Preclinical Study from Children’s Hospital of Philadelphia Highlights Innovative Approach to Replacing Brain Cells in the Treatment of Leukodystrophies

Research shows potential for brain-specific replacement over Hematopoietic Stem Cell Transplantation, which impacts the entire immune system

Excerpt from the Press Release:

PHILADELPHIA, April 30, 2025 /PRNewswire/ — Researchers at Children’s Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania announced the first ever direct approach to the depletion and replacement of microglia, the native immune cells of the central nervous system (CNS). The approach leverages microglial replacement in Krabbe disease to demonstrate its broader therapeutic potential across other neurological conditions. The preclinical findings were published in the journal Immunity.

Microglia provide an essential function to the nervous system, acting like tiny cleaners responding to the site of an injury or infection by removing germs or damaged cells to help protect the CNS and keep it healthy. Unfortunately, specific diseases cause microglia dysfunction, which can lead to issues like impaired development, increased susceptibility to infections and difficulties in repairing brain injuries. However, enthusiasm for therapeutically treating microglia is rapidly growing among researchers and clinicians as more research validates the key role they play in disease.

In recent research, clinicians indirectly targeted microglia with hematopoietic stem cell transplantation (HSCT) in Krabbe disease, also named Globoid Cell Leukodystrophy (GLD). This severe leukodystrophy, caused by galactosylceramidase (GALC) mutations, primarily affects infants and young children, causing symptoms such as irritability, feeding difficulties, seizures, muscle stiffness and developmental delays.

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