CMD co-founder receives Prize4Life award for discovery of new ALS biomarker

Convergence Medical Devices (CMD) today announced that its co-founder, Dr. Seward Rutkove, Chief of the Division of Neuromuscular Disease at Beth Israel Deaconess Medical Center and Associate Professor of Neurology at Harvard Medical School, received Prize4Life's prestigious award for the discovery of a new biomarker for ALS (Amyotrophic Lateral Sclerosis), also known as Lou Gehrig's disease. Cambridge, Mass.-based Prize4Life is a non-profit organization dedicated to accelerating the discovery of a cure for ALS by offering incentives to drive innovation. The organization announced its $1 million ALS Biomarker Challenge in 2006 with the goal of finding a biomarker that could reduce the cost of Phase II clinical trials. Dr. Rutkove's research demonstrated the potential to reduce the cost of Phase II trials by 50 percent or more, which could facilitate more rapid development of new and better treatments for ALS.

“As a practicing neurologist, I regularly witness the devastating effects of ALS”

ALS is a rapidly progressive, invariably fatal neuromuscular disease that affects an estimated 20-30,000 people in the U.S. alone. Most victims die within two to five years, and there is no cure. Progress in finding a treatment for this disease has been limited in part because researchers have lacked an objective, reproducible and sensitive way to evaluate how well potential treatments acted against the disease. The standard way to evaluate efficacy has been to measure life expectancy but these studies can be long and expensive. This has contributed to the fact that today just one treatment for ALS exists, Riluzole, which extends survival by approximately three months.

"We need new and better treatment for ALS," said Merit Cudkowicz, Professor of Neurology at Harvard Medical School and Co-Chair of the Northeast ALS Consortium. "ALS is a serious neurological disorder characterized by progressive muscle weakness. There have been great advances in understanding the pathways that are affected in people with ALS. While there are hopeful treatment approaches being developed, it is critical to identify technologies that have improved ways to measure the effects of treatment. The new biomarker discovery brings us one step closer to the day when ALS might be slowed, stopped and ultimately prevented."

The biomarker research has identified a new way to measure how sick a muscle is and track its changes over time: It offers researchers an objective, quantifiable way to measure how well a potential ALS treatment is working to halt disease progression. Called electrical impedance myography or EIM, the prize-winning technique is based on the observation that as a muscle becomes more diseased, electrical current moves through it differently. The practice of measuring electrical impedance has a long history of providing valuable information across a variety of medical and non-medical applications. However, it has never been optimized to measure localized muscle changes associated with neuromuscular disease. The biomarker discovery gives drug developers a highly precise way to measure the effectiveness of experimental therapies earlier, potentially reducing the time and cost of Phase II clinical trials and propelling the availability of new and better treatments for ALS.

"The ALS community has long been searching for a new biomarker for ALS," said Melanie Leitner, PhD, Chief Scientific Officer of Prize4Life. "There are currently no precise measures of ALS disease progression that allow for short-term monitoring of the disease and the assessment of treatment efficacy. Dr. Rutkove's careful and thorough body of work addresses this need, offering renewed hope for the development of new and varied treatment options for the many patients and their families suffering from ALS."

EIM: Proven Technology Specifically Designed for Neuromuscular Disease

Dr. Rutkove co-founded Convergence Medical Devices so that his EIM research, which spans more than a decade and includes over 25 published studies, could inform the creation of a device optimized to measure the health of the muscle. Plans are under way to use CMD's investigational device in human clinical trials to collect device data for further algorithm development, to support a premarket submission to the Food and Drug Administration, and to measure treatment effects as a biomarker along with other endpoints in a Phase II drug trial.

The company's product is intended to be a portable, hand-held, non-invasive device that a physician or researcher could place over a muscle or group of muscles of interest to provide data that correlates with the health of that muscle. The device would work by delivering an imperceptible current through the muscle. A combination of EIM technology, sophisticated electronics and a highly intelligent algorithm would capture and process more than 1,000 different data points in a single measurement. After approximately 10 seconds, the device would provide the physician or researcher with a score that would reflect the status of the muscle. The device could measure a variety of muscles—even the tongue—which is important because ALS progression can vary significantly among different muscles. For therapy researchers, the changes in the obtained values would provide reliable, powerful measures of a potential treatment's efficacy. For patients, a physician could follow a patient's numerical score over time to assist in making treatment modifications. Once new and better treatments for ALS exist, the vision is that the device could be used to manage ALS treatment, similar to the way blood tests are used to make treatment decisions for diseases like HIV or Lupus today.

"As a practicing neurologist, I regularly witness the devastating effects of ALS," said Dr. Rutkove. "I am honored to receive Prize4Life's $1 million ALS Biomarker Prize, and will continue my research to expand the application of EIM to other neuromuscular diseases. It is my greatest hope that someday I could say to patients, 'Great news! Although your muscles deteriorated by 12 percent last year, since you've started this new therapy they've actually gotten three percent better.' Today, as far as ALS is concerned, I can only tell them they are getting worse."

Convergence Medical Devices has formed partnerships with several neuromuscular disease organizations, which plan to use the company's investigational EIM device in future clinical trials. These include agreements with Charley's Fund and the Nash Avery Foundation, two leading organizations which provide research funding to find a cure for Duchenne Muscular Dystrophy (DMD), and the Northeast ALS Consortium, a group of leading physicians dedicated to ALS clinical trials research. The company has also received multiple Small Business Innovation Research grants from both the National Science Foundation and the National Institutes of Health.