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Stem cells and regenerative medicine

Motor neurone disease

Motor neurone disease (MND) is a devastating progressive disorder dominated by weakness and paralysis that results from death of the nerve cells that connect the brain, spinal cord and muscles.

For reasons that are largely unknown, these particular nerve cells — called motor neurons — die one after another, which means that bit by bit the patients lose control over their movements. This gradual decline can start almost anywhere including the limbs, swallowing  and speaking muscles. Ultimately it progresses, leading to complete paralysis with involvement of the breathing muscles that is a common cause of death.

Because there are no treatments for this condition, and in general deterioration is rapid with average survival from diagnosis of only a few years, the number of prevalent cases is comparatively small. For example, there are around 400 patients living with MND in Scotland at any one time.

In order to develop treatments we need to understand better the underlying causes and drivers of MND. One exciting model system that is a focus of research at CRM is using stem cells, including from patients, to model aspects of MND 'in a dish'.

Work at CRM

  • Prof Siddharthan Chandran’s lab, in partnership with Prof Wilmut’s lab, generates cultured motor nerves and their supporting cells (called glia) from human embryonic and induced pluripotent stem cells, to study the mechanisms of degeneration and test potential new therapies. Some of the stem cells are obtained from a skin biopsy donated by patients with an inherited form of MND. Using such cells means that the derived neurons in the dish have the same defect as those in the patient’s brain and spinal cord. The cells can then be used to study these defects and screen for drugs to restore their normal function. Prof Chandran is also the Director of the Euan MacDonald Centre for Motor Neurone Disease Research, a Scotland-wide collaboration of researchers studying all aspects of motor neurone disease.
  • Prof Sir Ian Wilmut and his group also develop technologies using adult cells to generate nerve cells, in order to better understand the requirements of these cells, and potentially use them for cell therapies.