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Functional Neurosurgery Research Group, Clinical Neurosciences, School of Clinical Sciences, Faculty of Health Sciences, University of Bristol

Learning & Research Building (Level 1).Southmead Hospital BS10 5NB Page 1

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Abbie's Army for Collaborative DIPG research project

Final Report

Name:

Dr Ali Bienemann

Title of Project:

Identifying novel drugs for the treatment of brainstem

tumours by direct infusion into the brain

Dates of Project:

01/05/2015 - 30/04/2016

1. Technical Progress

Background:

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 (Figure 1). We have

found this technique to be safe and to show promising signs of efficacy (Figure 2).

Importantly the risk of children suffering intolerable side-effects when chemotherapy

is administered by CED is very low.

Figure 2. Treatment with CED of carboplatin has so far proven

. safe and well tolerated with some promising signs of efficacy.

Figure 1. We have developed a unique implantable catheter system (a) which allows us to

repeatedly administer drugs to the brain through a port mounted on the skull (b). Using custom

Functional Research Group

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