A class of anti-cancer drugs may be able to prevent brain disorders, including Down syndrome and Fragile X syndrome, new research says. Down syndrome and Fragile X are the two most prevalent genetic causes of intellectual disabilities.
Down syndrome is caused by an extra copy of chromosome 21, while Fragile X is caused by a mutation in a single gene.
The researcher found that giving the leukemia drugs nilotinib or bafetinib to fly larvae with Fragile X-like symptoms prevented the wild overgrowth of cell problems associated with the disorder. “This study proposes a potential therapeutic approach for treating brain disorders associated with dysregulated expression of the Dscam protein, which is seen in both Down syndrome and Fragile X syndrome,” said senior study author Bing Ye from University of Michigan in the US.
In a series of experiments outlined in the study, the researchers showed that the Dscam protein activates another protein known as Abelson tyrosine kinase (Abl). The scientists then took genetically modified flies that produced high levels of Dscam and gave them the cancer drug, which acts by blocking the action of Abl. “Although there is an amazing amount of similarity between flies and humans, more study is needed before we’ll know if this could be a safe and effective treatment for human patients,” Ye noted.