The Muscular Dystrophy Association (MDA) and The ALS Association today announced a joint commitment to provide funding for a phase 2 clinical trial that will test efficacy of the NeuRx Diaphragm Pacing System (DPS) in people with amyotrophic lateral sclerosis (ALS).
ALS (also known as Lou Gehrig's disease) attacks the nerve cells that control muscles, ultimately resulting in paralysis of all voluntary muscles, including those used for breathing. Average life expectancy for people with the disease is three to five years after diagnosis; respiratory failure is the most common cause of death.
The NeuRx Diaphragm Pacing System, developed by Synapse Biomedical in Oberlin, Ohio, requires minimally invasive laparoscopic surgery, in which electrodes are surgically implanted in the diaphragm (the main muscle used in breathing). The device activates the diaphragm, supplementing breathing ability and possibly delaying diaphragm shrinkage (atrophy).
The system received approval from the U.S. Food and Drug Administration (FDA) on Sept. 29, 2012, as a "humanitarian use device" (HUD) for the treatment of chronic hypoventilation (inadequate breathing) in ALS. This type of approval is given by the FDA to medical devices intended for use in rare diseases when there is no proof that the device is beneficial but little evidence that it is harmful. The FDA requires sufficient evidence that HUD devices do not pose "an unreasonable or significant risk of illness or injury, and that the probable benefit to health outweighs the risk of injury or illness from its use."
"The DPS is available commercially for humanitarian use, but although Synapse has presented data suggesting the device works, there is no definitive proof that it is effective," said Sanjay Bidichandani, M.B.B.S., Ph.D., MDA vice president of research. "Now MDA and The ALS Association have come together to fund a trial that is expected to determine whether the DPS can cause a significant improvement in respiratory function and improve quality of life for people with ALS."
MDA awarded a grant totaling $750,000 over three years to support the new trial. The award was made through MDA Venture Philanthropy (MVP), the drug development arm of MDA's translational research program. The ALS Association has committed $450,000 over three years through its Translational Research Advancing Therapies for ALS (TREAT ALS™) program. Synapse Biomedical also is providing support for the trial.
"The ALS Association is very pleased to be partnering with MDA on this important study to establish whether diaphragm pacing is beneficial in people with ALS, improving quality of life and increasing survival," said Lucie Bruijn, Ph.D., chief scientist of The ALS Association. "We are also pleased to be using the TREAT ALS/NEALS Network of clinical centers to facilitate the research."
Kirsten Gruis, M.D., associate professor of neurology at SUNY Upstate Medical University in Syracuse, N.Y., will be the principal investigator for the trial, and Jonathan Katz, M.D., director of the neuromuscular diseases program at California Pacific Medical Center in San Francisco, will serve as co-principal investigator.
The phase 2 trial is expected to enroll 180 people with ALS. Of that number, 120 will receive the DPS, and 60 will receive treatment in line with the current standard of care. Trial investigators will compare survival between the standard-of-care (control) and DPS groups. In addition, the investigative team will compare measures of diaphragm function, dyspnea (shortness of breath) and quality of life over time between the two groups.
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