Year: 2019
Type: article
Abstract: Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease where substantial heterogeneity in clinical presentation urgently requires a better stratification of patients for the development of drug trials and clinical care. In this study we explored stratification through a crowdsourcing approach, the DREAM Prize4Life ALS Stratification Challenge. Using data from >10,000 patients from ALS clinical trials and 1479 patients from community-based patient registers, more than 30 teams developed new approaches for machine learning and clustering, outperforming the best current predictions of disease outcome. We propose a new method to integrate and analyze patient clusters across methods, showing a clear pattern of consistent and clinically relevant sub-groups of patients that also enabled the reliable classification of new patients. Our analyses reveal novel insights in ALS and describe for the first time the potential of a crowdsourcing to uncover hidden patient sub-populations, and to accelerate disease understanding and therapeutic development.
Source: Scientific Reports
Authors: Robert Kueffner, Neta Zach, Maya Bronfeld, Raquel Norel, Nazem Atassi, Venkat Balagurusamy, Barbara Di Camillo, Adriano Chio, Merit Cudkowicz, Donna Dillenberger, Javier Garcia-Garcia, Orla Hardiman, Bruce Hoff, Joshua Knight, Melanie L. Leitner, Guang Li, Lara Mangravite, Thea Norman, Liuxia Wang, Rached Alkallas, Catalina Anghel, Jeanne Avril, Jaume Bacardit, Barbara Balser, John Balser, Yoav Bar-Sinai, Noa Ben-David, Eyal Ben-Zion, Robin Bliss, Jialu Cai, Anatoly Chernyshev, Jung-Hsien Chiang, Davide Chicco, Bhavna Ahuja Nicole Corriveau, Junqiang Dai, Yash Deshpande, Eve Desplats, Joseph S. Durgin, Shadrielle Melijah G. Espiritu, Fan Fan, Philippe Fevrier, Brooke L. Fridley, Adam Godzik, Agnieszka Golińska, Jonathan Gordon, Stefan Graw, Yuelong Guo, Tim Herpelinck, Julia Hopkins, Barbara Huang, Jeremy Jacobsen, Samad Jahandideh, Jouhyun Jeon, Wenkai Ji, Kenneth Jung, Alex Karanevich, Devin C. Koestler, Michael Kozak, Christoph Kurz, Christopher Lalansingh, Thomas Larrieu, Nicola Lazzarini, Boaz Lerner, Wojciech Lesinski, Xiaotao Liang, Xihui Lin, Jarrett Lowe, Lester Mackey, Richard Meier, Wenwen Min, Krzysztof Mnich, Violette Nahmias, Janelle Noel-MacDonnell, Adrienne O’Donnell, Susan Paadre, Ji Park, Aneta Polewko-Klim, Rama Raghavan, Witold Rudnicki, Ehsan Saghapour, Jean-Bernard Salomond, Kris Sankaran, Dorota Sendorek, Vatsal Sharan, Yu-Jia Shiah, Jean-Karl Sirois, Dinithi N. Sumanaweera, Joseph Usset, Yeeleng S. Vang, Celine Vens, Dave Wadden, David Wang, Wing Chung Wong, Xiaohui Xie, Zhiqing Xu, Hsih-Te Yang, Xiang Yu, Haichen Zhang, Li Zhang, Shihua Zhang, Shanfeng Zhu, Jinfeng Xiao, Wen-Chieh Fang, Jian Peng, Chen Yang, Huan-Jui Chang, Gustavo Stolovitzky
Institutions: Icahn School of Medicine at Mount Sinai, Teva Pharmaceuticals (Israel), IBM (United States), IBM Research - Thomas J. Watson Research Center, Massachusetts General Hospital, University of Padua, University of Turin, Universitat Pompeu Fabra, Trinity College Dublin, Sage Bionetworks, Amazon (United States), Zillow Group (United States), McGill University, Ontario Institute for Cancer Research, École Polytechnique, Newcastle University, Veristat, LLC (United States), Ben-Gurion University of the Negev, National Cheng Kung University, Princess Margaret Cancer Centre, University of Kansas Medical Center, Massachusetts Institute of Technology, University of Pennsylvania, Centre de Recherche en Économie et Statistique, Moffitt Cancer Center, Sanford Burnham Prebys Medical Discovery Institute, University of Białystok, University of Cambridge, RTI International, KU Leuven, University of Colorado Boulder, FHWien der WKW, Chinese Academy of Sciences, National Center for Mathematics and Interdisciplinary Sciences, Academy of Mathematics and Systems Science, Stanford University, Tel Aviv University, Helmholtz Munich, Fudan University, Microsoft Research New England (United States), Wuhan University, Children's Mercy Hospital, LinkedIn (United States), Interdisciplinary Centre for Mathematical and Computational Modelling, University of Warsaw, University of Warsaw, Isfahan University of Medical Sciences, Centre de Recherche en Mathématiques de la Décision, Université Paris-Est Créteil, Laboratoire d’Analyse et de Mathématiques Appliquées, Université Paris Dauphine-PSL, University of Moratuwa, University of California, Irvine, University of Washington, City University of Hong Kong, University of Maryland, Baltimore, University of Illinois Urbana-Champaign, National University of Tainan, Monash University
Language: English
Subfield: Neurology
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $1,790
Funders: Sanofi, Microsoft Research, Teva Pharmaceutical Industries, University of Pennsylvania, City University of Hong Kong, University of Washington, RTI International, McGill University, Moffitt Cancer Center, Prize4Life, Regeneron Pharmaceuticals, Children's Mercy Hospital, Northeast Amytrophic Lateral Sclerosis Consortium, Newcastle University, Chinese Academy of Sciences, Fudan University, Isfahan University of Medical Sciences, KU Leuven, Ben-Gurion University of the Negev, Wuhan University, National Cheng Kung University, University of Moratuwa, Perelman School of Medicine, University of Pennsylvania, University of California, Irvine, Helmholtz Zentrum München, Interdyscyplinarne Centrum Modelowania Matematycznego i Komputerowego UW, National Center for Mathematics and Interdisciplinary Sciences, Chinese Academy of Sciences, Core Research for Evolutional Science and Technology
OpenAlex ID: W2795490756