MDA and Answer ALS Form New Partnership to Support the Largest and Most Comprehensive ALS Dataset to Date
MDA will fund the development of computational tools that can integrate and analyze the complex disease networks
NEW YORK, June 20, 2018 — The Muscular Dystrophy Association (MDA) today announced that it has awarded a research infrastructure grant totaling $550,000 to Jeffrey Rothstein, M.D., Ph.D., Professor of Neurology and Neuroscience and Director, Robert Packard Center for Answer ALS Research, Johns Hopkins University, School of Medicine. Answer ALS is a nationwide consortium assembling one of the most comprehensive clinical, genetic, molecular, and biochemical assessments of amyotrophic lateral sclerosis (ALS) to date.
The Answer ALS and MDA partnership will develop a series of tools, known as probabilistic graphical models (PGMs), to specifically interrogate and interpret the combined Answer ALS datasets. PGMs have been used extensively in applications from finance to artificial intelligence and healthcare diagnostics. These flexible, interpretable models are able to identify and quantify changes in cellular pathways. Creation and application of PGMs to enable integrated models of this comprehensive dataset can enhance understanding of disease mechanisms and advance the discovery of targets for effective therapy.
Answer ALS is directed by longtime MDA grantee Jeffrey Rothstein, professor of neurology and neuroscience at Johns Hopkins University School of Medicine in Baltimore.
“This is the largest initiative aimed at understanding subgroups of ALS and finding drug targets,” says Rothstein. “The data is key. The Answer ALS initiative could pull more than six million data points per patient and help uncover multiple pathways of disease, pinpoint new drug targets and biomarkers, and discover novel ways to integrate the data into drug discovery.”
Through the Answer ALS program, 1,000 ALS patients across the U.S. will be followed for one year. Disease measures such as breathing function and muscle strength will be captured at clinical visits, but participants will also use an “app” created to track disease progression at home. Biospecimens such as blood and cerebrospinal fluid will also be collected, allowing for analysis of DNA, RNA, protein, and cellular pathways, as well as for generation of stem cell lines from ALS patients used to model disease in a dish. The overarching goal of this program is to better understand, and ultimately end ALS. This comprehensive dataset will generate over 20 trillion data points and MDA’s grant will fund the development of new data analytics approaches to support the integration and analysis of this massive amount of data, which will be shared openly with the global research community.
ALS is a progressive disease of the nerve cells that leads to loss of voluntary muscle control, paralysis, difficulty speaking, swallowing and ultimately, breathing. Today, it is recognized that ALS is unlikely to be one disease; rather, it may be a collection of subtypes and variants, each of which requires a different approach for optimal treatment.
“Mining this rich, complex dataset with the goal of identifying ALS disease subtypes, requires sophisticated computational tools,” says MDA Scientific Program Officer Amanda Haidet-Phillips, Ph.D. “The project investigators will develop a series of tools to specifically interrogate and interpret the combined Answer ALS datasets leading to an improved understanding of ALS.”
The Answer ALS initiative has garnered wide support from not only foundations, but also from major organizations such as the National Football League, Google, IBM Research, American Airlines, Microsoft and the PGA Tour.
“We are incredibly fortunate to have partnered with Professor Ernest Fraenkel and his team at MIT, true experts in the field of data analytics, as we embark on this remarkable study,” Rothstein says.
MDA is leading the fight to free individuals — and the families who love them — from the harm of muscular dystrophy, ALS and related muscle-debilitating diseases that take away physical strength, independence and life. We use our collective strength to help children 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 AmericaLearn how you can fund cures, find care and champion the cause at mda.org.
About Answer ALS
Answer ALS is the single largest & most comprehensive ALS research project in history, producing more ALS data than has ever been amassed, while openly sharing with the global research community. We will use that data to investigate the unique pathways of each variation of ALS & begin to develop the right treatment or cure. We believe that only a large-scale concerted and coordinated effort will make the difference in finding the answers to ALS. Answer ALS is an unprecedented approach to understanding and defeating the disease.
Director, Public Relations & Communications
Answer ALS, Public Relations
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