An image of the human brain.
(photo credit: REUTERS)
The voluntary organization Prize4Life-Israel has helped launch a global open-science data analysis competition this month to develop more personalized approaches for the research, prognosis and treatment of amyotrophic lateral sclerosis (ALS), the devastating neurological disease that attacks mostly young adults.
Together with Sage Bionetworks, the challenge – called DREAM ALS Stratification Prize – brings together patients, clinical researchers, data scientists and pharmaceutical industry leaders to computationally identify for the first time different subgroups of ALS patients in the hope of paving the way for potential new treatments.
ALS, also known as Lou Gehrig’s disease, is a fatal, rapidly progressing neurodegenerative disease that leads to paralysis and eventually death. One in 1,000 individuals will live with – and die of – ALS. There is no cure, and the average lifespan with the disease is three to five years, but a rare few, like British astrophysicist Prof. Stephen Hawking, live for decades with it. Hawking, 73, was diagnosed at the age of 21 and has lived longer than almost any other ALS patient.
“We believe the ALS stratification challenge holds great promise to unlock the mysteries of how ALS develops and accelerates the development of ALS treatments and a cure,” said Shay Rishoni, Prize4Life CEO and an ALS patient. “We established the database in 2012 – with the support and partnership of the Northeast ALS Consortium, the ALS Therapy Alliance and, of course, drug companies – exactly for these goals, which are to bring hundreds of fresh new minds into the world of ALS research.
“A breakthrough for ALS research will eventually come; we invite the brilliant minds of the computational research community to participate in that victory,” he said.
The ALS stratification challenge is running from June until the middle of September, and aims to spur the development of quantitative solutions to stratify ALS patients based on their disease progression or survival, said Avi Kremer, an ALS patient diagnosed in 2004 who with his friends established Prize4Life.
The organization, a non-profit dedicated to the discovery of treatments or a cure for ALS, uses financial incentives to attract people to the research and drive innovation.
Prize4Life is providing the largest open ALS clinical trials database in the world that will serve as the basis for the challenge, whose participants can access and analyze the data, work alone or in teams and share their ideas and analysis results with other participants.
The key goal of the challenge is to identify the attributes that differentiate ALS patients. Such information will help patients and families plan accordingly to increase their quality of life and will also help guide the development of stratified ALS clinical trials that could enroll specific ALS subgroups for the testing of new treatments.
The first of the challenges focuses on predicting the progression and survival of ALS patients. It will use a database of more than 8,000 cases as the “challenge training set” and feature several unpublished datasets that will be used for model validation.
“We believe the ALS stratification challenge holds great promise to unlock the mysteries of how ALS develops and accelerates, the development of ALS treatments, and a cure,” the organizers said on Sunday.
Without an ability to distinguish between patients with very different disease progressions, clinical trial efforts to evaluate potential new ALS treatments end up being expensive and doomed to failure.
The top-performing teams will each receive a $7,000 cash prize, be invited to present the winning model at DREAM’s conference this fall, and have the opportunity to co-author a challenge overview paper that the Nature Biotechnology journal has expressed interest in considering.
Sage Bionetworks is a nonprofit biomedical research organization that was founded in 2009 with a vision to promote innovations in personalized medicine by enabling a community-based approach to scientific inquiries and discoveries.
Prize4Life and DREAM have already demonstrated the power of open-science challenges to advance ALS disease research. The ALS Prediction Prize challenge conducted in 2012 had more than 1,000 registrants from 63 countries, and the winning approaches – described in an article in the November 2014 issue of Nature Biotechnology – outperformed the predictions of more than 12 expert ALS clinicians.
Leading research-based bio-pharmaceutical companies Biogen and Eli Lilly are helping fund the challenge effort and providing key advice, and IBM is providing cloud computing.
The cash prizes for the challenge were raised by running a crowdfunding campaign called “Fund the Prize” that ran in last fall and succeeded in raising $28,000.