Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease, is a medical condition that affects the cells connecting the brain to muscles, called motor neurons. ALS sufferers progressively lose control of their muscles which can eventually lead to paralysis, losing the ability to walk, swallow and even breathe. There is currently no cure for the disease.
Departing from traditional animal models, Ivo Lieberam and Stephanie Hynes, in collaboration with the National University of Singapore, published their findings in Advanced Biosystems, describing their innovative way of investigating the connection between the brain and muscle in ALS. Their model, called ‘motion in a dish’, is based on artificially grown tissues resembling nerve and muscle tissues grown directly from stem cells.
Nerve and muscle tissues are connected by motor neurons in their model which have been genetically engineered to carry optical probes allowing researchers to transmit signals to them with light flashes.
The team validated their model in two ways; firstly, they activated the nerve-muscle circuit with light over several days and observed that, true to life, this strengthened the connections between motor neurons and muscle fibres. Secondly, they showed that a genetic variation associated with ALS in humans precipitated the degeneration of nerves and the loss of connections between nerves and muscle fibres. They reversed these effects with Necrostatin, a candidate ALS drug.
Their innovative research opens up several new avenues for working towards treating ALS. The next steps for this model are to investigate how some people are genetically protected from developing ALS and how this could be mimicked with drugs. The model offers the exciting potential for scientists to discover new drugs for ALS faster, more cheaply and without animal experimentation.
Article by Hannah Forrest