How do we get better clinical trials? Better “preclinical” research.

If you weren’t familiar with the term “clinical trials” before Covid-19 rocked our worlds, for sure you’ve heard it by now. Lesser known — but equally important — is the work that comes before an experimental vaccine or medicine is ready to be tested in humans. Improving how we choose which drugs go into clinical trials is critical: better preclinical research could save many years, millions of dollars, and even lives. In 2016, we launched an initiative called ‘Of Mice and Measures’ to tackle this very problem.

As part of this initiative, in January 2020, we launched a study focused on gathering new data to improve how we use two particularly important models in DMD. The ultimate goal is that improved work at the preclinical level will mean more successful clinical trials for DMD treatments, as ineffective treatments should be identified earlier in the research process. Charley’s Fund, Duchenne UK, Michael’s Cause, Pietro’s Fight, and Ryan’s Quest pitched in to provide support, and two leading Duchenne labs (one in the Netherlands and one in Italy) began the work. Then came Covid.

The research suffered a brief delay, but thanks to our tremendous teams it did not stop and is now back on track. Our scientists worked through closed borders, unprecedented lab restrictions, remote training requirements, and increased material costs to keep the science moving. We just reviewed a midway check-in report, and the results already offer insights that will enhance how our research community chooses which drugs to advance to human testing.

Before we share conclusions, we need to wait for the full dataset and analyses. In the meantime, we want to assure you that the work continues despite the crazy hurdles that 2020 threw our way. And we want to say thank you to Annamaria, Maaike, Paola, Annemieke, and their dedicated teams of consummate scientists for forging on through Covid and continuing to fight for our boys, men, and rare girls living with Duchenne.

To read more about the study and its goals, check out our original blog post here: