Revolutionizing Neurological Disorder Treatments: 3D Self-Assembled Human Brain Model Soars to Space Station for Precision Medicine Testing

The Global Impact of Neurological Disorders:

Neurological disorders have a profound impact on global health, affecting nearly one billion individuals worldwide, which accounts for approximately one in six people. From Alzheimer's and Parkinson's disease to epilepsy and migraines, these conditions demand urgent attention for improved treatments. In response to this pressing challenge, researchers at the biotechnology start-up, Axonis, are embarking on a ground-breaking mission to leverage the unique environment of the International Space Station (ISS) National Laboratory. By exploring the effects of microgravity on the maturation of human brain cells, which self-assemble into three-dimensional spheroids resembling aspects of the human brain, this innovative research aims to propel disease modelling and pave the way for novel therapies to combat neurological disorders on Earth.

Leveraging the International Space Station for Medical Advancements:

Researchers from biotechnology start-up Axonis are working to help improve treatments for patients with neurological disorders by leveraging the unique environment of the International Space Station (ISS) National Laboratory. The microgravity conditions on the ISS offer a novel platform to study how human brain cells mature in three-dimensional spheroids, mimicking certain aspects of the human brain.

Advancing Disease Modelling through Space-based Research:

The research team's primary focus is to examine how microgravity affects the maturation of human brain cells that form these three-dimensional spheroids. These models hold immense potential for advancing disease modelling and could lead to the development of new therapies to treat neurological disorders in patients on Earth.

Securing Funding for the Pioneering Project:

Axonis secured a significant grant for this ambitious project through the Technology in Space Prize. The prize was made possible by generous contributions from Boeing and the Center for the Advancement of Science in Space, Inc. (manager of the ISS National Laboratory), in partnership with the MassChallenge start-up accelerator program.

Launching the Investigation to the ISS:

The investigation, set to launch on Northrup Grumman's 19th Commercial Resupply Services mission, involves converting induced pluripotent stem cells (iPSCs) into different types of brain cells—neurons, microglia, and astrocytes—on Earth. Cultures of these cells will then be sent to the orbiting laboratory, where they are expected to assemble into three-dimensional spheroids.

A Game-Changing Approach to Studying the Human Brain:

According to Shane Hegarty, Axonis' Chief Scientific Officer, the 3D self-assemblies represent a revolutionary approach to studying the human brain. Unlike traditional organoids, which start as a single cell and grow into organ-like structures, these spheroids form by assembling together, resulting in increased maturity and potential for more accurate modelling.

Overcoming Limitations of Traditional Organoids:

Hegarty explains that traditional brain organoids have limitations, including incomplete maturation, and variations in cell maturity can occur, leading to less precise models. Furthermore, the lengthy growth process of organoids prompted the team to explore the more efficient 3D self-assembly approach.

Personalized Models for Tailored Treatments:

The self-assembled spheroids offer a remarkable advantage as they can be made from a patient's own skin cells. By reprogramming skin cells into iPSCs and converting them into brain cells, researchers can create patient-specific spheroids, providing individualized models for tailoring treatment options.

Advancing Therapeutics and Drug Approval Process:

Results from this investigation hold significant potential to improve therapeutics for neurological disorders and expedite the drug approval process. The FDA's increasing preference for human data over animal data may lead to more approvals based on non-animal disease modelling, and Axonis' engineered human tissue approach aligns perfectly with this direction.

Testing the Efficacy of Precision Gene Therapy:

Beyond spheroid formation, the investigation will also evaluate the effectiveness of a specialized gene therapy designed to target neurons specifically. The therapy utilizes a fluorescent protein that glows green upon reaching the cells. Understanding its success in reaching neurons will provide essential insights for potential precision medicine treatments.

Conclusion:

Axonis' pioneering research involves utilizing 3D self-assembled human brain models aboard the International Space Station (ISS) to advance treatments for neurological disorders. With the aim of propelling disease modelling to new heights, the team explores how microgravity impacts the maturation of brain cells, closely mimicking aspects of the human brain. This ground-breaking initiative not only holds immense promise for precision medicines but also embraces the potential to revolutionize neurological disorder treatments worldwide.

Guided by unwavering determination and compassion, Axonis ventures into the uncharted realm of space-based research. Their journey into the celestial laboratory of the ISS offers a unique opportunity to uncover the complexities of neurological conditions, armed with insights that transcend Earthly laboratories. By embracing the microgravity environment, their pioneering approach seeks to inspire a brighter future for millions afflicted by neurological disorders, as it paves the way for innovative treatments and fosters a shared victory for humanity's quest for understanding and healing.