The Medical Research Council and the Motor Neurone Disease (MND) Association have awarded researchers from King’s College London (KCL) and University College London (UCL) £3.9m to investigate an early disease pathology in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).
The team aims to explore and systematically investigate the axonal dysfunction in neurodegeneration and design therapeutic strategies to address the disease at an early stage.
Axonal dysfunction occurs when there is a complete degeneration of axons – parts of the neurons that allow them to carry signals and transmit information to other cells. These results are seen in neurodegenerative diseases such as ALS and FTD.
ALS is a fatal type of MND that causes progressive degeneration of nerve cells in the spinal cord and brain, while FTD is the result of damage to neurons in the frontal and temporal lobes of the brain.
Combined, these conditions are estimated to affect more than 35,000 people in the UK.
Previous studies have observed that axons exhibit abnormal functions, including hyperexcitability, abnormal plasticity and reduced synapse function.
UCL and KCL researchers intend to investigate these early axonal pathologies to develop novel therapeutic strategies to restore axon function and establish its role in disease progression.
Researchers aim to develop human neuronal culture models that carry mutations found in patients with ALS and FTD to characterise altered axon functions and investigate the specific molecular mechanisms behind the dysfunction.
At the final stage of the project, researchers will aim to restore normal neuronal activity and communication and potentially prevent neuronal cell death.
Lead investigator of the project, Juan Burrone, professor of developmental neurophysiology, Centre for Developmental Neurobiology, KCL, commented: “This is an exciting collaborative project that aims to understand the cellular pathologies behind ALS/FTD to identify a new therapeutic angle.”
Most recently, in July, Health Data Research UK and Dementias Platforms UK received £2m in funding to launch a new initiative to accelerate the discovery of new diagnostics and treatments as well as improve care for MND patients.

https://pharmatimes.com/news/mrc-and-mnd-association-award-3-9m-to-kcl-and-ucl-for-als-ftd-research/