Promising new research to develop gene therapy for MND secures major investment thanks to charity backing

A potential new treatment discovered by a team at University College London (UCL) has secured a multi-million pound investment from venture firms in the US. My Name’5 Doddie Foundation, alongside the MND Association, and medical research organisation LifeArc, funded the initial discovery phase of research.

Launched earlier this week, US biopharmaceutical company Trace Neuroscience announced it has secured a $101 million capital investment to take a therapy that could one day be used to treat MND and other neurological conditions including dementia, into clinical trials.

The ultimate aim is 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, was backed by My Name’5 Doddie Foundation, MNDA and Lifearc, with a joint award of £500,000.

The treatment aims to work by restoring levels of a protein called UNC13A, which plays an important role in the healthy communication between nerve and muscle cells. People with conditions like MND have reduced levels of this protein. This reduction can be caused by changes in another protein (TDP-43), which occur in 97% of people living with MND. It is for this reason that these therapies have great potential for the majority of people with disease, as well as other conditions including dementia.

My Name’5 Doddie Foundation Interim Director of Research, Dr Madina Kara, said: “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.”

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 neuron degeneration, targeting a pathological process which has been linked to 97% of all cases of MND.“

Dr Paul Wright, Head of Motor Neuron Disease at LifeArc, adds: “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.”

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. Watch the short documentary here.