GROUNDBREAKING! Lab-grown 'mini human brains' can help cure Alzheimer's, Parkinson's & other killer disease
Scientists have successfully cultivated miniature human brain models in the unique microgravity of space, paving the way for potential breakthroughs in treating Alzheimer's, Parkinson's, and spinal cord injuries.
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FollowIn a groundbreaking leap for medical research, scientists have successfully cultivated miniature human brain models in the unique microgravity of space, paving the way for potential breakthroughs in treating Alzheimer's, Parkinson's, and spinal cord injuries.
According to DailyMail, researchers, onboard the International Space Station (ISS), achieved in 72 hours what typically takes months on Earth. These brain organoids, artificially-grown cell clusters mimicking human brain structure and function, served as the perfect medium to test a revolutionary gene therapy technique.
Developed by Axonis Therapeutics, the novel treatment uses a reprogrammed virus—termed a "viral vector"—to deliver gene therapy directly to central nervous system (CNS) cells. This innovation holds promise for combatting some of the most debilitating neurological conditions.
Each year, nearly 600,000 Americans are diagnosed with Alzheimer's or Parkinson's, while approximately 18,000 suffer new spinal cord injuries. Testing effective treatments for these conditions has long been a scientific challenge. Gene therapies that succeed in mice often falter when applied to humans due to differences in complexity.
Earth-based cell cultures fail to replicate the three-dimensional organization of the human CNS due to gravity's limiting effects. By venturing into space, Axonis Therapeutics aimed to overcome this hurdle. "Doing research in space is not something you'd ever think about normally," remarked Shane Hegarty, co-founder and chief scientific officer of Axonis Therapeutics.
"But the opportunity to leverage microgravity conditions can unlock a lot of untouched potential by pushing the boundaries of science in a unique environment."
In August 2023, Axonis Therapeutics sent frozen vials containing viral vectors, mature neurons, and astrocytes—cells critical for neuron development—aboard a resupply mission to the ISS. Researchers onboard injected the materials into BioCell, a state-of-the-art cell-culturing system.
To validate the treatment's effectiveness, the team engineered the viral vector to carry a fluorescent protein gene. Success would be evident if the human neurons absorbed the gene, causing them to glow neon green.
Within 72 hours, the results exceeded expectations. Not only had the cells organized into three-dimensional brain organoids, but they also emitted a striking green fluorescence. "The pivotal day was when the crew was doing the microscopy, and we saw big aggregates of green, glowing 3D tissue," said researcher Sheila Nielsen of BioServe Space Technologies. "Shane and I were sending images of the organoids back and forth to each other, and we were pretty ecstatic because this is not something they had ever seen on the ground."
“It was very exciting—we were able to both validate the gene therapy targeting ability and show that we indeed rapidly formed 3D assembloid models of the human brain,” Hegarty explained.
Viruses, known for efficiently inserting genetic material into cells, are often reprogrammed by scientists to act as vehicles for beneficial therapies. Axonis' research demonstrated that such reprogrammed viral vectors could effectively target human neurons, potentially leading to revolutionary treatments for currently untreatable diseases.
"The major breakthrough is that you can self-assemble mature neurons and astrocytes rapidly into a model of the brain in microgravity conditions, which you can't do on the ground," Hegarty emphasized.
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