The work led by Bristol-based neurosurgeon Professor Steven Gill 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 and a technique called Convection Enhance Delivery (CED). This enables delivery of these viruses directly to the neurons in the brain. Drug delivery to affected neurons to correct or repair defects has been a major challenge.
Professor Gill is known for his pioneering work with delivering drugs directly into the brain with CED to treat Parkinsons 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 patients 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.
The cost of the pre-clinical stage is £150,000. Thanks to My Name5 Doddie Foundation we are a third of the way there! Please help support our campaign by donating and together we can give a future to those suffering.
Update message from Funding Neuro 28/08/19
Thank you to everyone that has donated to the MND campaign so far, and to all of the wonderful fundraisers who are doing lots of challenges and events. Some are crazy, some are physically challenging but ALL are invaluable and allow us to continue this vital research.
We are almost half way towards the fundraising target, but with what we have Professor Gill has been able to continue his work.
The news so far is good, the expectations have been met regarding getting the therapy to where it can work. Most importantly this has been achieved safely. The next stage is to manufacture the drugs so that this can be tested in man. This process is expensive and we are liaising with a number of organisations to make this happen as quickly as possible.
Your contributions are making a real difference, thank you so much for being a part of this, please continue your tremendous fundraising efforts and we will continue to support Professor Gill and his team.