Biomarker Could Offer More Precise Way to Track Treatment Efficacy, Reduce Clinical Trial Costs and Speed Drug Development for DMD
CAMBRIDGE, Mass., Oct. 10, 2012 /PRNewswire/ -- DART Therapeutics, LLC, an innovative, new-model biotechnology firm focused on developing therapies for Duchenne Muscular Dystrophy (DMD), announced today the successful completion of a study to evaluate a promising new biomarker for DMD. The biomarker, electrical impedance myography (EIM), is a non-invasive and simple-to-use technique that allows the operator to measure the health of a muscle and track its changes over time. The information can help researchers evaluate how well a treatment is working to halt disease progression, which could support more precise drug therapy for DMD boys and faster, less expensive drug development for the disease overall.
DMD is a progressive and fatal neuromuscular disease for which there is no effective treatment. Current drug development efforts rely on patient outcomes measurements that are either invasive and poorly tolerated or highly subjective and prone to variability. This can have a negative impact on the value of the studies, which must generate results that can be measured and reproduced over time. EIM is based on the observation that as a muscle becomes progressively diseased, an applied weak electrical current moves through it differently. The technique could provide drug developers with a highly precise way to measure the effectiveness of experimental therapies earlier, potentially reducing the time and cost of clinical trials and propelling the availability of treatments for DMD.
"EIM could be a promising new biomarker for DMD," said Julie A. Parsons, Associate Professor of Pediatrics and Neurology at the University of Colorado School of Medicine. "As with other neuromuscular diseases, the six-minute walk or climbing-four-stairs tests, which are standard methods used to track muscle health in DMD boys, can produce highly varied results from one patient encounter to the next. In our experience doing the study, EIM is quick, painless and very well tolerated, even by three- and four-year old boys. From a clinician's perspective, the technology is exceedingly intuitive. The ability to track muscle health over time in a manner that is easy on the boys as well as easy for clinicians could have a very positive impact on the way DMD research is done moving forward."
Researchers conducted the prospective study in 92 boys (61 DMD and 31 healthy boys) at five U.S. medical centers, including Children's Hospital Colorado (Aurora, Colorado), Massachusetts General Hospital (Boston, Massachusetts), Washington University (St. Louis, Missouri), Cincinnati Children's Hospital (Cincinnati, Ohio) and Seattle Children's Hospital (Seattle, Washington). The study began enrollment in February 2012 and was complete in August 2012. Additional detail is available at clinicaltrials.gov using study identifier NCT01523964.
"Our model allows us to invest in research that could benefit the entire DMD community, not just DART's pipeline," said Gene Williams, CEO of DART Therapeutics. "EIM has the potential to benefit not only DMD research but also that belonging to the entire field of neuromuscular diseases, which has long needed more precise outcome measurements to speed drug development and reduce costs. EIM could be an important tool in developing therapies for DMD and moves us closer to our goal of making DMD a chronic, manageable disease. We plan to use it in our future trials and hope others can benefit as well."
There is a long history of measuring electrical impedance to provide valuable information across a variety of medical and non-medical applications. Dr. Seward Rutkove, Chief of the Division of Neuromuscular Disease at Beth Israel Deaconess Medical Center and Professor of Neurology at Harvard Medical School, first used EIM to measure localized muscle changes associated with neuromuscular disease in ALS (Amyotrophic Lateral Sclerosis). In February 2011, Dr. Rutkove received the $1M ALS Biomarker Prize for his discovery of EIM as a biomarker for ALS from Prize4Life, an organization that aims to accelerate a cure for ALS by offering incentives to drive innovation. In the study supporting Dr. Rutkove's award application, EIM demonstrated the potential to reduce the cost of clinical phase 2 ALS trials by 50 percent or more.
DART's study results inform algorithm development for an investigational EIM device conceived by Convergence Medical Devices, a Woburn, Massachusetts-based firm Dr. Rutkove co-founded. CMD has already developed a portable, hand-held, non-invasive device that a physician or researcher can place over a muscle to evaluate its health. When applied, the device delivers an imperceptible current through the muscle. With the data gathered here, a highly intelligent algorithm captures and processes the data, which could include more than 1,000 different data points in a single measurement. The device then provides the physician or researcher with a score that reflects the health of the muscle and the overall status of the child. Researchers can track scores over time to evaluate the effect of a drug on one or more muscles. Physicians can use the information to help DMD boys and their families better understand disease progression and tailor treatment. CMD expects to submit a 510k application to the Food and Drug Administration in 2013.
About Convergence Medical Devices
Based in Woburn, Mass., Convergence Medical Devices, Inc. is an early-stage medical device company dedicated to supporting early, effective care for neuromuscular disease through the development of a non-invasive technology for quantitatively monitoring muscle health. Convergence Medical Devices brings together clinical, technical and commercial expertise with the goal of moving electrical impedance myography from the research stage into regular clinical practice. Based on more than a decade of clinical studies, the company may help drug developers and researchers reduce the cost and time associated with conducting clinical trials, and may someday help clinicians more effectively manage their patients. For more information, please visit www.cmdevices.com.
About DART Therapeutics
DART Therapeutics, LLC, is a biotechnology firm applying a new model for drug development to pediatric rare diseases. In the DART model, patient foundations join with biotechnology industry veterans to impact a central problem in pediatric rare diseases: Rapid therapy development. Co-founded and funded by Patient Foundations Charley's Fund and the Nash Avery Foundation, DART is focused on Duchenne Muscular Dystrophy, a fatal neuromuscular disease for which there is no effective treatment. For more information, please visit www.dartrx.com.
Shanti Skiffington, principal
Samvega Public Relations
SOURCE DART Therapeutics