Motor Neurone Disease MND
This campaign will lay the foundations for human trials of a pioneering therapy for Motor Neurone Disease (MND). The fatal disease affects the brain and nerves and causes weakness over time. There is currently no cure for the condition.
The work led by Bristol-based neurosurgeon Professor Steven Gill brings combines three elements. Gene Therapy with Viral Vectors delivered via Convection Enhanced Delivery CED.
It is known that specific viruses can have a therapeutic effect on motor neurons. With a technique called Convection Enhance Delivery (CED). This is able to deliver these viruses directly to the neurons in the brain. For many diseases such as MND. Delivery of genes to affected neurons to correct or repair defects has been a major challenge.
Professor Gill, known for his pioneering work with delivering drugs directly into the brain with CED to treat Parkinson’s Disease and brain tumours. Professor Gill has conducted a number of pre-clinical tests with gene therapy targeting Motor Neurone Disease.
The tests have shown promising results. The next ‘pre-clinical’ stage is to evaluate the safety and efficacy of the treatment. This crucial phase must be completed before clinical trials in humans can be started.
Professor Steven Gill said: “The standard treatments that are currently available for Motor Neurone 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.
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