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Modeling Interactions Could Further Research of Treatments for Chronic Muscular Diseases

“Current drug development processes take anywhere from 10-15 years and cost somewhere in the region of $1.2 billion.”

Neuromuscular junction studies are mainly concerned with understanding the connection between the nervous system and the muscular system, specifically with the synapses that transmits information across and back. By modelling this interaction in the lab, scientists hope to investigate subtle changes in muscle functional outputs in response to treatment with novel modulatory and inhibitory compounds. Such a system may prove invaluable in furthering research efforts aimed at the development of effective treatments and reducing the overall cost of clinical trials.

James J. Hickman, Ph.D., Professor of Chemistry, Biomolecular Science and Electrical Engineering at the University of Central Florida (UCF) said that modern clinical trial success rates are in the region of 1 in 5. “If we can improve this success rate, we believe the cost per drug will be dramatically reduced and the development process significantly streamlined since far fewer clinical trials will produce negative results.”

Dr Hickman, along with the team at UCF's NanoScience Technology Center have been working on developing a system to monitor the neuromuscular junction functions in the lab for the past 20 years. Dr Hickman said, “It is no use developing a system that produces functional cells if you have no means to monitor that functionality.”

 When asked for examples of issues faced when trying to recreate natural human functions in the lab, Dr Hickman explained that the natural environment of neuromuscular junctions (i.e in our body) allows for a continuous bombardment of signals delivering information to and from the body. This is hard to replicate in the lab, thus there are some underdeveloped cultured neuromuscular junctions, leading to inconsistent cultures and therefore hard to replicate or even determine a working simulation for repetition.

Research in the area of neuromuscular junctions are all part of a bigger picture — the development of 'body-on-a-chip' or the 10-organ culture platform. The aim of the 'body-on-a-chip' technology is to utilize human cells, representing the means to supplement and hopefully remove animals from pre-clinical testing. Dr Hickman said, “The pharmaceutical industry is in desperate need of highly predictive pre-clinical screening systems to streamline the drug development process and shorten current validation protocols, which can take a decade to implement.”

By reducing the time and cost consumed for drug development in clinical trials, and doing away with often uninformative, and ethically questionable, animal testing techniques in the process, the Dr Hickman and the team's research will be able to significantly improve healthcare delivery. 

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