Progress & Media

Johns Hopkins University, Cedars-Sinai and Massachusetts General Hospital Announce Groundbreaking ALS Research Project of Answer ALS

Sep 9, 2015
Categories: Media

Research Designed to Fully Understand and Defeat ALS; "Big Data" Technology Will Drive Unprecedented Analysis Capability

$20 Million in Funding from Committed Team of Donors, including the National Football League, the PGA TOUR, ALS Finding a Cure (a project of the Leandro P. Rizzuto Foundation), Travelers, The Fishman Family and The Bari Lipp Foundation

BALTIMORE, LOS ANGELES and BOSTON, September 9, 2015 -- Johns Hopkins University's Robert Packard Center for ALS Research, Cedars-Sinai Medical Center's Regenerative Medicine Institute and the Massachusetts General Hospital Neurological Clinical Research Institute today announced the launch of an Amyotrophic Lateral Sclerosis (ALS) research project of Answer ALS, the largest single coordinated and comprehensive effort to end ALS.

ALS is a devastating, progressive and fatal neuromuscular illness for which there is no effective treatment. In ALS, gradual degeneration of motor nerve cells in the brain and spinal cord causes increased difficulty swallowing and breathing, and eventually leads to paralysis. A new person is diagnosed with ALS every 90 minutes in the United States.

The mission of Answer ALS is to:

  • Discover what causes ALS;
  • Develop sophisticated human cell models to understand ALS and screen for drug and cellular therapies;
  • Initiate individualized ALS therapy programs based on patients' own cellular data from brain cells;
  • Discover therapies that could substantially slow, halt and ultimately cure ALS; and
  • Provide all gathered information free for worldwide use by clinicians, scientists and pharma to rapidly advance therapeutics.

"This is an unprecedented approach to understanding and defeating disease," said Dr. Jeffrey Rothstein, Director of the Brain Science Institute and the Robert Packard Center for ALS Research at Johns Hopkins University and the Executive Director of Answer ALS. "This project will bring together world-renowned ALS research scientists to work against an aggressive timeline for understanding, treating and eventually finding a cure for this disease. The substantial initial funding from these generous supporters is a critical step forward in our effort to provide hope to those affected by ALS."

"We are combining big data, comprehensive biological analytics, technology and science in a way that would not have been possible a decade ago," said Clive Svendsen, PhD, Director of the Regenerative Medicine Institute at Cedars-Sinai Medical Center and Co-Director of Answer ALS. "What is so exciting and important is that all of this data will be made publicly available with free, open access to the ALS research community, so that we can make faster and greater strides together to find a cure for this disease."

"By leveraging clinical and sample data collection systems built by the Northeast ALS Consortium (NEALS) with the support of the ALS Association and ALS Finding a Cure Foundation, enrollment of participants and sharing of data and samples will be accelerated," said Merit Cudkowicz, MD, Chief of Neurology and Co-Director of the Neurological Clinical Research Institute at Massachusetts General Hospital and Co-Director of Answer ALS. "In partnership with our patients, their families and our NEALS research ambassadors, this collaborative project will accelerate finding treatments for people with ALS."

The project will create the largest and most comprehensive foundation of ALS data ever amassed, encompassing clinical, chemical, genetic, protein, historical and biological data from an enormous sampling of ALS patients in the United States. In collaboration with machine learning and big data technology, the data will be mined to uncover ALS causes, subtypes, pathways gone awry and drug targets. This will establish a large and critical foundation for new clinical trials, develop new ways to categorize patients to identify specific druggable pathways and subtype biomarkers and disease pathophysiology to aid not only in early diagnosis of the disease, but also to monitor the efficacy of newly developed treatments.

This research project also could potentially provide deeper understanding of Alzheimer's, Parkinson's and other neurodegenerative diseases.

Funded by Committed Private Donors

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

A funding effort led by the Leandro P. Rizzuto Foundation and the Robert Packard Center for ALS Research has resulted in $20 million raised for Answer ALS thus far. The initial stakeholder funders are the National Football League, the PGA TOUR, ALS Finding a Cure (a project of the Leandro P. Rizzuto Foundation), Travelers, The Fishman Family and The Bari Lipp Foundation.

Their funding will launch this research project in the coming months. A total of approximately $25 million is needed to complete the first phase of the program. Funding will be coordinated through the Robert Packard Center for ALS Research at Johns Hopkins University and Massachusetts General Hospital for the ALS Finding a Cure Foundation.

"I've always said, to change the course of this disease, as a community, we would have to be bigger than ALS," said Gleason. "I believe that if we commit to this effort, on this scale, we will have meaningful results in our lifetime. Reaching this amount of fundraising success in such a short amount of time is inspiring. I dared capable individuals to step up and help solve ALS. These committed individuals and organizations have boldly answered that challenge."

"The devastating impact of ALS was fully realized by the PGA TOUR's extended family in 2004 when it tragically claimed the lives of caddie Bruce Edwards and member Jeff Julian, just months apart," said PGA TOUR Commissioner Tim Finchem. "And now we learn that one of our dear friends and longtime associates, Jay Fishman of Travelers, has been diagnosed with ALS. We are extremely proud to lend our support to this concerted effort to finding a cure for this terrible disease."

Jay Fishman, Chairman and CEO of Travelers, who recently announced his ALS diagnosis, said, "After being diagnosed with ALS, I looked for the most aggressive project with the greatest possibility of yielding results through rapid benchmarks and milestones. This was that project."

Fishman continued, "The passion, commitment and collaborative spirit of this expert group of scientists and clinicians are nothing short of remarkable. I am deeply impressed by their knowledge and dedication."

"To accelerate development of treatments for people with ALS, we brought together a remarkable team of scientists, a true powerhouse, to answer one of the hardest and most important questions in the biology of ALS," said Peter Foss, President of the ALS Finding a Cure Foundation, and General Manager of the GE/NFL TBI research project. "We support open, collaborative science that tackles fundamental therapy-driven questions and includes real-time community sharing of data. This is the best approach to accelerate finding a cure for all people with ALS."

Driven by a Collaborative Team of Leading ALS Research Scientists and Clinicians

This Answer ALS research project will be directed by Dr. Jeffrey Rothstein, Dr. Clive Svendsen and Dr. Merit Cudkowicz.

Program team leaders include:

  • Clinical: Nicholas Maragakis, Johns Hopkins University and James Berry, Massachusetts General Hospital, Harvard University;
  • iPSC: Dhruv Sareen, Cedars-Sinai;
  • Omics: Leslie Thompson, University of California Irvine; and
  • Data Analysis: Steven Finkbeiner, Gladstone Institutes/UCSF and Ernest Frankel, Massachusetts Institute of Technology.

Dedicated to Aggressive, Comprehensive Research

Specifics of Answer ALS's research program include:

  • Large Patient Population: Urgently evaluating a very large patient population (at least 1,000+ ALS patients) and collecting comprehensive and longitudinal clinical data from each participant through 5-7 highly experienced, geographically diversified ALS clinics over several years.
  • Real-Time 24/7 Clinical Data: Utilizing industry-leading, wearable computing technology to extensively measure participants' disease experience and using this expansive data to form algorithms that will merge health, biological and historical data.
  • Generate iPSC-derived Brain Cells: Generating induced pluripotent cell lines (iPSC) from each patient and using these to create thousands of new and different brain cell lines (e.g., motor and cortical neurons, astroglia, oligodendroglia).
  • Comprehensive Biological and Clinical Data Sets: Performing the most comprehensive biological analytics ever proposed on those patients' iPSC-derived brain cells, including whole genome, epi-genome, RNA, protein, cellular metabolism and robotic imaging analyses of actual growing human ALS patients' brain cells.
  • National Collaborative Consortium: Amassing a collaborative team of the world's experts on iPSC cells, DNA, RNA and protein analytics, clinical indices, clinical trial design and, most importantly, big data analytics.
  • Large Data Technology and Analytics: Bringing together some of the leading experts on large data sets to employ machine learning approaches to ALS.

To learn more about the Answer ALS program or to get involved, please visit www.answerals.org or call 1-800-490-2375.

About the Robert Packard Center for ALS Research at Johns Hopkins
The Robert Packard Center for ALS Research at Johns Hopkins is the only international collaborative scientific operation dedicated solely to curing the disease. The Center is unique in its approach to fighting ALS, a motor neuron disease, in that it combines and facilitates academic, clinical and scientific collaboration and ALS research with fundraising for the development of new disease models, new treatments and to finding a cure. Founded in 2000, the Packard Center brings ALS scientists from all over the world together to combine forces in ALS research. Each month, brainstorming sessions identify promising research approaches and evaluate progress. Packard grantees, their post-docs and lab staff, Packard scientific advisors, collaborating clinicians and a bare-bones administrative staff work together in this dedicated network. The vast majority of tools to understand ALS and to discover new therapies and new therapeutic candidates are developed by Packard Center researchers. For more information about the Packard Center and its current research portfolio, please visit their website: www.alscenter.org.

About Cedars-Sinai Board of Governors Regenerative Medicine Institute
Cedars-Sinai is a leader in providing high-quality healthcare encompassing primary care, specialized medicine and research. Since 1902, Cedars-Sinai has evolved to meet the needs of one of the most diverse regions in the nation, setting standards in quality and innovative patient care, research, teaching and community service. Today, Cedars-Sinai is known for its national leadership in transforming healthcare for the benefit of patients. Cedars-Sinai houses the Board of Governors Regenerative Medicine Institute that specializes in the production of induced pluripotent stem cells for both modeling and treating human diseases such as ALS. For more information, please visit: www.cedars-sinai.edu.

About the Massachusetts General Hospital (MGH) ALS Program
Founded in 1811, Massachusetts General Hospital (www.massgeneral.org) is the original and largest teaching hospital of Harvard Medical School. Since 1984, Massachusetts General Hospital has been providing the highest-quality care to individuals with amyotrophic lateral sclerosis (ALS), along with access to the latest treatments and research opportunities for patients, through the MGH ALS Multidisciplinary Clinic (www.massgeneral.org/als). As the clinical coordination center for ALS clinical trials, the MGH ALS Team and the Neurological Clinical Research Institute (www.massgeneral.org/ncri) are known for their leadership and innovation in bringing treatments forward efficiently and expeditiously to trials for people with ALS. Partnered with scientists at MGH and throughout the world, the MGH ALS team is committed to improving the health and care of people and families with ALS.

About Team Gleason
The Gleason Initiative Foundation, known as Team Gleason, is a charitable 501c3 non-profit corporation founded by former NFL player Steve Gleason who was diagnosed with ALS in 2011. Team Gleason's mission is to help provide individuals with neuromuscular diseases or injuries with leading edge technology, equipment and services; to create a global conversation about ALS to ultimately find solutions and an end to the disease; and, to raise public awareness toward ALS by providing and documenting extraordinary life adventures for individuals with muscular diseases or injuries. For more information, please visit: www.teamgleason.org.

About ALS Finding a Cure Foundation
Formed in spring of 2014, the sole purpose of the ALS Finding a Cure Foundation is funding research to find a cure. The foundation's goal is to translate funding into cutting-edge development tools, provide an open platform for neurological research material, understand disease heterogeneity to develop treatments and promote early detection for those affected by ALS. To break down barriers to finding a cure for people with ALS, we support collaborative science through partnerships, including with General Electric, the ALS Association and now Answer ALS. General Electric Healthcare, one of our most important collaborators, has developed breakthrough technology to better understand the brain and spinal cord. In partnership, we bring their technology and knowledge to understanding ALS and accelerating therapy development. Our team of researchers is led by our Chief Medical Officer Dr. Merit Cudkowicz and the foundation is overseen by Peter N. Foss. ALS Finding a Cure is a program of the Leandro P. Rizzuto Foundation. We are proud to report that 100% of donations goes directly to funding research and that all outside donations to ALS Finding a Cure are currently matched by The Leandro P. Rizzuto Foundation. ALS Finding a Cure is inspired by and is a tribute to Christie Rizzuto, who was diagnosed with ALS in 2009 at the age of 41. It is Christie, and the many others like her, who drive our work. For more information, please visit www.alsfindingacure.org.

Media Contacts:
Shawna Williams, Johns Hopkins, 410-955-8236, shawna@jhmi.edu
Vanessa McMains, Johns Hopkins, 410-502-9410, vmcmain1@jhmi.edu
Cara Martinez, Cedars-Sinai, 310-423-7798, Cara.martinez@cshs.org
Terri Ogan, Massachusetts General Hospital, 617-726-0954, togan@mgh.harvard.edu
Clare Durrett, Team Gleason, 504-450-4677, clare@teamgleason.org
Chris Collins, ALS Finding a Cure, 203-351-9156, Chris_Collins@conair.com