Overview

• A Science study shows that bone marrow transplantation–mediated microglia replacement stopped disease progression in eight ALSP patients for at least 24 months.
• Participants sustained stable MRI findings along with unchanged cognitive and motor function throughout the two-year follow-up.
• The approach takes advantage of patients’ CSF1R mutations to allow donor microglia engraftment without the need for pharmacological depletion.
• In CSF1R-mutant mouse models, microglia replacement achieved over 90% cell turnover, reversed myelin and axonal pathology, and improved motor and cognitive performance.
• Researchers are moving ahead with larger clinical trials and assessing the potential of microglia replacement therapy for conditions like Alzheimer’s disease.