The ALS Association

Carrie Munk
The ALS Association


The ALS Association Announces Initial Commitment of $21.7 Million from Ice Bucket Challenge Donations to Expedite Search for Treatments and a Cure for ALS

Additional $12.5 Million in Matching Gifts Secured Bring Total Commitment to $34.2 million

Washington, D.C. (October 2, 2014)—After the generous outpouring of support from people all around the globe due to this summer’s Ice Bucket Challenge, the Board of Trustees of The ALS Association has approved an initial expenditure of $21.7 million in funding to support six programs and initiatives to expedite the search for treatments and a cure for amyotrophic lateral sclerosis (ALS). Additionally, $12.5 million in matching donations bring the total commitment to $34.2 million.

Four of these projects involve global research cooperative alliances that would not have moved forward without this significant funding from The Association, made possible through the generosity of donors worldwide, along with matching gifts.

“We recognize the sense of urgency felt by people living with the disease and their families and I want to assure everyone that our number one commitment is to making decisions that get treatments to patients in the fastest way possible,” said Barbara Newhouse, President and CEO of The ALS Association. “Our roadmap to treatments involves collaboration with other ALS organizations and with industry, university investigators, government agencies, pharmaceutical and biotech companies and other nonprofit organizations committed to the fight against ALS.”

During a 30-day period this summer, The ALS Association received more than $100 million in donations. Over the last few weeks, The Association has actively convened key stakeholder groups, including a panel of advisors made up of people living with ALS, to provide input into a comprehensive plan that The Association will release in early November after approval from the Board of Trustees.

The bulk of The ALS Association’s initial $21.7 million commitment—$18.5 million—will advance four new cooperative alliances for the next one to three years involving research that has been identified as critical to finding new treatments for ALS: ALS Accelerated Therapeutics (ALS ACT), The New York Genome Center, the Neuro Collaborative, and Project MinE.These projects would not have been possible without Ice Bucket Challenge donations.

There will be synergies between these four initiatives that will increase the quantity, and most importantly, the value of data openly available to the ALS research community worldwide. The ALS Association will play a pivotal role in coordinating these efforts.

Under the leadership of The ALS Association’s Chief Scientist, Lucie Bruijn, Ph.D., M.B.A., The Association has been involved in the planning of each of these new collaborations, and, in each case, has sought the advice and evaluation of leading researchers to assess scientific merit, to gather additional ideas for each project, and to ensure the maximum relevance to future therapies.

ALS Accelerated Therapeutics (ALS ACT)

ALS ACT is a novel academic-industry partnership to accelerate treatments for people living with ALS, which is able to proceed with a $10 million commitment from The ALS Association and matching gift of $10 million from The ALS Finding a Cure Foundation.

The combined contributions from The ALS Association and The ALS Finding a Cure Foundation will significantly expedite therapy development. The ALS Finding a Cure Foundation is led by Peter N. Foss, Lee Rizzuto and Denis Rizzuto. The foundation was created in April of 2014 and is a tribute to Denis’ wife, Christie Rizzuto, who was diagnosed with ALS five years ago at age 41. ALS ACT will enact a multi-pronged approach to expediting clinical trials in ALS. Efforts will include (1) developing neuroimaging tools as potential biomarkers for ALS progression, a key unmet need in trials; (2) development of therapeutic approaches to decrease production of misfolded proteins within motor neurons and reverse neuroinflammation, two major contributors to the disease process; (3) a challenge grant program to overcome key roadblocks in the search for therapies; (4) support for phase IIA pilot clinical trials using biomarkers. Merit Cudkowicz, Co-Chair of the Northeast ALS Consortium (NEALS) and Chief of Neurology and the ALS Program at Massachusetts General Hospital (MGH), notes that these efforts will accelerate diagnosis, speed development of new treatments for people with ALS, and remove road blocks to finding a cure for ALS.

In addition, ALS ACT will strengthen ongoing collaborative efforts in support of clinical trials, including NeuroBANK, a central repository for clinical research data in ALS, and the NEALS (Northeast ALS Consortium) Biorepository. Initially established through the Translational Research Advancing Therapies for ALS (TREAT ALS™) NEALS Clinical Trials Network, NeuroBANK will host, curate, and disseminate proteomic, genomic and clinical data.

Investigators: Merit Cudkowicz, M.D., MSc, Massachusetts General Hospital; Robert J. Brown, Jr. M.D., D.Phil., University of Massachusetts; Stanley H. Appel, M.D., Houston Methodist Hospital System; and Clive Svendsen, Ph.D., Cedars-Sinai; Nadeem Ishaque, Ph.D., and Tom Gentile Senior Vice President General Electric.

New York Genome Center (NYGC)

The ALS Association and its Greater New York Chapter will match a $2.5 million gift from The Tow Foundation in support of a new project at the NYGC, which will bring together for the first time a world-class scientific team to further understand the genetic basis of ALS. Recent discoveries have indicated that genes may contribute to a much larger percentage of ALS cases than previously thought. Finding these genes and understanding how they work will allow development of new therapeutic approaches. Under the leadership of Drs. Robert Darnell and Hemali Phatnani together with their advisors, including Tom Maniatis and Marc Tessier-Lavigne , the NYGC will spearhead a cooperative and multidisciplinary effort to provide open-source “big data” to benefit the entire ALS research community.

Investigators: Robert B. Darnell, M.D., Ph.D.; Hemali Phatnani, Ph.D.; Tom Maniatis, Ph.D.; Marc Tessier-Lavigne, Ph.D.; Merit Cudkowicz, M.D., MSc; Robert J. Brown, Jr. M.D., D.Phil.; Virginia Lee, Ph.D.; John Q. Trojanowski, M.D., Ph.D., Alex Sherman; James Berry, MD; Neil Shneider, M.D., Ph.D.; David Goldstein, Ph.D.; and Tom Jessell, Ph.D.

Neuro Collaborative

The Neuro Collaborative will combine the efforts of three world renowned California labs focused on ALS: Cedars-Sinai in Los Angeles, the University of California San Diego and the Gladstone Institutes, an affiliate of the University of California San Francisco, to develop and expedite therapeutic approaches for ALS. The collaborative, which now has funding to proceed with $5 million in Ice Bucket Challenge funding from The ALS Association, will focus on the following: (1) development of antisense therapy for the C9orf72 gene, the most common genetic cause of ALS, in partnership with Biogen-Idec and ISIS Pharmaceuticals; (2) gene therapy to down regulate SOD1, the second-most common ALS gene; (3) establishment of a Stem Cell and Motor Neuron Core Facility to create clinical-grade induced pluripotent stem (iPS) cell lines, which will be openly shared with the ALS research community. iPS cells have emerged as a key research tool and potential source of therapeutic cells in ALS. 4) Using a novel screening tool, identify new targets for drug development and in partnership with the industry these leads will be developed further with the potential of new treatment options in the clinic. The Golden West Chapter of The ALS Association has played a key role in the planning and development of this project.

Investigators: Don Cleveland, Ph.D. UCSD; Steven Finkbeiner, M.D., Ph.D., the Finkbeiner lab at the Gladstone Institutes; Clive Svendsen, Ph.D., Cedar Sinai Medical Center; and collaborators, Martin Marsala, M.D., UCSD; and Brian Kaspar, Ph.D., Children’s Hospital and Ohio State University.

Project MinE

Project MinE is a global collaboration with the goal to sequence the genomes of at least 15,000 people with ALS in an effort to discover new genes that affect ALS, either increasing the risk for the disease or protecting against it. The ALS Association’s funding commitment of $1 million will enable Project MinE to expand to the United States.

It has become clear that risk for ALS is likely influenced by variants in multiple genes, each of which is relatively rare. The development of advanced sequencing technologies has dramatically reduced the cost of screening large numbers of individuals for these rare variants. Discovering these variants, and understanding how they contribute to disease, or protect against it, is likely to lead to novel approaches to ALS therapies.

U.S. investigators: John Landers, Ph.D. University of Massachusetts, Worcester; Jonathan Glass, M.D., Emory University.

International partners include the Netherlands, United Kingdom, Ireland, Spain, Portugal and Belgium.

In addition to the abovementioned collaborative alliances to drive forward ALS research, The ALS Association’s Board of Trustees also approved two other projects to expedite the search for treatments and a cure for the disease through care services and public policy efforts.

Grants to Certified Treatment Centers of Excellence

The ALS Association and its network of chapters currently partner with 43 ALS Association Certified Treatment Centers of Excellence across the United States. Multiple studies have shown the value to a patient of attending a multidisciplinary clinic, including longer survival, increased quality of life, and improved access to potential therapies. One of the requirements in achieving certification through The ALS Association is for the institution to be actively involved in ALS-related research and to provide information to people living with the disease on research outside of their institution. Participation in clinical trials is imperative to the research process to find treatments for the disease. The Ice Bucket Challenge donations have enabled The Association to increase its annual grants to the centers from the presently budgeted $12,500 to $25,000 per center, which was the funding level pre-2008, for the next three years.

Regulatory Guidance to Expedite Drug Development

Organizations involved in two other neurological diseases (Alzheimer’s and Duchenne Muscular Dystrophy) have seen great benefits in working to develop guidance for companies to help them navigate the regulatory pathway for approval of effective therapies. The enactment of the patient-focused drug development elements of the Food and Drug Administration Safety and Innovation Act (FDASIA) presents a unique opportunity for The ALS Association to help expedite drug development by developing similar guidance for ALS. No such guidance exists today for ALS, which creates uncertainty and risk for what is already a difficult and costly process. By developing this guidance, The ALS Association will be able to build on and strengthen its engagement with the FDA, industry and people with ALS about drug development as a regulatory process, which will reduce obstacles that can slow and limit innovation and access to effective treatments.

About The ALS Association

The ALS Association is the only national non-profit organization fighting Lou Gehrig’s Disease on every front. By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through Certified Treatment Centers of Excellence, and fostering government partnerships, The Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure. For more information about The ALS Association, visit our website at

About The ALS Association

The ALS Association is the only national non-profit organization fighting Lou Gehrig’s disease on every front. By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, The Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure. For more information about The ALS Association, visit our website at

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