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Modelling Enables Breakthroughs in Neuromuscular Disease Research

A frozen Eppendorf tube held with tweezers.
Credit: AMSBIO.
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AMSBIO has published a case study that describes how a team of scientists, led by Dr. Roger Kamm from the Massachusetts Institute of Technology (MIT), has developed a protocol for the fabrication of a 3D microfluidic neuromuscular platform that can be used to model Motor Neuron Diseases (MND’s).

For one of the most fatal types of MND, amyotrophic lateral sclerosis (ALS), patients have a life expectancy of only 2 to 5 years. Despite widespread research efforts, effective treatments for MNDs remain elusive, largely due to the complex nature of these diseases and a lack of in-depth understanding of their underlying mechanisms.

Researchers at MIT have developed novel approaches to unravel the pathogenesis of MNDs using CELLBANKER® 1 and STEM-CELLBANKER® cryopreservation media from AMSBIO. These new developments hold significant promise in revolutionizing the approach to drug discovery and better understanding the development of MNDs.

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Dr. Kamm said “Traditional drug discovery methods for MNDs have often disregarded the role of skeletal muscle cells, focusing predominantly on their effect on motor neurons. However, our new 3D neuromuscular model offers a broader view of the pathology and presents a novel avenue for uncovering effective treatments targeting both motor neurons and muscle cells, that address the complex nature of MNDs”.

He added “Our protocol describes a development of a new microfluidic chip that contains compartments for each cell type, allowing for the separation of the motor neuron and muscle cell cultures to mimic the physiology found in living tissue. Induced pluripotent stem cells (iPSCs) from either healthy donors or ALS patients were differentiated into neural stem cells, which were expanded and cryogenically preserved using STEM-CELLBANKER® from AMSBIO. Using our innovative approach shows great promise to help tailor treatments to an individual’s genetics and consequent drug response to enhance their effectiveness while minimising side effects. By providing a more accurate representation of the disease, the 3D neuromuscular model could accelerate drug discovery and streamline the transition from laboratory research to clinical applications”.