Largest Clinical Trials Database of Amyotrophic Lateral Sclerosis Created to Find a Cure

Prize4Life (Cambridge, MA, USA), a nonprofit organization whose goal is to hasten the discovery of treatments and a cure for ALS, and the Neurological Clinical Research Institute (NCRI; Boston, MA, USA) 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).

Substantial data donations came from pharmaceutical companies including Sanofi, Novartis, Teva Pharmaceutical Industries, and Regeneron Pharmaceuticals, as well as academic institutions worldwide. “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, Biogen Idec’s (Zug, Switzerland) medical director, neurodegeneration clinical development.

PRO-ACT contains more than 8,500 fully deidentified unique clinical trial patient records, including medical history, demographic, lab, functional scores, and other data components. The dataset currently includes both placebo, and in most cases, treatment-arm data from 18 late stage (phase II/III) ALS clinical trials, resulting in over eight million longitudinally gathered data points. Prize4Life and the NCRI initiated this project with the conviction that making ALS clinical trial data globally available would catalyze more ALS discoveries to occur faster.

“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 Prize4Life’s chief scientific officer, Dr. Melanie Leitner. “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 tough to predict. Although the average life expectancy of an ALS patient is approximately three years, some individuals live for decades, whereas others die within months. This absence of probability makes the design of clinical trials for possible new treatments a long, expensive, and complicated process. ALS research will advance when researchers are able to identify the patterns hidden in the millions of data points in PRO-ACT, which were collected from thousands of ALS patients involved in earlier 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 findings gathered by use of the PRO-ACT database will benefit more than just the scientific and medical research fields; they will also impact ALS patients and their families potentially by giving them answers to their questions about what to expect following diagnosis.

“When my father was first diagnosed with ALS, one of the things my family and I struggled with the most was the time it took for him to receive a definitive diagnosis amid the ambiguity of the disease,” said Peter Bowen, former Prize4Life board fellow and primary fundraiser for the DREAM-Phil Bowen ALS Prediction Prize4Life Challenge named in honor of his late father, Phil Bowen. “It is my hope that the PRO-ACT database will provide answers which allow doctors to be able to tell an ALS patient and his or her family sooner, rather than later, what the future may hold.”

The PRO-ACT database is freely accessible to the global research community for analysis and downloads online (please see Related Links below).

Prize4Life is a) nonprofit organization that uses powerful incentives to drive innovation. Founded in 2006 by Avi Kremer, who was diagnosed with ALS at the age of 29, Prize4Life encourages and rewards creative approaches that will provide results for ALS patients.

The Neurological Clinical Research Institute (NCRI) at Massachusetts General Hospital accelerates translational research in neurologic disorders through initiating and assessing innovative therapies.

Related Links:
PRO-ACT database
Prize4Life
Neurological Clinical Research Institute