout the organisation and ensuring there is an integrated and multidisciplinaryapproach to treating childhood disease.

As Joint Lead Healthcare Scientist for Great Ormond Street Hospital (GOSH) Children National Health Service (NHS) Foundation Trust, and Principal Clinical Scientist in the Department of Haematology, Stuart is at the helm of this movement. He is part of a team using a groundbreaking chimeric antigen receptor T-cell (CAR-T) therapy, which is transforming outcomes for children who would otherwise have no treatment options left.

The therapy works by modifying T-cells to weaponise them against cancer cells. These cells play a central role in the immune system response. It’s cutting edge science and recently gained media attention due to both the impressive clinical trial results and the high cost of the treatment.

Crucially, the future success of CAR-T therapy will depend on conducting multidisciplinary tests to identify risk factors and determine the right therapy for a patient as early in the treatment pathway as possible. Here, we discuss the story behind CAR-T therapy and the steps being taken to ensure more patients can access it.

Can you outline your own work around CAR-T?

SA: CAR-T cell therapy is an innovative immunotherapy that modifies the body’s own immune T-cells to start attacking cancer cells. Immunotherapy has been around for many years, but this is the first time that the patients’ own T-cells have been taken out, genetically modified and put back in to target cancer cells.

There are a number of trials being undertaken throughout the world, but the first licensed product, Novartis’ Kymriah, is now available for the treatment of leukaemia and is live at GOSH. It’s working well, but it’s not perfect. Some patients have responded wonderfully but others unfortunately do relapse.

It’s expensive, and patient selection must improve so that each person receiving treatment benefits. You don’t want to put a child through a £250,000 therapy that is ultimately going to do them no good.

What are your goals for the treatment?

SA: The real goal is that when a patient has the diagnostic bone marrow taken, we can say whether this child will relapse or not following CAR-T therapy. The only way we’re going to get that kind of data is through a large retrospective analysis. As scientists, we’re using the data to work out which patient is most suitable for which treatment. Once you know if a patient profile does or doesn’t respond well to something, you can drill down into why and pick out the best treatment. It’s an exciting time for CAR-T therapy.

How does the current structure at GOSH help to bring cutting-edge treatment like CAR-T into play?

SA: It’s an integrated, holistic approach. One brilliant thing about Great Ormond Street is the integrated lab working, where lots of disciplines work together, so we’re able to really tailor the treatment approach for each patient.

In other places where tests happen and restructures have occurred, there is often a lack of communication between researchers and physicians. If those groups would have communicated, patients would have benefitted. That siloed approach is about as far removed from precision medicine as you can get because it’s like one big factory – it’s one size fits all for every patient.

There is clear intent at NHS England that personalised medicine is the way forward, and the realisation that researchers and physicians must be brought together to facilitate it. It’s tough, but at places like GOSH it works beautifully. It means that even if a patient belongs to a discipline that might be outside the team’s normal scope, they can still work with them. This approach can make targeted therapies like CAR-T a reality.

A good example at GOSH is in bone marrow transplants. Patients come from multiple disciplines – haematology, metabolic, gastro, immunology – then they are assessed in multidisciplinary

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