Newswise — An experimental cancer drug could make thinking easier for individuals with Rett syndrome, a rare disorder linked to autism, according to new research from the University of California San Diego — a discovery that could lead to therapies for patients with other neurological conditions. 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 created from individuals with Rett syndrome — a disorder primarily found in females that features loss of speech, purposeful use of hands, mobility and muscle tone, among other symptoms — as well as from neurotypical individuals. Mesci then added healthy microglia to the Rett syndrome brain organoids and found that .