Innovative Stem Cell Microtissues Enable Personalized Drug Testing for Neurodegenerative Diseases

Researchers have developed advanced microtissues that mimic the human spinal cord using stem cell technology, offering promising tools for studying neurodegenerative diseases like amyotrophic lateral sclerosis (ALS). These 'spinal cord-like' microtissues are created from stem cells derived from individual patients, enabling personalized testing of disease mechanisms and potential treatments.

ALS is a rapid-progressing neurodegenerative disorder characterized by the loss of motor neurons in the spinal cord, leading to muscle paralysis and ultimately, death within an average of three years. The molecular causes of ALS remain unclear, but neuroinflammation—a harmful overactivation of immune responses within the spinal cord—is believed to play a key role in motor neuron death.

To better understand this process, scientists from the Memorial Sloan Kettering Cancer Center, led by Elisa Giacomelli and Lorenz Studer, generated spinal microtissues containing both motor neurons and immune cells from stem cells. Their study, published in Stem Cell Reports, revealed that microtissues derived from ALS patients secreted higher levels of inflammatory proteins compared to those from healthy individuals. Over time, these ALS microtissues also showed progressive motor neuron death, accurately recreating key features of the disease.

Furthermore, the team tested 190 FDA-approved drugs on these microtissues to identify potential treatments. They discovered that a specific class of drugs could reduce inflammatory protein levels in ALS microtissues to those observed in healthy tissues, which correlated with decreased motor neuron loss. These findings suggest that these microtissues could serve as scalable platforms for high-throughput drug screening and for studying patient-specific neuroinflammatory processes in ALS.

This innovative approach opens new avenues for understanding neurodegeneration and developing targeted therapies, bringing hope for more effective treatments for ALS and similar conditions.

Source: https://medicalxpress.com/news/2025-06-stem-cell-derived-spinal-cord.html