AANEM News Express

AANEM News Express

What’s Happening in the Foundation Research?


Report on the AANEM-MDA Development Grant

by K. Ming Chan, MD, FRCP(C), Research Committee Chair

The AANEM Foundation first started the co-funded development grant competition with the Muscular Dsytrophy Association (MDA) in 2015. These applications are well vetted. In addition to being highly vigorous, the adjudication process also gives the AANEM a chance to select proposals that are well aligned with the goals and interests of our organization. The winners, among a highly selected group of young investigators in advanced stages of training, are selected on the basis of showing great promise to become outstanding independent scientists. Unfortunately, under the current restrictive funding climate, reality is that even great proposals from bright individuals would often go unfunded. The co-funding arrangement from the AANEM Foundation therefore fills an important gap in allowing these individuals a chance to pursue these projects and to demonstrate their capability.

At the December 2016 AANEM-MDA Development Grant competition, the recipient was Mattia Quattrocelli from Northwestern University in Illinois.  The proposal “Glucocorticoids in Fibre Repair and Regeneration in Dystrophic Mice ” has since come to fruition and has just been published in a highly regarded peer reviewed journal – Journal of Clinical Investigation. Apart from showing highly convincing results in a well-designed experiment using cutting edge molecular tools, the findings also have important clinical implications to patients with Duchenne muscular dystrophy. Although steroid has been a commonly used drug in Duchenne patients for many decades, major side effects have limited its use. This study has investigated in depth the underlying mechanisms of the effects of steroid on muscle fibre repair in mice. The vigorous basic science investigations provide much needed evidence to support its use and on how the negative impacts of the treatment can be minimized.  With increasing emphasis on evidence based medicine and best practice, this is invaluable. With the relatively small amount of funding needed to support a junior investigator, the potential value for return of investment is great. The funding would go much further than just supporting a single project. It also provides the opportunity for the trainee to establish a track record and to use it to attract additional funds. If successful, they would likely stay in the field in years to come and continue to be valuable contributing members. The outcomes of this funded project well illustrate the potential impacts of the co-funded AANEM-MDA Development Grant.

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