From research to reality: progress in Motor Neuron Disease treatments at UCL
Motor Neuron Disease (MND) is a serious and uncurable condition. Researchers at UCL are working on new treatments thanks to a £78 million investment.
Around 5,000 adults in the UK are currently living with Motor Neuron Disease (MND).
This rare condition affects the brain and nerves, causing muscle weakness that gets worse over time. There is currently no cure for the condition, and it has a serious impact on patients’ everyday life and life expectancy.
But there may be hope on the horizon.
Researchers at UCL Queen Square Institute of Neurology and the Francis Crick Institute have been working on a new gene therapy which has the potential to treat MND in the future.
In 2021, researchers worked out that a major cause of damage to motor neuron cells was the loss of a protein called UNC13A. This protein helps nerve cells pass messages to each other. In patients with MND, the instructions for how to make this protein become corrupted and the cells are no longer able to make UNC13A.
This discovery is now being used to develop treatments that target UNC13A.
The research has been funded by a £78m investment from a UCL spinout biopharmaceutical company, Trace Neuroscience, co-founded by UCL Queen Square Institute of Neurology’s Professor Pietro Fratta. The researchers hope the therapy will move to human safety trials next year, followed by full clinical trials.
In this video, Professor Pietro Fratta, Professor Elizabeth Fisher (UCL Queen Square Institute of Neurology) and Dr Oscar Wilkins (Francis Crick Institute) talk about the pace of the research’s progress.
The advancements being made in the research are cause for celebration for both patients and clinicians alike. In this video, Dr Puja Mehta (UCL Department of Neuromuscular Diseases) shares what inspired her to contribute to the research as well as the message she hopes to one day deliver to her own patients.
The progress in the lab is being documented in a series produced by Quickfire Media. This ongoing project not only captures the researchers' work but also shines a spotlight on the lives of people living with MND. The 10-minute documentary is narrated by Patrick Darling, a young musician with the condition. His voice has been affected by his diagnosis, but researchers have been able to recreate his voice by using AI technology based on his YouTube channel content.
Watch the documentary in full below.
Biography
Professor Pietro Fratta is an MRC senior clinical fellow and MNDA Lady Edith Wolfson fellow at the UCL Queen Square Institute of Neurology and honorary consultant neurologist at the National Hospital for Neurology and Neurosurgery.
Professor Elizabeth Fisher is Professor of Neurogenetics in the Department of Neuromuscular Diseases, leading extensive research into neurodegeneration. Elizabeth is a Fellow of the Academy of Medical Sciences, a Member of EMBO, and a Fellow of the Royal Society of Biology.
Dr Oscar Wilkins is a post-doctoral fellow based at the Francis Crick Institute and UCL, in the lab of Pietro Fratta.
Dr Puja Mehta is an academic neurologist in London. She secured a Wellcome Trust fellowship in 2021 to undertake a laboratory-based PhD in motor neuron disease (MND) research at the UCL Queen Square Institute of Neurology.