Access to treatments

Latest updates on Of Mice and Measures: improving data and decision-making in DMD preclinical research

In 2020, Duchenne UK joined with US-based charity Charley’s Fund to improve decision-making in DMD preclinical research.

In recent years, the Duchenne muscular dystrophy (DMD) field has seen an increasing number of medicines being tested in clinical trials. Selecting which treatments to advance to human testing is a critical decision, especially given DMD’s aggressive progression and urgent unmet medical need, on top of the recruitment challenges faced by rare disease populations.

Currently, however, there is a degree of variability and inconsistency in preclinical research practices. There are also many unknowns, as we continue to develop new models and measurement techniques. This makes this decision-making process harder.

Together, Duchenne UK and Charley’s Fund provided a $314,000 USD grant to the labs of Professor Annemieke Aartsma-Rus and Dr. Maaike van Putten (Leiden University Medical Center) and Professor Annamaria De Luca (University of Bari) for a study to identify new and updated best practices in the use of two mouse models of Duchenne when evaluating potential new drugs.

You can read the latest project update from Charley’s Fund below

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 Italy and one in the Netherlands) 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