Could MRI be better than muscle biopsy in measuring disease progression and therapy effectiveness in Duchenne muscular dystrophy?
The use of magnetic resonance imaging (MRI) as an assessment tool in boys with Duchenne muscular dystrophy (DMD) is being studied by former MDA grantee Krista Vandenborne, through a $7.5 million grant from the National Institutes of Health (NIH).
The goal of the study is to assess whether MRI technology can be used as a precise, noninvasive measure of muscle tissue, gauging both disease progression and the effectiveness of therapies tested in children with DMD.
The NIH grant was based on evidence from an earlier, MDA-supported study that suggested MRI boasts a number of advantages over traditional muscle biopsies, which are invasive and provide researchers with an incomplete view of the muscle tissue.
About the study
The NIH grant was awarded based in part on preliminary data from an MDA-supported study completed in December 2009 by Vandenborne, professor and chair of the department for physical therapy at the University of Florida in Gainesville.
The MDA-funded study initially focused on the development of MRI and spectroscopy [another type of imaging] strategies to monitor dystrophic tissue in mouse models of DMD and limb-girdle muscular dystrophy (LGMD). Vandenborne and her team then used that preclinical data to establish techniques designed to evaluate muscle damage and disease progression in boys with DMD.
Vandenborne will be the lead investigator for the NIH study, which will include three sites in addition to the University of Florida: Children's Hospital of Philadelphia; the University of Pennsylvania, also in Philadelphia; and Oregon Health and Science University in Portland.
Investigators at the four study centers will conduct MRI measurements of muscle in 100 boys with DMD, ages 5 though 14, over a five-year period.
Meaning for people with DMD
Results of the study are expected to provide researchers with clear guidelines for how MRIs should be performed in DMD to monitor the progression of the disease.
Importantly, the use of magnetic resonance imaging in routinely and reliably evaluating muscle tissue may help facilitate and speed clinical trials.
"The inclusion of MRIs in clinical trials is likely to make it easier for boys to participate in studies, as muscle biopsies can be avoided," Vandenborne said. In addition, the MRIs should "provide additional information about the disease process that may or may not be gained from strength or functional tests."