Motor neuron disease discovery offers new insights into potential treatment targets

Scientists have discovered how certain forms of motor neuron disease begin and progress at cellular and molecular levels, revealing potential new ways to slow down or even stop this process. The team are already working closely with pharmaceutical companies to use this knowledge to develop new treatments for motor neuron disease and other neurodegenerative conditions. By studying cells from patients with motor neuron disease, also known as amyotrophic lateral sclerosis (ALS), the team from the Francis Crick Institute and UCL revealed a detailed picture of how motor neurons - nerve cells in the brain and spinal cord that control our muscles and allow us to move, talk and breathe - decline and die. The research, published in Cell Reports , also shows that healthy neuron-supporting cells called astrocytes may play a role in the survival of motor neurons in this type of ALS, highlighting their potential role in combating neurodegenerative diseases. The work was co-led by Dr Sonia Gandhi and Dr Rickie Patani, group leaders at the Francis Crick Institute and UCL, and consultant neurologists at the National Hospital for Neurology and Neurosurgery, Queen Square. "Understanding how and why neurons die is clearly vital in neurodegenerative diseases, but part of the puzzle is also understanding the emerging role of astrocytes in this context," said lead author Dr Sonia Gandhi (UCL Institute of Neurology). The team took skin cells from healthy volunteers and patients with a genetic mutation that causes ALS, and turned them into stem cells capable of becoming many other cell types.