Getting PRO-ACTive about ALS

Prize4Life launches PRO-ACT ALS trials database

Lloyd Dunlap
CAMBRIDGE, Mass.—Prize4Life, a nonprofit organizationdedicated to accelerating discovery of treatments and a cure for amyotrophiclateral sclerosis (ALS, also known as Lou Gehrig's disease), has launched thePooled Resource Open-Access ALS Clinical Trials (or PRO-ACT) database, said tobe the largest ALS clinical trial database ever created.
 
 
PRO-ACT merges data from existing publicly and privatelyconducted ALS clinical trials and makes these data widely available forresearch, creating a valuable resource for accelerating discovery in the fieldof ALS. Prize4Life and the NeurologicalClinical Research Institute (NCRI) at Massachusetts General Hospitalcreated the PRO-ACT database with funding from the ALS TherapyAlliance and in partnership with the Northeast ALSConsortium (NEALS). Data donations came from pharmaceuticalcompanies including Sanofi,Novartis,TevaPharmaceutical Industries and RegeneronPharmaceuticals, as well as several academic institutions.
 
 
"With the creation of the PRO-ACT database, Prize4Life andthe NCRI have given companies like ours cause to rethink priorities andstrategies regarding ALS, since the abundant clinical and patient datacontained within it will help us develop viable Phase II and Phase IIItreatments for ALS," said Dr. Doug Kerr, medical director of neurodegenerationclinical development at Biogen Idec. The company currently has one ALS drugtherapy in Phase III trials. The drug was developed with Knopp Biosciences andis described as a novel oral neuroprotective therapy. 
 
PRO-ACT contains more than 8,500 fully de-identified, uniqueclinical trial patient records, including demographic, lab, medical history,functional scores and other data. The dataset currently includes both placeboand, in most instances, treatment-arm data from 18 late-stage (Phase II/III)ALS clinical trials, resulting in more than 8 million longitudinally collecteddata points. Prize4Life and the NCRI initiated this project with the beliefthat making ALS clinical trial data globally available would expedite more ALSdiscoveries. 
 
"The vision for PRO-ACT is that the ALS community and otherinterested researchers around the world will have access to enough data toanswer previously unanswerable basic questions, such as how much does ALSdiffer between men and women," said Dr. Melanie Leitner, chief scientificofficer of Prize4Life. "PRO-ACT will also help to answer more complicated questions,such as: Can we identify subgroups of people who may actually have responded totreatment in any of the completed trials?"
 
 
The course of ALS is difficult to predict. Although theaverage life expectancy of patients is about three years, some people live fordecades, while others succumb within months. This lack of predictability makesthe design of clinical trials for potential new treatments a long, costly andcomplex process. One key to better predictability in the future lies in thepast—ALS research will move forward when scientists are able to identify thepatterns hiding in the millions of data points in PRO-ACT, which were collectedfrom thousands of ALS patients involved in previous clinical trials.
 
 
"As clinicians who see ALS patients every day, we recognizethe huge potential impact of having access to vastly more patient data thananyone has ever had before. As just one example, being able to identify factorsthat determine the rate of progression in people with ALS will allow us toimprove clinical trial design," says Dr. Merit Cudkowicz, director of the NCRI,neurology chief at Massachusetts General Hospital and NEALS co-chair. "Becauseof PRO-ACT, ALS researchers will be better able to design trials that needfewer participants."
 
 
Prize4Life is a results-oriented nonprofit founded in 2006by Avichai "Avi" Kremer, and two of his Harvard Business School classmates,with the sole purpose of finding a cure for ALS. Avi was diagnosed in 2004 atthe age of 29 during his first semester at HBS. He and his friends decided topilot an innovative new way to accelerate ALS research. The group offerssubstantial prizes to scientists who solve the most critical scientificproblems preventing the discovery of an effective ALS treatment. The Prize4Lifeconcept is inspired by other prize awards for stimulating research, such as theX-Prize for commercial space travel and DNA-decoding, the U.S. government'sH-Prize for hydrogen renewable energy and Eli Lilly & Co.'s venture,InnoCentive.

Lloyd Dunlap

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