CORE Update: Drug Discovery Progress at the Kirchhoff Family Drug Screening Unit

The core of THE CORE—its primary drug assessment platform—is the Kirchhoff Family Drug Screening Unit, where CORE researchers develop new cellular models of ALS, and screen thousands of drug candidates on these ALS patient-derived laboratory models. In its first two years, the team has:

  • Tested >1700 chemical compounds and FDA approved drugs for other indications in our novel first-generation “ALS in a Dish” model, which uses motor neurons developed from ALS patient blood and skin samples
  • Optimized two of these compounds—prosetin and alsterpaullone­—for use in ALS
  • Moved 6 top performing compounds to further assessments, including blood brain barrier penetrance, pharmacokinetics, and effectiveness in other ALS models
  • Collaborated with >20 other academic groups and biotechs to assess their ALS compounds of interest
  • Developed a next-generation, barcoded “ALS in a Dish” model that will allow for rapid drug screening on multiple ALS sub-types at the same time

The CORE’s drug screening unit has a dual goal: to make new laboratory models that will better predict how a potential drug will impact an actual person with ALS, and to rapidly screen compounds on them. It takes its name, and its mission, from the Kirchhoff family, who have driven ALS drug discovery efforts since Tom Kirchhoff was diagnosed with ALS in 2010. From the start, the Kirchhoff family pushed Project ALS researchers to apply their basic science findings toward meaningful ALS drugs—and to move promising therapies to patients faster.

“My family decided to support drug discovery efforts because we feel as if it is a big part of the future of Project ALS. We were fortunate enough to visit the Core last summer and were super impressed and proud of the work that was going on. The team at the Core is incredibly intelligent and we could not think of another group better for the job!” – Bryn Kirchhoff

Tom passed away from ALS in 2015, but his wife Staci and children Brynley, Sam, Tommy, and Ty, are devoted to inspiring and funding Project ALS drug discovery efforts so that in the future, ALS patients will have meaningful treatments, and families will be able to fight back against this disease.

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