Amyotrophic lateral sclerosis, commonly known as ALS, is an enduring mystery for life scientists–even those who have dedicated their careers to understanding it and finding a cure. Terminal, essentially untreatable, and frustratingly hard to predict, the disease has eluded us for years–and has achieved a sort of “orphan” status that makes it hard to draw attention and research dollars to ALS.
But an innovative new approach, drawing on the disciplines of both medical science and data science, offers the promise of an unprecedented breakthrough in the search for a cure. For the first time, Big Pharma and Big Data are coming together to foster insights in ALS. Specifically, working with nonprofits like mine, Teva ($TEVA), Novartis ($NVS), Sanofi ($SNY), Regeneron ($REGN), Knopp Biosciences, and other companies have opened up their clinical trials databases to help create the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database, an open-access repository of ALS patient and clinical trial data made available to researchers, patients, and members of the public. Although recent efforts by Big Pharma’s two major trade groups, EFPIA and PhRMA, will increase the amount of clinical trial data available in the future, PRO-ACT has already vastly increased the breadth and depth of ALS clinical data available.