2016 – 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.

Oops! We could not locate your form.

Related News

Feb 22 @ 5:17 pm

Looking Forward to Research Advances in 2024

Dear Friends, As 2023 is wrapping up, we want to thank all of you who support the A-T…

more
Feb 22 @ 5:17 pm

A-T Biomarker Development Initiative Now Recruiting

On December 18, the A-T Children’s Project sent out the below email to A-T parents in the United…

more
Feb 22 @ 5:17 pm

A-T Caregiver Weekend 2024 Announced

On December 15, the A-T Children’s Project sent the below email to parents inviting them to join us…

more
Feb 22 @ 5:17 pm

Giving Tuesday Update 2023

It’s Giving Tuesday, and anonymous donors have joined together to match the first $60,000 in donations we receive!

more