Title: Tessa Jowell BRAIN-MATRIX
Funder: The Brain Tumour Charity

Abstract: Aim Run a clinical trial that will establish critical infrastructure to deliver precision medicine for all glioma patients in the UK Why Brain cancer is a challenging disease to treat due to how the brain defends itself (its natural defences), accessibility of the tumours, their ability to spread rapidly and the complexity of brain cancer. In addition to the biology, there are challenges in the clinical research setting such as lack of infrastructure, which mean that progress in treating brain cancer has not matched that of other cancers. What BRAIN-MATRIX is a feasibility study to see if it is possible to create a way of setting up an infrastructure, or platform, to address the biological and clinical challenges of treating gliomas (a type of brain tumour). This platform will bring together many different people including patients, brain doctors, cancer doctors, technicians, scientists and drug companies. BRAIN-MATRIX will use molecular stratification. This means that patients with a glioma will be grouped, based on the specific characteristics of their tumour, such as the types of tumour cells and the substances, or “mutations”, that they have within them. This information can help to decide which “targeted” treatment would be best for each patient with the glioma, so that they get the best possible treatment. How We will establish a network of patients, doctors and scientists that is supported by the platform. This platform will deliver: • Rapid and accurate molecular diagnosis which means that subsets of patients can be identified and grouped for targeted treatment • A network of clinical centres • Robust ways of collecting, processing and storing brain tumour tissue, images and clinical data • Links with global high quality basic and clinical science • Access to novel and repurposed drugs and technologies through partnerships with industry and other organisations • Long-term sustainability Any patient diagnosed with a glioma that is able to have surgery will be allowed to enter the trial through one of ten UK-based centres. What happens next depends on the type of glioma that the patient has. At the beginning of this study patients will follow one of two routes: 1. If no “treatable” substances, or mutations, are identified in an individual patient’s brain tumour a. The way they respond to the surgery, b. Whether their cancer comes back, and/or c. Whether they do any better following a second surgery will be monitored. This is “Arm 0”; a prospective study of extent of surgical resection in gliomas. 2. If BRAF V600E is found within an individual’s brain tumour, that patient will be given a type of treatment specific to this substance/mutation. This is “Arm 1”. The goal is to find more distinctive substances/mutations that can have specific “targeted” treatments, and therefore more “Arms” to be added to the platform. Benefit With more targeted treatments we will be able to reduce the possible side effects of brain cancer treatments and increase the survival of glioma patients.

Lay abstract: Aim Run a clinical trial that will establish critical infrastructure to deliver precision medicine for all glioma patients in the UK Why Brain cancer is a challenging disease to treat due to how the brain defends itself (its natural defences), accessibility of the tumours, their ability to spread rapidly and the complexity of brain cancer. In addition to the biology, there are challenges in the clinical research setting such as lack of infrastructure, which mean that progress in treating brain cancer has not matched that of other cancers. What BRAIN-MATRIX is a feasibility study to see if it is possible to create a way of setting up an infrastructure, or platform, to address the biological and clinical challenges of treating gliomas (a type of brain tumour). This platform will bring together many different people including patients, brain doctors, cancer doctors, technicians, scientists and drug companies. BRAIN-MATRIX will use molecular stratification. This means that patients with a glioma will be grouped, based on the specific characteristics of their tumour, such as the types of tumour cells and the substances, or “mutations”, that they have within them. This information can help to decide which “targeted” treatment would be best for each patient with the glioma, so that they get the best possible treatment. How We will establish a network of patients, doctors and scientists that is supported by the platform. This platform will deliver: • Rapid and accurate molecular diagnosis which means that subsets of patients can be identified and grouped for targeted treatment • A network of clinical centres • Robust ways of collecting, processing and storing brain tumour tissue, images and clinical data • Links with global high quality basic and clinical science • Access to novel and repurposed drugs and technologies through partnerships with industry and other organisations • Long-term sustainability Any patient diagnosed with a glioma that is able to have surgery will be allowed to enter the trial through one of ten UK-based centres. What happens next depends on the type of glioma that the patient has. At the beginning of this study patients will follow one of two routes: 1. If no “treatable” substances, or mutations, are identified in an individual patient’s brain tumour a. The way they respond to the surgery, b. Whether their cancer comes back, and/or c. Whether they do any better following a second surgery will be monitored. This is “Arm 0”; a prospective study of extent of surgical resection in gliomas. 2. If BRAF V600E is found within an individual’s brain tumour, that patient will be given a type of treatment specific to this substance/mutation. This is “Arm 1”. The goal is to find more distinctive substances/mutations that can have specific “targeted” treatments, and therefore more “Arms” to be added to the platform. Benefit With more targeted treatments we will be able to reduce the possible side effects of brain cancer treatments and increase the survival of glioma patients.

Research activity codes: 2.6 RESOURCES AND INFRASTRUCTURE (AETIOLOGY);4.5 RESOURCES AND INFRASTRUCTURE (DETECTION);5.9 RESOURCES AND INFRASTRUCTURE (TREATMENT DEVELOPMENT);8.5 RESOURCES AND INFRASTRUCTURE (HEALTH SERVICES)
Health categories: CANCER AND NEOPLASMS