The research hopes to develop a therapy that will deliver viral and gene therapy directly in to the brain to restore the function of motor neurons. Lab work led by Bristol-based neurosurgeon Professor Steven Gill has shown that specific viruses that are modified to carry therapeutic genes can be transported into the spinal cord using a technique called Convection Enhanced Delivery.
Professor Gill, known for his pioneering work with delivering drugs directly into the brain with CED to treat Parkinson’s Disease and brain tumours, has seen positive results pre-clinically with gene therapy targeting Motor Neuron Disease.
Early testing has shown the technique to provide safe and effective gene delivery to motor neurons and the next ‘pre-clinical’ stage is to evaluate the most effective dose of the virus that can be safely delivered.
This is a critically important step in bringing gene therapies for MND closer to being used by patients. Nevertheless, before clinical trials in humans can be started researchers need to determine the most effective doses that can be delivered safely.
The cost of the pre-clinical stage is £150,000 and Glasgow-based Funding Neuro’s campaign to raise the money has been kick started by a £50,000 donation from the My Name’5 Doddie Foundation.
Funding Neuro has previously worked with Professor Gill to raise money for research to reverse Parkinson’s Disease and for treating children with Diffuse Intrinsic Pontine Glioma (DIPG), a brain stem cancer that affects young people.
Professor Steven Gill said: “The standard treatments that are currently available for Motor Neuron Disease may enhance a patient’s quality of life but there is yet to be a therapy to combat the disease. Backed by Funding Neuro, our project has the potential to offer a fast track approach to a neuroprotective and regenerative treatment for the disease.
“By bringing together experts in neurosurgery, gene therapy, medical engineering and biotech pharma we have developed the technology and knowhow to successfully deliver gene therapies to the central nervous system.
“We are now in a unique position to rapidly translate these promising lab results to clinical trials to protect and repair motor neurons.”
Doddie Weir of the My Name’5 Doddie Foundation said: “We are pleased to be supporting such an innovative programme. Success has been shown in the lab using gene therapy to combat the disease, however to date this has not been translated successfully into a patient.
“We hope that by combining gene therapy with a delivery system that is able to get the therapy to where it can be effective, will provide real hope for people with the condition.”
Dr Brian Dickie, Director of Research Development at the Motor Neuron Disease Association, said: "One of the major challenges in treating neurodegenerative diseases such as MND is the successful delivery of drugs into the brain and spinal cord. Professor Gill and colleagues are adopting an innovative approach that could increase the chances of success when testing experimental therapies."