From orbit to clinic: HIV drug enters Rett syndrome trial after space-based brain study
Research using human brain organoids onboard the International Space Station helped lead to a clinical trial of a potential therapy for Rett syndrome, a debilitating neurological disorder in children. Scientists are now expanding the space-based approach to study other brain diseases and find new treatments.
A clinical trial of a potential therapy for Rett syndrome, a childhood neurological disorder, has begun following groundbreaking research using human brain organoids aboard the International Space Station. Scientists observed that these organoids, grown from human stem cells, display molecular changes linked to aging and neurodegeneration sooner than Earth-based models.
In Rett syndrome models, researchers noticed dormant genetic elements activating, which triggered inflammation in brain support cells. This discovery prompted a test of lamivudine, an HIV antiretroviral drug, as a possible treatment to reduce this inflammation. The promising results have led to a Rett syndrome clinical trial underway.
Researchers aim to expand space-based studies to other brain diseases, seeking faster identification of potential treatments.
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