New Hope for Rett Syndrome: Experimental Cancer Drug Shows Promise
Researchers at the University of California San Diego have discovered that an experimental cancer treatment might help people with Rett syndrome, a rare disorder linked to autism, think more clearly. This breakthrough could lead to new medicines for other neurological conditions.
Understanding Rett Syndrome
Rett syndrome is a disorder mostly found in females, causing loss of speech, hand use, mobility, and muscle tone. It is linked to problems with the MECP2 gene, which affects brain development.
The Role of Microglia
Microglia are white blood cells in the central nervous system that act like ‘janitors,’ cleaning up the brain by removing foreign substances and dead cells. They also help optimize brain function by ‘pruning’ synapses, where neurons connect and communicate.
Research Findings
Lead researcher Pinar Mesci, PhD, used ‘mini brains’ grown from stem cells to study Rett syndrome. She found that adding healthy microglia to these mini brains improved synapse function. This was due to the restoration of phagocytosis, the process by which microglia clean up the brain.
Experimental Drug ADH-503
The team tested various drugs and found that ADH-503, an experimental cancer drug, could restore phagocytosis in microglia. This drug, also known as GB1275, is a regulator of CD11b, a protein involved in the cleaning process.
Future Implications
Senior author Alysson Muotri, PhD, and other researchers believe this discovery could lead to new treatments for Rett syndrome and other neurological disorders. Jonathan Kipnis, PhD, from Washington University, praised the study for highlighting microglia as a potential therapeutic target.
Mesci hopes this research will improve the quality of life for patients with Rett syndrome and other similar conditions.
Doubts Revealed
Rett Syndrome -: Rett Syndrome is a rare genetic disorder that affects brain development, causing severe physical and mental disabilities. It mostly affects girls.
Experimental Cancer Drug -: An experimental cancer drug is a new medicine that scientists are testing to see if it can treat cancer. In this case, they are testing it for Rett Syndrome.
ADH-503 -: ADH-503 is the name of the experimental drug being tested. It was originally made to treat cancer but might help with Rett Syndrome.
University of California San Diego -: The University of California San Diego is a big school in the United States where people study and do research. Scientists there are working on this new drug.
Microglia -: Microglia are special cells in the brain that help keep it healthy. They are like tiny cleaners that help the brain work better.
Synapse -: A synapse is a tiny gap between nerve cells in the brain where signals pass through. It’s important for thinking and moving.
Neurological conditions -: Neurological conditions are problems with the brain, spine, or nerves. They can affect how you think, move, or feel.
