Delivering ATMPs to the human brain in early phase clinical trials

We work with the NHS and companies running clinical trials to offer cutting-edge treatments for people with
brain diseases.

Complimenting this, our active research programmes help us understand and optimise therapy delivery. This ranges from advanced magnetic resonance (MR) imaging and modelling, to developing better surgical devices and refining clinical trial design.

We are focussed on ensuring that these critical early clinical trials don’t fail because of inadequate delivery.

This work aims to bring the promise of these ground- breaking therapies into clinical reality for our patients.

The image shows a patient mid-surgery in an MRI scanner with the neurosurgeon in cap, mask, gown and gloves preps them for surgery.

A patient being prepped for surgery in an MRI scanner.

How do we get therapies into your brain?

The blood-brain barrier blocks most ATMPs from reaching the brain through the blood stream. However, there are different ways we can deliver advanced therapies during clinical trials.

Methods of delivery we might use include:

Intravenous

Delivers therapies to the brain through the blood, via a cannula inserted into a vein. We use this for ATMPs that we know will cross the blood-brain barrier.

Intrathecal

Delivers therapies directly to the cerebrospinal fluid (CSF) that surrounds the brain, via a lumbar puncture, which is when a needle is inserted into the lower back, between the bones of the spine.

A graphic showing the profile of a person's head receiving treatment via brain surgery

Neurosurgical

Delivers therapies directly to specific areas of the brain using surgically placed devices while the patient is in an MRI scanner, to see exactly where the therapy is going in real-time.

We are the only centre in the UK (and one of just five worldwide) with the expertise and facilities to safely deliver these neurosurgical treatments.

Clinical trials we are delivering include:

ASPIRE-FTD

Treating participants with frontotemporal dementia with GRN gene mutations, sponsored by AviadoBio.

REGENERATE-PD

A study of AB-1005 (AAV2-GDNF) gene therapy in adults with moderate Parkinson’s disease sponsored by AskBio.

AMT-130

AMT-130: The first gene therapy clinical trial in Huntington’s disease sponsored by UniQure.

Be Part of Research

Be Part of Research is a free service which makes it easy to find and take part in vital health and care research across the UK.

Learn how you can participate in research and clinical trials in the UK: bepartofresearch.nihr.ac.uk