£76M investment backing into potential MND treatment, underlines benefit of charity funding

Promising new research to develop gene therapy to treat motor neuron disease and other conditions like dementia secures big investment thanks to charity backing.

A potential therapy for multiple conditions

A new potential treatment discovered by a team at University College London (UCL) and backed by the MND Association, My Name’5 Doddie Foundation and LifeArc, has moved one step closer to people living with MND after securing a multimillion pound investment from venture firms.

Launched earlier this week, US biopharmaceutical company Trace Neuroscience announced it has secured a $101 million capital investment to take forward a potential therapy for motor neuron disease (MND) and other neurological conditions including dementia, into clinical trials which could one day preserve and improve muscle function.

This work ultimately aims to develop a cutting-edge gene therapy to slow down, stop or potentially even reverse disease progression in MND. The potential treatment being investigated is an emerging area of drug development that targets a key aspect of diseases like MND and dementia at the genetic level. Research by co-founder of Trace Neuroscience, Professor Pietro Fratta at UCL, has been backed by the MND Association for many years and is currently being supported by the MND Association, medical research charity, LifeArc, and My Name’5 Doddie Foundation who have jointly awarded £500,000 to the UCL team.

MND is a rapidly progressing terminal illness which stops the signals from the brain reaching the muscles. This can cause people to lose the ability to walk, talk, eat and drink, and eventually breathe unaided. Average life expectancy is just 18 months from diagnosis. There is currently no cure or effective treatment.

The treatment works by restoring levels of a protein UNC13A, which plays an important role in the healthy communication between nerves and muscle cells. People with conditions like MND have reduced levels of this protein. Around 97% of people with MND will have changes in a protein known as TDP-43 which causes reduced levels of UNC13A. Therefore, these therapies have great potential for the majority of people with MND, and other conditions including dementia.

“There is an urgent need for treatments for MND patients so we’re really pleased to see that this work, which could slow disease progression, is now one step closer to reaching them. Our mission at LifeArc is to get treatments like this to those who need them much more quickly. If successful, this gene therapy could signal a major breakthrough – not only for patients with MND, but also potentially those living with dementia and other neurological conditions.”

Dr Paul Wright, Head of Motor Neuron Disease Translational Challenge, LifeArc

MND Association, Director of Research Development Dr Brian Dickie said: “This announcement marks a substantial investment into the further development of this specific area of MND research and will allow what we hope will be the rapid development of a promising new approach to treating motor neurone degeneration, targeting a pathological process which has been linked to 97% of all cases of MND.

“The MND Association has supported Professor Pietro Fratta and his lab over many years. Indeed, it was our joint Fellowship award, with the Medical Research Council, to Prof Fratta which led to the groundbreaking discovery in 2022 that has led to this promising new therapeutic approach. This news demonstrates how a modest amount of funding from the Association and others has wide reaching implications, in this instance and others providing the calatyst for further and large-scale investment to take a programme forward. It is exciting to see the direct impact the MND Association’s support is making in the search for effective treatments for this devastating disease.”

My Name’5 Doddie Foundation Director of Research, Dr Madina Kara, adds: “This announcement demonstrates the key role charity funding plays in enabling progress and helping leverage additional funds. There is currently huge momentum in MND research and this is a significant step forward in progressing genomic medicine for people living with MND. Using antisense oligonucleotides designed to restore levels of essential proteins, could enable treatments that can slow down, stop or reverse progression of MND.

“We continue to support the important work of Professor Fratta and his lab at UCL and recently awarded him a £500,000 grant, through our Advancing Treatment Awards, to develop a new approach to delivering gene therapies for MND that could benefit the great majority of people living with the condition.”

A short documentary ‘Turning the Tide’, backed by MND charities and the MND scientific community including the MND Association shows the progress of Pietro Fratta and his team’s work on UNC13A at UCL and shares first-hand the impact on the lives of people living with the disease and their hopes for ongoing research in this field.