Alec DeSimone, PhD, currently a postdoctoral researcher at Yale School of Medicine, was the winner of the Best Poster Award at the 2021 International Research Congress, held virtually on June 24-25. The title of his poster “A live-cell drug screening platform for FSHD therapeutics,” co-authored with Drs. Angela Lek and Monkol Lek, describes a cost-effective, higher throughput, and clinically relevant drug-screening platform for use in research.
The platform combines a physiologically relevant system (patient-derived muscle cells), sensitive detection tools (DUX4-reporters), along with time-lapse video imaging to track DUX4 expression and the downstream consequences of it, such as cell death. Because DUX4 expression is not only a rare even but also difficult to detect, his system incorporates a sensitive DUX4 activity visualization tool, that when combined with video imaging, can detect rare DUX4 activation in a population of muscle cells over time. This readout can also be combined with other measures that monitor muscle cell health to track what happens after DUX4 is activated. Another advantage of the system is that it is sufficiently miniaturized to allow simultaneously track up to 96 experiments at one time. This “high-throughput” platform is amenable for automation and is the preferred platform for pharmaceutical companies, allowing them to efficiently test different drugs that may stop DUX4 from damaging muscles. This poster presentation now makes this approach accessible to other academic groups interested in screening drugs and other therapeutic approaches to treat FSHD.
Congratulations to Dr. DeSimone!