WALLOPS FLIGHT FACILITY (VA), July 25, 2023 – Nearly one in six people around the world—which amounts to approximately one billion people—are affected by neurological disorders ranging from Alzheimer’s and Parkinson’s disease to epilepsy and migraines. Researchers from biotechnology startup Axonis are working to help improve treatments for patients with such disorders by leveraging the International Space Station (ISS) National Laboratory. The research team will examine how microgravity affects the maturation of human brain cells that form three dimensional spheroids that mimic certain aspects of the human brain. Findings will help advance disease modeling and could lead to the development of new therapies to treat neurological disorders in patients on Earth.
Axonis was awarded a grant for this project through the Technology in Space Prize, funded by Boeing and the Center for the Advancement of Science in Space, Inc., manager of the ISS National Laboratory, in partnership with the MassChallenge startup accelerator program.
In the investigation, which is launching on Northrop Grumman’s 19th Commercial Resupply Services mission, the research team will convert induced pluripotent stem cells (iPSCs) into different types of brain cells—neurons, microglia, and astrocytes—on Earth. The team will then then send cultures of these cells to the orbiting laboratory, where the various cell types should assemble into three-dimensional spheroids. These spheroids act as models for the brain that can be used for disease modeling and drug testing.
According to Shane Hegarty, Axonis chief scientific officer, these 3D self-assemblies are a game-changing approach to studying the human brain. “They’re assembling together to form this kind of spheroid rather than starting as one cell growing, growing, growing until you get an organoid, which is a little bit different and can’t get very mature,” he said.
Hegarty explained that the trouble with organoids is that the cells can only mature so much, and oftentimes, part of them dies more quickly than the rest, which leaves researchers with an incomplete model. Brain organoids also take a long time to grow, and this experiment seeks to streamline that process. “Organoids take many months to create on Earth, and you might never get the maturity that you could potentially get with the 3D self-assembly approach,” Hegarty said.
The self-assembled spheroids are also valuable because they can be made from a patient’s own skin cells. Skin cells from a patient can be reprogrammed into iPSCs that are then converted into brain cells that self-assemble into spheroids. Because the spheroids are made from a patient’s own cells, they can serve as individualized models that allow researchers to tailor treatment options to the particular patient’s needs.
Results from this investigation could not only improve therapeutics for neurological disorders but also help speed up the drug approval process, Hegarty said. “The U.S. Food and Drug Administration (FDA) has decided that human data is preferable to animal data, so in the future, we could see more and more approvals based on nonanimal disease modeling,” he said. “This experiment could help with that, as it uses engineered human tissue as opposed to rodent models.”
In addition to evaluating how well self-assembled spheroids form in space, the investigation will also test the ability of a therapeutic to reach cells within the assembly. The team is using a special gene therapy that consists of a fluorescent protein that glows green when it reaches cells. The therapy is designed to target only neurons, so when the payload returns to Earth, Hegarty and the team will evaluate how well the therapeutic reached neurons and no other cell type.
The mission is targeted for launch from Wallops Flight Facility no earlier than August 1 at 8:30 p.m. EDT. This mission will include more than 20 ISS National Lab-sponsored payloads. To learn more about all ISS National Lab-sponsored research on this mission, please visit our launch page.
Download a high-resolution photo for this release: 3DI Neuronal Cells Cultured in Microgravity
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About the International Space Station (ISS) National Laboratory: The International Space Station (ISS) is a one-of-a-kind laboratory that enables research and technology development not possible on Earth. As a public service enterprise, the ISS National Lab allows researchers to leverage this multiuser facility to improve life on Earth, mature space-based business models, advance science literacy in the future workforce, and expand a sustainable and scalable market in low Earth orbit. Through this orbiting national laboratory, research resources on the space station are available to support non-NASA science, technology and education initiatives from U.S. government agencies, academic institutions, and the private sector. The Center for the Advancement of Science in Space (CASIS), Inc. manages the ISS National Lab, under Cooperative Agreement with NASA, facilitating access to its permanent microgravity research environment, a powerful vantage point in low Earth orbit, and the extreme and varied conditions of space. To learn more about the ISS National Lab, visit website.
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