Answer ALS: A Revolutionary Project to Cure ALS

Answer ALS: A Revolutionary Project to Cure ALS

The launch of Answer ALS, the largest comprehensive research project to cure Amyotrophic Lateral Sclerosis (ALS) was announced in a press release by the Johns Hopkins University’s Robert Packard Center for ALS Research in Baltimore, MD, the Cedars-Sinai Medical Center’s Regenerative Medicine Institute in Los Angeles, CA, and the Massachusetts General Hospital Neurological Clinical Research Institute, Boston, MA.

ALS is a rapidly progressive and fatal disease characterized by a degeneration of motor neurons. As disease evolves, the brain loses its ability to control muscle movement, eventually leading to body paralysis. There is no effective treatment for this disease, which annually affects 2-4 people in every 100,000 in Europe and the US.

Answer ALS aims at: i) finding the causes of ALS; ii) develop human cell models to understand ALS and screen for drugs and therapies; iii) implement individual ALS therapies based on each patients’ own brain cell data; iv) establish therapies that could improve ALS symptoms and eventually cure them; and v) freely disseminate all the information collected to worldwide in order to advance therapy development.

To achieve these goals, the project will have an aggressive and comprehensive research policy – real-time 24/7 monitoring and thorough clinical and biological data collection of a large patient population (minimal 1,000 patients); generation of pluripotent cell lines from each patients’ brain cells to create novel cellular models; simultaneous analysis of all data gathered using comprehensive algorithms that include patients’ clinical, cellular, biochemical, genetic, steam cells and robotic images; and the creation of a collaborative consortium with world’s experts on clinical, cellular, biochemical, genetics, stem cells and big data analysis fields.

Dr. Jeffrey Rothstein, Director of the Brain Science Institute and the Robert Packard Center for ALS Research at Johns Hopkins University and the Executive Director of Answer ALS stated “This project will bring together world-renowned ALS research scientists to work against an aggressive timeline for understanding, treating and eventually finding a cure for this disease. The substantial initial funding from these generous supporters is a critical step forward in our effort to provide hope to those affected by ALS.”

The project is expected to generate the world’s largest comprehensive ALS data, encompassing all clinical and biological aspects of each patient enrolled in the project. Machine learning and big data technology will be used in data analysis to unveil ALS causes, subtypes, key pathways and drug targets. This information will help in the development of new clinical trials and in novel categorization methods for patients to identify specific ALS pathways, biomarkers and disease pathophysiology, which will aid in the early diagnosis of the disease and treatment efficacy.

The project is a result of the 2013 ALS Team Gleason Summit – an initiative of the former NFL player Steve Gleason, who lives with ALS – in which leading ALS researchers, patients, caregivers and advocates came together to decide what measures were necessary to overcome this devastating disease. At the moment, the project encompasses nearly two dozen US research institutes, being directed by Dr. Jeffrey Rothstein co-aided by Dr. Clive Svendsen, Director of the Regenerative Medicine Institute at Cedars-Sinai Medical Center and Dr. Merit Cudkowicz, Chief of Neurology and Co-Director of the Neurological Clinical Research Institute at Massachusetts General Hospital.

Answer ALS is funded by a Committed Team of Donors that include the ALS finding a cure project from the Leandro P. Rizzuto Foundation, the Robert Packard Center for ALS Research, the National Football League, PGA TOUR, Travelers, The Fishman Family and The Bari Lipp Foundation. Thus far, $20 Million have been contributed. A total of about $25 million is required for the first phase of the program. Funding will be coordinated through the Robert Packard Center for ALS Research and the ALS Finding a Cure Foundation.

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