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Brain tumours kill more children and adults under the age of 40 than any other cancer

Bridging the gap: from basic brain tumour research to clinical trials

by Katie Sheen, Research Manager

There is always a buzz at this vibrant conference, and the Society of British Neurological Surgeons (SBNS) Spring Meeting 2019 in Manchester certainly lived up to its excellent reputation. There was new neurosurgical technology on display at stands all around the exhibition hall and a continuous stream of research presentations in multiple lecture theatres. So, what are the emerging themes of most relevance to patients and their families?

The keynote Sir Victor Horsley Lecture was delivered by Professor Evanthia Galanis, a dynamic research lead from the Mayo Clinic, US. She talked about drug development for brain tumours and how to bridge the gap from the essential initial laboratory research to clinical trials in patients, and how molecular profiling is changing the way clinical trials are being designed.

Whilst everyone wants to get promising new treatments quickly from the laboratory to clinical trials, Professor Galanis emphasised the importance of gaining strong pre-clinical evidence to ensure that only the most promising treatments are taken forwards.

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It is also crucial that time, money and resources are focused on clinical trials that only recruit patients who fulfil the molecular profile likely to benefit from a specific drug.

The expanding field of molecular characterisation of brain tumours provides new levels of understanding as to why drugs may only work for particular subgroups of patients. For example, a drug might previously have been trialled in a large number of patients with a particular type of tumour, but only have worked for some of them. This could make the drug look unsuccessful, but if you can identify the molecular characteristics of the tumours that did respond positively and isolate that patient group for future treatment with that drug, suddenly your results might look much better and open the way to new combinations of drugs for that group.

So it makes sense that researchers across the globe, including those in our UK Research Centres of Excellence, are now working to identify the multiple mutations in different types of brain tumours, then identifying drugs that can influence them, and only then opening clinical trials that are tightly limited to those patients most likely to respond.

Whilst this raises the challenge of potentially small patient numbers when ideally you need a large group in order to clearly identify benefit, it does increase the likelihood of success, and collaboration amongst multiple treatment sites can overcome the challenge of recruiting enough patients to make the trial results reliable. Such detailed molecular knowledge simply wasn’t available until relatively recently and therefore this level of trial design simply wasn’t possible.

There are over 120 different types of brain tumour, so not only can the tumour type vary between each patient, but even those diagnosed with the same type of tumour may have molecular characteristics that vary, and different proportions of these molecular mutations in each tumour, Therefore if a drug can influence a particular molecular pathway that makes up a large percentage of a patient’s tumour, that individual is going to respond more positively than another patient with a lower percentage of the same mutation.

To evaluate, monitor and analyse individuals in such detail is very challenging but as we move ever deeper into the world of personalised medicine, it is becoming clear just how important this is in order to bring us closer to a cure.

Thank you to the Society of British Neurological Surgeons (SBNS) and Professor Evanthia Galanis for highlighting such important issues, for suggesting ways in which they can be overcome, and for their invaluable dedication to enhancing the quality of life and improving the prognosis for brain tumour patients.

The vital basic research carried out at our Centres of Excellence is essential for laying the groundwork for more extensive specific research, clinical trials and ultimately new therapies to combat this devastating disease.

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