First projects supported by the MND Translational Research Fund announced

24 January 2023

My Name’5 Doddie Foundation, LifeArc and Motor Neurone Disease Association have jointly awarded £1 million to researchers at University College London (UCL) to progress two pioneering new treatments for MND. These are the first two projects to be funded by the MND Translational Research Fund that the three charities joined up to create, with the total funding available for research being £1.5 million.

The first project, led by Professor Pietro Fratta of UCL Queen Square Institute of Neurology together with the Nucleic Acid Therapy Accelerator (NATA), aims to develop a gene therapy for MND. Using antisense oligonucleotides (ASOs), designed to rescue a specific gene lost during disease, the researchers hope to slow down, stop or reverse progression of MND. ASOs are an exciting development for MND, following the clinical trial of another ASO, Tofersen, for SOD1-MND, for which results were published last year.

The second project, led by Dr Barney Bryson and Professor Linda Greensmith of UCL Queen Square Institute of Neurology, explores an innovative treatment that has the potential to restore lost muscle function and prevent further muscle wastage in people with MND. The team’s unique therapeutic strategy involves injecting light-sensitive motor neurons derived from genetically modified human adult stem cells into nerves that control muscles in the limbs, hopefully restoring function of the paralysed muscles in response to pulses of light.

Sean McGrath, Medical Strategy Lead at My Name’5 Doddie Foundation, said: “We’re fortunate to have exceptional researchers in the UK making great strides in MND research, and these two projects are examples of important work in the field that we’re delighted to support. Translational research is critical as it focuses on moving lab discoveries to outcomes that can deliver real benefits for patients. This investment in MND research aligns with our strategy at My Name’5 Doddie Foundation and supports our commitment to giving hope to those living with MND.”

Read the full press release here.