MDA VENTURE PHILANTHROPY GRANT TO SUPPORT DEVELOPMENT OF ALS BIOMARKERS

The Muscular Dystrophy Association is pleased to announce the award of an MDA Venture Philanthropy (MVP) grant totaling $233,200 to Scottsdale, Arizona-based Iron Horse Diagnostics to support development of a prognostic test for ALS.

T his prognostic Such a test could help improve and accelerate clinical trials and speed the development of life-saving drugs to people with ALS (amyotrophic lateral sclerosis). MVP is MDA's drug development program, which is exclusively focused on funding the discovery and clinical application of treatments and cures for neuromuscular disorders. MVP evaluates and makes targeted investments in for-profit and not-for-profit companies and academics developing therapeutics.

Not only will the new test be important in helping physicians predict the likely course of disease — for example, fast versus slow progression — in individuals with ALS, but it will help inform clinical trial enrollment and potentially accelerate the pace of drug development for ALS.

"MDA is excited to support this work at Iron Horse Diagnostics, with the goal to address one of the most pressing challenges to ALS drug development — the need for prognostic biomarkers," said MDA Scientific Program Officer Amanda M. Haidet-Phillips, Ph.D. "In addition to its usefulness in planning and conducting clinical trials, the prognostic test under development at Iron Horse could be helpful for individuals and families affected by ALS as they prepare for financial and life planning purposes and wish to have this additional information."

The prognostic test will measure specific protein-based biomarkers in blood and cerebrospinal fluid that indicate the presence of neurodegenerative disease. "MDA funding is critical to our efforts to finalize development of our biomarkers and to launch the test to market," said Iron Horse Diagnostics Chief Scientific Officer Andreas Jeromin, Ph.D. "Our prognostic test will be an essential tool to enhance drug development for ALS."

MDA is leading the fight to free individuals from the harm of muscular dystrophy, ALS and related muscle-debilitating diseases that take away physical strength, independence and life.

MDA has dedicated more than $363 million to ALS research and support services since 1950 and currently is funding 38 active ALS grants with a total funding commitment of $9.6 million. This year, MDA is funding 150 different research projects around the world. ALS (amyotrophic lateral sclerosis) is a disease of the parts of the nervous system that control voluntary muscle movement. In ALS, motor neurons (nerve cells that control muscle cells) are gradually lost. As these motor neurons are lost, the muscles they control become weak and then nonfunctional. Loss of voluntary muscles can lead to an inability to walk, to swallow and to speak.

Those in the advanced stages of ALS may become unable to eat, move or breathe independently. ALS onset usually begins in people at late middle age or older, although it also occurs in young adults and even in children, as well as in very elderly people. Men are slightly more likely to develop ALS than are women. Although no one knows for sure, reports suggest approximately 30,000 people in the United States have ALS; every year, doctors tell about 8,000 people that they have it. Life span is typically three to five years after diagnosis.

MDA is leading the fight to free individuals — and the families who love them — from the harm of muscular dystrophy, ALS and related muscledebilitating diseases that take away physical strength, independence and life. We use our collective strength to help kids and adults live longer and grow stronger by finding research breakthroughs across diseases; caring for individuals from day one; and empowering families with services and support in hometowns across America. Learn how you can fund cures, find care and champion the cause at mda.org Iron Horse Diagnostics is an early-stage biotech company focused on developing diagnostic and prognostic tests for neurological conditions with high unmet medical need. Founded in 2012 by Dr. Robert Bowser, Iron Horse has developed assays for ALS, traumatic brain injury/concussion and for multiple sclerosis relapse. The U.S. launch for the ALS diagnostic test is set for late 2017.•