Research Proposal

Research Proposal Lay Summary

In vitro evaluation of the effect of cannabidiol as an adjuvant therapy for paediatric brain tumours

Dr Lisa Storer, Associate Professor Saoirse O’Sullivan and Professor Richard Grundy

“Brain tumours are the leading cause of cancer related death in children and are one of the most challenging childhood cancers to diagnose and to treat. Only 50-60-% of children diagnosed with a brain tumour are cured and 60% of those cured are left with a disability affecting quality of survival. Ependymoma and high grade glioma account for up to a quarter of all children diagnosed with a brain tumour. Compared to other childhood brain tumours 5-year survival rates are relatively poor. It has been reported that 39-64% of those diagnosed with an ependymoma will still be alive 5 years after diagnosis and that 15-35% of those diagnosed with a high grade glioma will survive this length of time. In addition, tumour recurrences are common with treatment options becoming more limited and many children dying from the disease. We therefore would like to investigate other treatment options which could be used in conjunction with standard therapies.

Cannabidiol (CBD) is one of the active cannabinoids found in the Cannabis plant. It has been shown to have a lack of side effects, including a lack of psychoactive side effects which are more commonly experienced with other active agents found in cannabis. It has also been shown not to interfere with several psychomotor and psychological functions and therefore it is under consideration for a wide range of potential medical applications. CBD is already in use in childhood epilepsy patients so it has already been subject to rigorous testing, clinical trials and safety examinations and there are currently a number of clinical trials in progress to obtain safety and efficacy data on CBD in other conditions including Dravet syndrome and Tuberous Sclerosis Complex. This means that it could quickly be re-purposed as an anti-cancer drug.

We are therefore interested in investigating whether CBD can be used as a way of attacking cancer cells. In order to do this we would like to grow brain tumour cells and normal brain cells in the presence of CBD. The brain tumour cells we will use have all been grown from either Ependymoma or high grade glioma tumours. We will grow them under standard conditions in the presence and absence of CBD. After 7 days we will measure the level of cell death and how many viable cells are present by two different assays. We will then perform different stains on the cells to see how many of the cells are dividing, whether the cells are undergoing cell death and also for a group of proteins which regulate the expression of genes [peroxisome proliferator-activated receptors (PPARs)]. These PPARs play essential roles in the regulation of cell differentiation, development, metabolism and the formation of cancer. We expect the cells (brain tumour and normal brain) grown in our standard conditions to be healthy and actively dividing. We expect that normal brain cells grown in CBD to also be healthy. However, we expect the brain tumour cells grown in CBD to be unhealthy and dying.

The James Lind Alliance priority setting partnerships were created in 2015 to identify clinical research questions of greatest importance to the patients and families actually affected by brain tumours. One of the questions raised was “How effective are complementary and alternative medicines, which include supplements and herbal remedies, compared to standard treatment alone, for extending and improving quality of life in malignant brain tumour patients?” We would include the use of CBD in this category and therefore feel that it is timely to perform this research which will give us vital information into the potential clinical effectiveness of CBD in paediatric brain tumours.”