Many drugs now taken by millions of people were discovered by high-throughput drug screening, a process in which scientists use robotics, data processing and control software, liquid handling devices and sensitive detectors to quickly perform millions of chemical tests until they find compounds that correct a biomolecular pathway that scientists believe is relevant to a disease. Now, the laboratory of Ed Gilmore, MD, PhD will begin to use funding from the A-T Children’s Project to apply this approach to ataxia-telangiectasia (A-T) and to discover drugs that treat the disease’s neurological problems.

Based at Case Western Reserve University in Ohio, Dr. Gilmore believes that when the A-T protein is missing, a certain type of naturally occurring DNA damage, called double-strand breaks, isn’t repaired properly, ultimately leading to the brain cell death and neurological problems faced by children with A-T. His team will therefore focus on isolating brain cells from mice that have been engineered to have A-T and then identifying compounds that prevent double-strand breaks in those cells.

Once Dr. Gilmore’s team identifies promising compounds, the A-T Children’s Project will quickly pursue the many subsequent steps required in drug discovery and development to advance effective and safe drugs that will slow or prevent the brutal neurological decline faced by patients.

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