Pilot Study of Biomarkers for SMA Announced

The Spinal Muscular Atrophy (SMA) Foundation has announced the launch of a pilot study of Biomarkers for Spinal Muscular Atrophy (BforSMA).

The Spinal Muscular Atrophy (SMA) Foundation has announced the launch of a pilot study of Biomarkers for Spinal Muscular Atrophy (BforSMA). The Foundation is a nonprofit organization dedicated to accelerating the development of a treatment for SMA.

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.

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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 project.

"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.

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