A representative culture from a brain organoid in which the gene MECP2 — causative in Rett syndrome — has been “knocked out,” as shown through a fluorescent microscope. Because the culture was treated with experimental cancer drug ADH-503, new synapses formed. Credit: Muotri Lab / UC San Diego Health Sciences New research from the University of California San Diego suggests that an experimental cancer drug may improve cognitive function in individuals with Rett syndrome, a rare condition associated with autism.
This discovery holds potential for developing treatments for other neurological disorders. The findings, published July 25 in Stem Cell Reports , highlight the role of microglia — a type of white blood cell found in the central nervous system — in the formation of the human brain. While such cells have been better studied in neurodegenerative disorders like Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), and multiple sclerosis, “very little information has existed on their role in early stages of neural development” because access to fetal tissue is limited, said Pinar Mesci, Ph.
D., the study’s lead researcher. Now employed elsewhere, she completed work on the project while at the university.
In a bid to better understand their function, Mesci instead used brain organoids — “mini-brains,” essentially, that mimic the developing brain of an embryo — grown from skin-derived stem cells of consenting patients. Such organoids were create.