A brain in a dish: Developing a sophisticated system to test MS treatments

Michele Binder

Florey Institute of Neuroscience and Mental Health

February 2022

specialisation: Neurobiology

focus area: A cure via repair and regeneration

funding type: Incubator

project type: Investigator Led Research

Summary

The lack of therapies for individuals with progressive MS remains a challenge. Currently, there’s a concentrated effort on developing treatments that facilitate myelin repair—the protective sheath around nerves—to address progressive MS. However, a significant obstacle lies in translating promising therapies from laboratory models to clinical application. Another hurdle is the absence of suitable human models for testing new treatments.

To address these gaps, Michele Binder and her team aim to create a “brain in a dish” (brain organoids) using stem cells, forming a complex 3D culture mimicking some brain functions, including myelin formation damaged in MS. The goal is to establish a method using these “mini-brains” to replicate MS-related myelin damage. Ultimately, this model will serve as a testing ground for new treatments for progressive MS.

This innovative approach is pivotal in bridging the gap between laboratory models and human trials, enhancing the prospects of success in a clinical setting.

Outcome

Michele Binder and her team have achieved significant milestones in replicating and studying myelin damage, a hallmark of MS, using advanced techniques. They have successfully created ‘mini-brains’ and have established methods to myelinate and induce demyelination in these brain organoids. By incorporating important brain immune cells known as microglia into these structures, they’ve replicated demyelination induction using lysolecithin, an agent commonly used in demyelinating laboratory models. This discovery demonstrates a significant breakthrough in understanding the progression of MS.

The larger context here is the pressing need for treatments targeting progressive MS, which lacks effective therapies. Bridging the gap between laboratory models and human trials is important, and this project addresses this.

Ultimately, this innovative organoid model holds promise as a platform to test and develop new treatments for progressive MS, potentially revolutionising therapeutic approaches by providing a more accurate human-based testing environment.

Updated 31 March 2023

lead investigator

total funding

$25,000

start year

2022

duration

1 year

STATUS

Current project

Stages of the research process

Fundamental laboratory Research

Laboratory research that investigates scientific theories behind the possible causes, disease progression, ways to diagnose and better treat MS.

Lab to clinic timeline

10+ years

Translational Research

Research that builds on fundamental scientific research to develop new therapies, medical procedures or diagnostics and advances it closer to the clinic.

Lab to clinic timeline

5+ years

Clinical Studies and Clinical Trials

Clinical research is the culmination of fundamental and translational research turning those research discoveries into treatments and interventions for people with MS.

Lab to clinic timeline

3+ years

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A brain in a dish: Developing a sophisticated system to test MS treatments