Microglia Replacement Therapy Stops Progression of Rare Brain Disease

Speedy Summary

• Topic: Regenerative medicine research on Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia (ALSP).
• Publication: Nature Biotechnology, 12 August 2025, authored by Iris Marchal.
• Background: ALSP is a rare and fatal brain disease caused by mutations in the CSF1R gene expressed in microglia. Symptoms include myelin pathology, axonal swelling, and cognitive decline.
• Breakthrough Study:

– Wu et al. developed mouse models mimicking ALSP symptoms using specific CSF1R hotspot mutations.
– Healthy microglial replacement therapy was tested through two approaches:
– Bone marrow transplant after depleting mutated microglia via CSF1R inhibition.
– Bone marrow transplant without prior microglial depletion.
– Both methods showed improvements in neural signal transduction as well as cognitive and motor functions of the treated mice.

Indian Opinion Analysis

This study holds promising insights for treatment methodologies not just limited to ALSP but possibly other neurological disorders linked to microglial dysfunctions. The demonstrated efficacy of bone marrow transplantation provides a viable therapeutic option that can counter critical neurodegenerative conditions efficiently without necessitating pre-depletion steps in certain specific cases. From India’s viewpoint-given its growing investment into biotechnology research-this advancement underscores an opportunity for local researchers to explore similar regenerative therapies tailored to indigenous health challenges like genetic diseases or Alzheimer’s disease-related presentations reported among Indian populations.

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