The goal of this study is to identify a potential biomarker
or panel of biomarkers that can be used as objective measures of SMA disease
severity. The identification of such biomarkers may help to assess drug
efficacy and shorten the duration of clinical trials.
To ensure wide outreach and quick completion of the study,
18 clinical investigators have been recruited to participate in BforSMA. The
first patient is expected to enroll in October. The study aims to enroll 100
children with SMA, making this one of the largest research efforts in SMA
history. The Foundation has also recruited the New England Research Institutes
(NERI), BG Medicine and Expression Analysis as research partners in this
"The SMA Foundation is taking a bold initiative to
identify markers of disease severity in spinal muscular atrophy," noted Richard
Finkel, MD, a principal investigator for the trial at the Children's Hospital
of Philadelphia. "The team of SMA clinicians is eager to partner with
experienced industry pioneers, including BG Medicine and NERI, towards
realizing this goal. Having such biomarkers will greatly enhance the
development of new and novel therapies for this disabling disorder."
Cynthia Joyce, Executive Director for the SMA Foundation,
added, "This pilot project has the potential to be a landmark study for
therapeutics development - rapid completion will help make future clinical
trials more efficient and more informative.