ANN ARBOR, Mich. A drug originally developed to stop cancerous tumors may hold the potential to prevent abnormal brain cell growth and learning disabilities in some children, if they can be diagnosed early enough, a new animal study suggests.
The surprising finding sets the stage for more research on how anti-tumor medication might be used to protect the developing brains of young children with the genetic disease neurofibromatosis 1 -- and other diseases affecting the same cellular signaling pathway.
The findings, made in mice, are reported in the journal Cell by scientists at the University of Michigan Medical School and their colleagues. The results are also important to understanding the stem cells that become different brain cells.
Neurofibromatosis 1, or NF1, affects one in every 3,000 children, and causes benign tumors to grow throughout the body, large head size and other issues. Many children with NF1 also struggle with learning to read, write, do math and behave well.
This impact on brain function is considered the most common serious issue caused by NF1, and often appears before other symptoms, except for brown patches on the skin that are often mistaken for birthmarks. But while the tumors that erupt mostly later in life have been well-studied, NF1's effect on brain function isn't understood.
In the new paper, the team studied neural stem cells a kind of master cell that can become any type of neural tissue. In newborn mice with two copies of the genetic mutation that causes NF1, neural stem cells in a key area of the brain were far more likely to produce a kind of "helper" nerve cell called glia. They produced far fewer cells called neurons, which send and receive crucial signals in the brain and body.
The scientists then took aim at this abnormal cell growth by giving the mice an experimental drug that has already been used in clinical trials for advanced cancer. Called PD0325901,
|Contact: Kara Gavin|
University of Michigan Health System