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Join Our Research Efforts

 

Thank you for visiting our poster “Insights and ongoing efforts from Of Mice and Measures, a collaborative project to improve preclinical methodology in Duchenne muscular dystrophy” at MDA 2020. Our poster outlines a project to improve pre-clinical research in DMD. It also highlights an effort to bring together two discrete research initiatives — one preclinical, one clinical — to explore a unique opportunity to evaluate therapeutics for translational potential on the preclinical-clinical continuum. You can download a copy of the poster here.

Charley’s Fund works with leading labs and researchers around the world to improve our tools to conduct preclinical and clinical research in Duchenne muscular dystrophy. Electrical impedance myography and ‘Of Mice and Measures’ are two of these efforts. Read more here about each project. We hope you will get involved. Contact Charley’s Fund President and COO Laura Dalle Pazze at laura[at]charleysfund.org to do so.

 

Of Mice and Measures

Optimizing preclinical tools and methods to evaluate therapeutic candidates is critical to improve decision-making about advancing therapies to clinical testing. Ongoing since 2016, Of Mice and Measures (“OMAM”) is a collaborative, ongoing effort comprising more than 20 partners from the academic, industry, and nonprofit worlds to improve the decision-making process for which drugs should advance to clinical testing. Charley’s Fund serves as a central coordinating party and works collaboratively with a Scientific Organizing Committee and contributing partners to identify opportunities, develop strategies, convene contributors, and undertake action steps. An initial October 2017 workshop in Paris provided a key grounding step, with an initiating focus on establishing SOPs for the emerging D2.B10-Dmdmdx/J (D2/mdx) mouse model as well as updated SOPs for the classic C57BL/10ScSn-Dmdmdx/J (Bl10/mdx) mouse model. And in pursuit of a tool with the potential to sensitively and quantitatively evaluate muscle condition and response to therapy across both preclinical and clinical testing, 3 universities, a medical device company, and Charley’s Fund have developed a plan to incorporate and validate the potential of a technology called EIM in the natural history study

A published workshop report summarizes findings and next steps, one of which is the initiation of the well-powered 52-week longitudinal replicated study referred to in our poster above in the two strains, enabling the identification of key pathological strain and disease-related differences so as to improve future preclinical drug development. This study will launch Q4 2019.

Opportunities to get involved:

  • Review and begin applying best practices highlighted in the initial workshop findings
  • Contribute data from Bl10/mdx or D2/mdx mouse model studies to our ongoing analyses
  • Speak with us about mouse model bio sample access and analyses planned in our upcoming natural history study

Additional references:

 

Electrical impedance myography (“EIM”)

Improved methods for assessing disease progression and response to therapy in Duchenne muscular dystrophy (DMD) are greatly needed to improve the speed, sensitivity, and accessibility of clinical trials. EIM is a painless, non-invasive, and relatively inexpensive technology that holds promise for application at any stage of disease. In EIM, a weak, high frequency electrical current is applied across two electrodes and surface voltages are measured with a second set of electrodes overlying a muscle of interest.

EIM has been studied successfully in boys with DMD and shown correlation to functional measures, change over time, and response to corticosteroid initiation. Additional highlights include:

  • Fast, easy-to-use (and train users), painless
  • Growing body of evidence for utility across age, stage of disease
  • Growing body of evidence suggests sensitivity to disease progression and treatment effects (showing changes before/after initiation of steroids)
  • Early but potentially interesting data on sensitivity to muscle fatigue with activity
  • Comparatively inexpensive
  • Application and data in other numerous neuromuscular indications (SMA, ALS, FSHD, CMD, and disuse atrophy) support promising outlook

Charley’s Fund seeks partners to integrate EIM into their clinical trials as an exploratory measure — contact Laura at laura[at]charleysfund.org to learn more 

Additional references:

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