DAISY’S TREATMENT

 

Daisy travelled to Bristol in July 2014 for ground breaking brain surgery where Professor Steven Gill and his pioneering team.

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Aims of the Functional Neurosurgery Research Group at the University of Bristol:

Identifying novel drugs for the treatment of brainstem tumours in children by direct infusion into the brain

The aim of the research is to identify new drugs for the treatment of brainstem tumours in children using a novel method of direct drug infusion into the brain. Suitable drugs are selected by undertaking a comprehensive program of pre-clinical research designed to determine whether the investigational drugs could be safe and effective.

Children with brainstem tumours have a very poor prognosis with almost all patients succumbing within 18 months of diagnosis. One of the major obstacles to the effective treatment of these tumours is the blood-brain barrier (BBB) which prevents the free passage of chemotherapies from the bloodstream into the brain. To date, no clinical trial of intravenous chemotherapy has shown a beneficial effect on the outcome for children with brainstem glioma. Children also suffer significant side-effects when chemotherapies are administered intravenously in high doses.

In Bristol we have been treating children with progressive brainstem tumours using a novel neurosurgical method of drug delivery to the brain called Convection-enhanced Delivery (CED). Our method involves the robotic implantation of very fine catheters into the brain and infusion of drugs directly into the tumour, thus bypassing the BBB. We have found this technique to be safe and to show promising signs of efficacy. Importantly the risk of children suffering intolerable side-effects when chemotherapy is administered by CED is very low.

 

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Figure 1. To date we have treated 6 paediatric patients with advanced brainstem tumours with CED of carboplatin. Using custom software we have planned and implanted up to 4 trajectories per patient in order to target the enhancing tumour and peritumoral tissue (a, blue). By performing real-time MR imaging during infusions we obtain a measure of drug distribution (a, red). We have also developed a robot-assisted method for catheter implantation which has proved both safe and highly accurate (b).

 

The research team at the University of Bristol is led by Professor Steven Gill, a consultant neurosurgeon who is a world leader in the technique of convection-enhanced drug. Professor Gill’s Functional Neurosurgery Research Group has extensive pre-clinical and clinical CED research experience and has successfully completed a number of studies which have led directly to clinical trials. This group has unique and world-leading CED catheter technology which is currently being used to treat patients with a range of neurological disorders including adults and children with malignant brain tumours and Parkinson’s Disease.

The ultimate aim of our work is to make an impact on this disease by testing novel drug combinations in accurate pre-clinical models, and by delivering the drugs directly into the brain. This approach could make a difference to children with brainstem tumours by bypassing the blood-brain barrier thus making them more effective and by significantly reducing the risks of children suffering intolerable side-effects from the chemotherapy.

If successful we envisage progression to a phase 1 clinical trial within 2 years using world-leading and unique CED catheter technology which allows drugs to be repeatedly delivered to the brain without repeated surgical intervention. We are currently implanting this catheter system in children with brainstem tumours and delivering the chemotherapy carboplatin. No other research group in the UK, or indeed the world, has comparable technology. We believe that if we are successful in identifying efficacious drug combinations, when delivered using our novel catheter systems, we could make an impact on this devastating disease.

 

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Figure 2. Our unique CED catheter system comprises implantable microcatheters and a skull anchored port which allows drugs to be repeatedly administered without the need for further surgery.

 

The treatment would not be possible without the very talented and dedicated team of Renishaw UK.

All precious funds raised go directly to the team who are making fantastic ground in finding a cure and saving lives.