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Apr 27

Answer ALS trial begins enrollment

Packard scientists head $25 million effort to gather unprecedented clinical information about ALS

For Peter Lubin, enrolling as a patient in the Answer ALS trial was an easy decision. The first sign that something might be wrong with the 59-year-old telecommunications executive was pain in his right shoulder and muscle twitches in his right arm after a hiking adventure out west. A visit to the neurologist revealed that this was more than just post-hiking fatigue. Lubin's diagnosis of ALS came out of the blue as this happily married father of one had no family history of the disease and had led an extremely healthy and active lifestyle. He even served as a member of the National Ski Patrol (think paramedics on skis).

Diagnosed in Philadelphia, Lubin and his wife Carole headed south to Johns Hopkins late last year for a second opinion with Jeffrey Rothstein, founder and director at the Packard Center and a noted Hopkins neurologist. That’s when he first heard about the Answer ALS trial.  “I told Dr. Rothstein on the first visit that I wanted to be a poster child for a cure,” Lubin stated.

Peter Lubin begins the enrollment process for the Answer ALS trial at the Johns Hopkins ALS Clinic. (photo credit: Milan Karol)

On a recent visit to the Johns Hopkins ALS Clinic in January, Lubin was enrolled in the Answer ALS trial. Enrollment involved giving a medical history, breathing and muscle strength testing and a brief functional and cognitive test.  This was followed by a blood draw and collection. The blood was sent to labs participating in the Answer ALS program for detailed DNA analysis (genomics), and the creation of stem cell lines for further in-depth biological analyses ("multi-omics") and banking. Lubin is one of what ALS researchers hope will soon be 1000 participants in the Answer ALS trial that will provide detailed clinical and biological information about the disease.

Answer ALS originated as a result of the 2013 ALS Team Gleason Summit, which brought together leading researchers, patients, caregivers and advocates. The event was spearheaded by former NFL player Steve Gleason who lives with ALS and founded the ALS advocacy group, Team Gleason. The summit resulted in a scientific business plan to urgently find a treatment or cure for ALS.

Nearly $25 million in cash commitments, with additional gifts of technologies and various expertise, has been raised thus far from, and with the assistance of, a broad and diverse coalition of financial backers and supporters including: ALS Finding a Cure (a project of the Leandro P. Rizzuto Foundation), The Jay Fishman Family, Team Gleason, Travelers Insurance, the National Football League Trust, the PGA Tour, The Bari Lipp Foundation, The William H. Adams Foundation for ALS Research, The Greater Hartford Community Foundation, Microsoft, and others.

The trial design was developed and is being led by Answer ALS Executive Director Jeffrey Rothstein, MD, PhD, Director of the Brain Science Institute and the Robert Packard Center for ALS Research at Johns Hopkins University; in concert with Answer ALS Co-Director Dr. Clive Svendsen, PhD,  Director of the Regenerative Medicine Institute at Cedars-Sinai Medical Center; and Answer ALS Co-Director Merit Cudkowicz, MD, Director, Neurological Clinical Research Institute at Massachusetts General Hospital. Through Answer ALS, Rothstein and Svendsen hope to bring ALS research and treatment to the forefront of personalized medicine while simultaneously improving our understanding of this heterogeneous disease. Even as scientists at the Packard Center and other institutions around the world drill down into the molecular biology of ALS, some of the very basic questions about the disease remain unanswered.

Answering these questions will require data, and lots of it. Rothstein and Svendsen are using what they call a “multi-omics” approach, which includes relatively well known approaches such as genomics, as well as other, less familiar techniques like proteomics (protein analysis), metabolomics (small molecule analysis), transcriptomics (mRNA analysis), and more. Establishing and understanding this detailed genetic and metabolic information will allow us to understand each participant’s "ALS Signature" and then identify ALS subtypes based on those signatures. This is a new level of fidelity never before accomplished in ALS research.

Answer ALS and its community partners are ensuring that their ability to collect, process, and securely store these massive amounts of information does not outpace the ability of researchers to analyze the data. But the scientists involved with the program say that they’re up to the challenge, confident that this kind of big data is what will bring results.

As of April 19, Answer ALS has recruited 108 patients so far, including Lubin. Six clinical centers are participating in the trial: Johns Hopkins University, Massachusetts General Hospital, Emory University, Ohio State University, Washington University in St. Louis, and Cedars-Sinai Medical Center.  

Answer ALS is Lubin’s second clinical trial. He is also currently a patient in the study of rasagiline, a drug used to treat Parkinson’s disease by preventing the breakdown of dopamine, at the University of Pennsylvania. “I’m excited about the prospect of being involved in this trial,” Lubin noted. “I truly believe that the answer to this (ALS) is in stem cell research and medication. I am hopeful I am on the ground floor of something here.”

Donors are excited as well, due in large part to the project’s broad scope and unique goals. Unlike most medical research, where data remains proprietary, even after the completion of the trial, the information gathered by Answer ALS will be securely accessible to the global research community at no charge (no identifying patient information will be shared). The program will also generate a website with information about ALS, progress on the study, and data analysis for anyone to see.  Sharing the data openly in the research community, Rothstein says, will bring answers more quickly, since more minds can wring answers from it, and we can all collectively work towards a solution as rapidly and efficiently as possible.

“If you are sitting around dwelling on the disease, try to focus on what you can do to make it better.” Lubin hopes his enthusiasm for ALS research and the work done by Packard Center and Answer ALS will catch on with other ALS patients. 


The Answer ALS program stretches across multiple disciplines, starting with the clinic, moving to basic science and ending with big data. Ultimately Answer ALS will generate the largest and most comprehensive collection of ALS data ever amassed. (Fig legend).