Prize4Life, a nonprofit organization dedicated to accelerating discovery of treatments and a cure for ALS (amyotrophic lateral sclerosis, also known as Lou Gehrig's disease), launched the PRO-ACT (Pooled Resource Open-Access ALS Clinical Trials) database, the largest ALS clinical trial database ever created.
PRO-ACT is a powerful tool that merges data from existing publicly and privately conducted ALS clinical trials and makes these data widely available for research, creating an invaluable resource for accelerating discovery in the field of ALS. Prize4Life and the Neurological Clinical Research Institute (NCRI) at Massachusetts General Hospital created the PRO-ACT database with funding from the ALS Therapy Alliance and in partnership with the Northeast ALS Consortium (NEALS). Generous data donations came from pharmaceutical companies including Sanofi, Novartis, Teva Pharmaceutical Industries and Regeneron Pharmaceuticals, as well as academic institutions around the world.
"With the creation of the PRO-ACT database, Prize4Life and the NCRI have given companies like ours cause to rethink priorities and strategies regarding ALS, since the abundant clinical and patient data contained within it will help us develop viable phase II and phase III treatments for ALS," said Dr. Doug Kerr, medical director, neurodegeneration clinical development, Biogen Idec.
PRO-ACT contains more than 8,500 fully de-identified unique clinical trial patient records, including demographic, lab, medical history, functional scores and other data elements. The dataset currently includes both placebo and, in most instances, treatment-arm data from 18 late stage (phase II/III) ALS clinical trials, resulting in over 8 million longitudinally collected data points. Prize4Life and the NCRI initiated this project with the belief that making ALS clinical trial data globally available would catalyze more ALS discoveries to happen sooner.
"The vision for PRO-ACT is that the ALS community and other interested researchers around the world will have access to enough data to answer previously unanswerable basic questions, such as, how much does ALS differ between men and women," said Dr. Melanie Leitner, chief scientific officer, Prize4Life. "PRO-ACT will also help to answer more complicated questions, such as: Can we identify subgroups of people who may actually have responded to treatment in any of the completed trials?"
ALS, a fatal disease, is difficult to predict. Although the average life expectancy of an ALS patient is about three years, some people live for decades, while others succumb within months. This lack of predictability makes the design of clinical trials for potential new treatments a long, costly and complex process. One key to better predictability in the future lies in the past—ALS research will move forward when scientists are able to identify the patterns hiding in the millions of data points in PRO-ACT, which were collected from thousands of ALS patients involved in previous clinical trials.
"As clinicians who see ALS patients every day, we recognize the huge potential impact of having access to vastly more patient data than anyone has ever had before. As just one example, being able to identify factors that determine the rate of progression in people with ALS will allow us to improve clinical trial design," said Dr. Merit Cudkowicz, director of the NCRI, neurology chief at Massachusetts General Hospital and NEALS co-chair. "Because of PRO-ACT, ALS researchers will be better able to design trials that need fewer participants."
The PRO-ACT database is freely accessible to the global research community for analysis and ca be found at www.ALSDatabase.org.