Researchers Secure £3.9M Grant to Explore Early Axonal Dysfunction in ALS and FTD

Researchers from King’s College London (KCL) and University College London (UCL) have received a £3.9 million grant from the Medical Research Council (MRC) and the Motor Neurone Disease (MND) Association to investigate axonal dysfunction in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The project seeks to develop therapeutic strategies to address these neurodegenerative diseases at their early stages.

ALS and FTD are devastating conditions that cause progressive nerve cell degeneration. ALS, a fatal type of MND, leads to the degeneration of nerve cells in the spinal cord and brain, while FTD results from damage to neurons in the frontal and temporal lobes of the brain. Together, these conditions affect over 35,000 people in the UK.

Axonal dysfunction, characterized by the degeneration of axons - the structures in the neurons responsible for transmitting signals to other cells - is a key pathology in both ALS and FTD. Early signs of axonal dysfunction include hyperexcitability, abnormal plasticity, and reduced synapse function. These abnormalities significantly contribute to disease progression.

The collaborative project, led by Professor Juan Burrone from the Centre for Developmental Neurobiology at KCL and Dr. Ivo Lieberam from the Centre for Gene Therapy and Regenerative Medicine at KCL, aims to systematically investigate these early axonal pathologies. The researchers will develop human neuronal culture models with mutations found in patients with ALS and FTD. These models will be used to characterize altered axon functions and uncover the specific molecular mechanisms behind the dysfunction.

"This is an exciting collaborative project that aims to understand the cellular pathologies behind ALS/FTD to identify a new therapeutic angle," said Professor Burrone.

The final stage of the project involves restoring normal neuronal activity and communication in the cultured neurons. If successful, this approach could potentially prevent neuronal cell death and halt the progression of these debilitating diseases.

This grant follows a recent £2 million funding awarded to Health Data Research UK and Dementias Platforms UK to launch a new initiative aimed at accelerating the discovery of new diagnostics and treatments and improving care for MND patients. These combined efforts highlight the ongoing commitment to understanding and combating neurodegenerative diseases.

In summary, this groundbreaking research funded by the MRC and the MND Association aims to pave the way for novel therapeutic strategies targeting early axonal dysfunction in ALS and FTD. Through this work, researchers hope to bring new hope to patients suffering from these currently incurable diseases.