The terminal condition currently affects around 5,000 people in the UK.
A new documentary has highlighted the discovery of a promising new target for new treatments of motor neurone disease (MND) made by researchers at University College London (UCL).
The short documentary, Turning the Tide, was funded as part of LifeArc’s MND Translational Fund and other MND charities, as well as the MND scientific community.
Currently affecting around 5,000 people in the UK, MND is a terminal condition that affects the nerve cells – motor neurons – in the brain and spinal cord, which control muscle movement.
Individuals with MND slowly and progressively lose the ability to walk, talk, eat and breathe as the cells die and muscles in the body start to waste away.
The new documentary provides insight into what it is like for those living with MND as well as their hopes for ongoing research in the field.
Researchers at the UCL Queen Square Institute of Neurology identified how the loss of a protein from the nucleus of motor neurons, TDP-43, corrupts the genetic instructions required to produce another key protein, UNC13A, which impacts the normal functioning of the cells.
Furthermore, researchers went on to identify how antisense oligonucleotide technology, a form of gene therapy treatment, could block this process and potentially slow down the profession of MND.
Backed by a £500,000 award from LifeArc, the Motor Neurone Disease Association and the My Name’5 Doddie Foundation, the potential treatment works by targeting the cause of the disease at a genetic level by restoring the production of the UNC13A protein.
They aim to secure further funding to advance the UCL research team and kickstart the process of getting a treatment clinically tested for people living with MND.
Stéphane Maikovsky, interim chief executive officer of LifeArc, said: “Funding pioneering research into the disease… is critical to unlock desperately needed treatments for patients.
“This discovery marks a significant advancement in our understanding of MND and suggests an exciting target for future treatments.”
