The ALS Association Help Find A Cure

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ALS Ice Bucket Challenge Commitments

The ALS Association is committed to transparency in how donor dollars are helping to fuel efforts to find treatments and a cure for the disease. In this graphic, we have illustrated anticipated spending over several years in key areas including research, patient and community services, public and professional education.

Below, you will find links and dollar figures associated with all of the projects and initiatives announced by The ALS Association that we have committed to fund using money raised during the 2014 ALS Ice Bucket Challenge.

To date, The ALS Association has announced $47.1 million* on specific projects and initiatives out of a total of $115 million donated. This figure will be updated as new commitments are added in the coming weeks and months. For questions, please contact inquiries@alsa-national.org.

Patient and Community Services

Grants to Treatment Centers - $2,700,000

Community and Support Grants - $8,500,000

Public and Professional Education

Regulatory Guidance to Speed Drug Development - $500,000

Research

ALS ACT - $10,500,000

New York Genome Center - $2,500,000

Neurocollaborative - $5,000,000

Project Mine - $1,000,000

Clinical Management Grant - $50,000

Clinical Research Training Fellow - $86,667

Biomarker Discovery and Validation Projects - $1,199,841

Cytokinetics, Inc. Phase 3 Trial - $1,500,000

2015 Annual Research Awards - $11,621,638

* Total includes $2 million in already paid external processing fees for credit card transactions and web overage charges due to increased volume of daily web visitors during the 2014 ALS Ice Bucket Challenge.


 

Grants to Treatment Centers - $2,700,000

The ALS Association and its network of chapters currently partner with 48 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. As announced last October 2014, the ALS 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.

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Community Support Grants - $8,500,000

The ALS Association has 39 chapters in communities across the country that are providing care and support to people living with ALS. The ALS Association gave $8.5 million in funding to chapters to augment local programs and services to people living with ALS. Chapters have been able to accomplish the following: restock and expand equipment loan closets; expand services into Idaho and Utah; launch a telemedicine program to help home-bound patients; and increase patient access to care. Other stories of how chapters are making a difference can be found here.

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Regulatory Guidance to Speed Drug Development - $500,000

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. As announced in the press release in October 2014, 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.

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ALS ACT - $10,500,000

In October 2014, The ALS Association committed a total of $10 million to ALS ACT, a novel academic-foundation-industry partnership to accelerate treatments for people living with ALS. (The Association put in an additional $500,000 after this initial commitment was made in order to fund one additional clinical trial.) In partnership with the recently formed The ALS Finding a Cure Team, composed of researchers from General Electric (GE) Healthcare and four academic Northeast ALS Consortium (NEALS) sites, ALS ACT will enact a multi-pronged approach to expediting clinical trials in ALS. The ALS Association’s funds are being matched by The ALS Finding a Cure Foundation, for a total of $20 million. The projects below have been peer reviewed and approved, and represent commitments from both organizations:

Alex Sherman, Merit Cudkowicz, M.D., Massachusetts General Hospital, $650,000
NeuroBANK as an Accelerated Clinical Research Environment: Development, Deployment and Services to ALS Research Community

Successful implementation of NeuroBANK™ will allow for a standardized patient-centric approach to clinical research in ALS with information linked across studies, locations and modalities. Standard Common Data Elements, standard operating procedures and GUID technology will lead to accelerated studies' review, approval, deployment and patient enrollment

Clive Svendsen, Ph.D., Cedars-Sinai, $1,913,025
Inflammatory Biomarkers, Stem Cells and DNA in people with ALS

The goal of this project is to identify biomarkers of ALS that will gives us clues to disease mechanisms and help us identify targets for ALS therapy development.  Specifically, we are planning to collect blood samples and skin biopsies from 50 people with ALS and 50 healthy volunteers. We will then use the blood samples to identify inflammatory markers of ALS and perform detailed DNA analysis. The skin biopsies will be used to generate stem cells from people with ALS and compare them to the stem cells from healthy volunteers. At the end of this project, we will identify inflammatory signatures of ALS and new targets and tools for drug development.    

Clive Svendsen, Ph.D., Cedars-Sinai (in partnership with General Electric), $268,384
Application of MultiOmyx to iPSC Models of ALS

Induced pluripotent stem cell technology (iPSC) holds great promise for accelerating our understanding of the molecular mechanisms and pathways leading to motor neuron degeneration in ALS. In vitro cellular model systems generated from patient-derived iPSC lines can recapitulate many aspects of in vivo cellular pathology, and beyond basic disease research may serve as a powerful test bed to screen potential therapies. Therapeutic screening on iPSC-derived models will likely be complimentary to, and in some aspects superior to, other approaches including animal models in terms of human-predictivity and speed.  

GE has developed a unique technology platform allowing the in vitro analysis of cells and tissues to an extent not previously possible (MultiOmyx, or “MO”).  An iterative labeling process allows repeated immunocytochemical visualization of 60 or more protein markers in a single sample, as well as RNA and DNA. Precision hardware and custom software for imaging and analytics enables extensive characterization of single cell structure and function as well as relationships with neighboring cells and environment.  Application of this technology to ALS iPSC systems is likely to advance the utility of these models and provide new insights into neurodegenerative pathways and mechanisms.  

Clive Svendsen, Ph.D., Cedars-Sinai (in partnership with General Electric), $389,643
Using Novel Imaging Agents as a Biomarker for ALS

Assessing the progression of ALS in both animal models and the human condition would provide the ideal biomarker for testing novel compounds rapidly and efficiently.  However, while MRI and other imaging modalities exist they have not been fine tuned for this neurological condition.  Our rational is that through better imaging of disease progression in ALS within individual patients, drug therapy throughput and success will be increased. 

We have just completed an extensive study mapping the exact progression of disease in the G93A rat model of familial ALS (fALS). Remarkably, this animal model of ALS reproduces many aspects of the disease including stochastic loss of motor neurons that could start in either cervical (20%) or lumbar (80%) regions of the spinal cord - mirroring the situation in patients. 

GE Global Research has developed a novel superparamagnetic iron oxide (SPIO) nanoparticle for MR imaging of neuroinflammation. The agent was shown to be efficacious in brain and spinal cord imaging of acute neuroinflammation in an experimental autoimmune encephalomyelitis (EAE) model in the rat, with MR signal correlated with macrophages present within the CNS.  

James Berry, M.D., Massachusetts General Hospital, $1,000,000
Infrastructure Improvement and Biofluid Collection Protocols for the Expansion of the NEALS Biorepository

The exact cause of amyotrophic lateral sclerosis remains unknown, and there are no tests to diagnose the disease or follow its progression.  Such tests, known as biomarkers, would be invaluable to speed the development of novel therapies for ALS.   In recent years, ALS scientists have begun to identify promising potential biomarkers in the blood and spinal fluid of people with the disease.  The next step to build on these discoveries, in many cases, is to test larger numbers of biofluid samples to confirm the findings.   

The Northeast ALS Consortium (NEALS) biorepository is a collection of biological samples, including blood and spinal fluid obtained from people with ALS that was created to be a resource for scientists conducting research in ALS.  This project will allow us to upgrade the NEALS biorepository infrastructure, including updating the computer systems, expanding the number of sample freezers, and revising the standard operating procedures for the repository.  The project also supports two efforts to expand the biorepository by collecting new blood and spinal fluid samples from people with ALS.  This will ensure the NEALS biorepository remains a vital resource to the ALS scientific community. 

Robert Brown, University of Massachusetts, $2,500,000
Development of AAV-Mediated SOD1 Gene Silencing Therapy in ALS

About 10 percent of ALS cases are familial.  Most of the known genetic defects in familial ALS act by triggering one or more toxic processes that impair the viability of motor neurons, leading to motor neuron death.  This project will use viruses to introduce reagents into the brain and spinal cord that block the mutant genes from triggering toxicity.  This is accomplished by turning off the activity of those genes using new technologies for so-called “gene silencing.”  The viruses are used to penetrate the blood-brain barrier to deliver the gene silencing reagents, which then inactivate the genes in question.  This technology will be tested first in cases of ALS arising from mutations in well-defined ALS genes (e.g. SOD1 and C9orf72).  This project is intended to take the gene silencing all the way to a pilot human trial.  We are hopeful that in the long term these studies will lead to a general platform or method for treating other types of familial ALS and potentially also some cases of non-familial ALS.

Nazem Atassi, M.D., Massachusetts General Hospital (in partnership with General Electric), $3,929,181
TRACK ALS

This is collaborative project between industry, academia, the Leandro P Rizzuto (ALS Finding a Cure (ALSFAC)) Foundation and The ALS Association. The overall goal of this project is to identify imaging markers of ALS that can be used to accelerate ALS diagnostic timelines and the pace of ALS drug discovery. This project will focus on studying the role of brain inflammation in people with ALS.   

In this project we plan to use innovative MRI and positron emission tomography (PET) tracer (contrast) that will show us the location and degree of nerve damage and brain inflammation in people with ALS.  We plan to compare MRI signals between 50 people with ALS and 50 healthy volunteers and to study these MRI signals over time in relationship with ALS clinical presentation and disease progression. In a subgroup of patients, we plan to compare brain inflammation between 20 people with ALS and 20 healthy volunteers and to study brain inflammation over time in relationship with ALS clinical presentation and disease progression.  

At the end of this project, we will identify the imaging inflammatory signature of ALS as a potential tool for early diagnosis and quick read out of drug efficacy in people with ALS.

Stan Appel, M.D., Methodist Neurological Institute, $2,270,585
Pilot Trial of the Safety and Tolerability of Expanded Autologous Regulatory T Cells Administered by Intravenous Infusion in People with ALS

The purpose of this study is to isolate T regulatory (Treg) cells from the blood using leukapheresis and expand Treg cells in the laboratory in twelve people with ALS. Leukapheresis is a laboratory procedure in which white blood cells are separated from a sample of blood. Once separated, the remainder of the blood is returned to the circulation.  The subject’s Treg cells will be grown (manufactured) in a specialized GMP laboratory facility, tested and purified. The subject’s own expanded Treg cells will then be injected into the skin with or without the addition of IL-2 (Interleukin-2).This study may lead to Treg cell therapy for symptom management or potential slowing of disease progression in ALS and related disorders.

Two Clinical Phase II Trials, $3,000,000

As discussed in the July 2014 press release, The ALS Association announced $3 million of funding of two Phase II clinical trials as part of ALS ACT. These include the following:

Robert Miller, M.D., California Pacific Medical Center, San Francisco, CA
A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of NP001 in Subjects with Amyotrophic Lateral Sclerosis (ALS)

In collaboration with NEALS and Neuraltus Pharmaceuticals, Inc., Robert Miller, M.D. of the California Pacific Medical Center will lead a placebo-controlled, six-month treatment study to confirm a signal observed in a previous study of NP001, an immune system regulator. Inflammation is thought to be a contributing factor to ALS disease progression and existing NP001 data suggest that it may have an effect on inflammation in the immune system.

Michael Weiss, M.D., of the University of Washington Medical Center in Seattle
Effects of mexiletine on cortical hyperexcitability in amyotrophic lateral sclerosis

In collaboration with NEALS, Michael Weiss, M.D., of the University of Washington Medical Center in Seattle, will lead an eight-week study comparing the effect of several dosages of mexiletine on markers of hyperexcitability. A prior study showed that mexiletine was safe to use in ALS.

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New York Genome Center - $2,500,000

Per the October 2, 2014 announcement, The ALS Association is funding the New York Genome Center’s Consortium for Genomics of Neurodegenerative Disease (NYGC CGND). The New York Genome Center is a state-of-the-art consortium, possesses the capability of generating and analyzing thousands of ALS patient DNA sequences. NYGC brings a wealth of knowledge combining the latest technology with esteemed Institutional Founding Members, Associate Members, a Founding Technology Member and internal faculty with joint appointments at the member institutions. The mission of the NYGC-CGND is to harness state-of-the-art genetic, genomic and bioinformatics tools to gain insights into motor neuron disease mechanisms and to use this knowledge to identify new diagnostic and therapeutic approaches to these devastating diseases.

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Neurocollaborative - $5,000,000

As discussed in the October 2, 2014 announcement, The Neuro Collaborative will combine the efforts of three leading California laboratories focused on ALS: The Svendsen lab at Cedars-Sinai in Los Angeles, the Cleveland lab at the University of California San Diego, and the Finkbeiner lab at the Gladstone Institutes, which is affiliated with UCSF.

The project is made possible by the unprecedented outpouring of support from the Ice Bucket Challenge. The Neuro Collaborative is one of four major new initiatives by The Association as a direct result of that support. The goal of the Neuro Collaborative is to discover and develop potential new therapies for ALS, which can be delivered to pharmaceutical companies for further development in clinical trials. Early development of potential therapeutics is a major bottleneck in ALS therapy development and represents a significant opportunity for accelerating new treatments.

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Project Mine - $1,000,000

As discussed in the October 2, 2014 announcement, The ALS Association has committed $1 million to Project MinE, an international effort to sequence the genomes of at least 15,000 people with ALS. The funding will be used to bring this effort to the United States, under the direction of researchers at University of Massachusetts Medical School in Worcester, Mass., and Emory University in Atlanta, Ga.

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Clinical management Grant - $50,000

Melinda Kavanaugh, Ph.D., Assistant Professor of Social Welfare at the University of Wisconsin in Milwaukee, $50,000

On May 12, 2015 The ALS Association announced funding of a study of young people who care for someone with ALS to better understand the needs of youth caregivers and to design support services to address those needs. While the proportion of families with a teen or child caring for a person with ALS is unknown, it is clear that many ALS families include youth who may assume caregiver roles. The new study will collect data on ALS families nationwide and conduct interviews with youth caregivers to better understand their experiences.

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Clinical Research Training Fellow - $86,667

The ALS Association and the American Academy of Neurology (AAN) announced that Hristelina Ilieva, M.D., Ph.D., of the Department of Neurology at the Johns Hopkins School of Medicine, Baltimore, Md., is this year’s recipient for the Clinical Research Training Fellowship. The purpose of the award is to recruit talented and promising young clinicians who propose innovative clinical research and to foster their development to make significant contributions to ALS clinical research.

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Biomarker Discovery and Validation Projects - $1,199,841

On June 3, The ALS Association announced the awarding of $1.2 million of new funding to advance the discovery of biomarkers that correlate with important clinical and pathological aspects of ALS disease progression. This funding will be paired with a $539,000 match donation intended for research. The awards will fund three projects, each of which focuses on the discovery of biomarkers in ALS:

Jeremey Shefner, M.D., Ph.D., Robert Bowser, Ph.D., and Shafeeq Lahda, M.D., of Barrow Neurological Institute in Phoenix, Arizona, $539,841
Expansion of NEALS Biorepository at Barrow Neurological Institute, Phoenix Ariz. and Deep Phenotyping

This project will carry out a longitudinal collection of blood and Cerebrospinal fluid (CSF) from 150 people with ALS over the course of their disease. At the same time, detailed clinical measurements will be made of respiratory function, cognitive function, muscle strength, and other aspects of the disease. The fluids and clinical data will be stored in the NEALS biorepository and be made available for biomarker discovery research. The NEALS biorepository is a major site for storage, curation, and research on ALS biosamples. This project will be led by Jeremey Shefner, M.D., Ph.D., Robert Bowser, Ph.D., and Shafeeq Lahda, M.D., of Barrow Neurological Institute in Phoenix, Arizona. This research project is also supported by a generous matching gift from Mary Lou and Ira Fulton.

Andreas Jeromin, Ph.D., and Robert Bowser, Ph.D., of Iron Horse Diagnostics of Scottsdale, Ariz., co-funded by the National Institutes of Health, $200,000
Biomarker Validation Study

This study will attempt to provide a validation of a proteomic signature of ALS in the CSF of 300 people with the disease. The validation study will build on previous results showing that the ratio provides 92 percent sensitivity and 94 percent specificity for presence of ALS. Iron Horse will further validate these tests and perform both a prospective clinical study and assay qualification within a commercially certified (CLIA) laboratory to commercialize the diagnostic tests. Validation of the biomarker would allow further development and, ultimately, the first commercially available clinical test for presence of ALS.

National Institute of Health Rare Diseases Clinical Research Consortium, the Clinical Research in ALS and Related Disorders for Therapeutic Development Consortium (CReATe), $460,000
Biosample Collection for CReATe

This collection will support the growth and expansion of a biorepository for a newly funded National Institute of Health Rare Diseases Clinical Research Consortium, the Clinical Research in ALS and Related Disorders for Therapeutic Development Consortium. CReATe will bring together biochemists, geneticists, clinicians, Pharma, and partner groups involved in patient education and advocacy including The ALS Association, Muscular Dystrophy Association, ALS Recovery Fund, Association for Frontotemporal Degeneration, Spastic Paraplegia Foundation, PatientsLikeMe, and the National ALS Registry. The successful completion of the CReATe Consortium objectives will lay the necessary foundation for future trials in genetically homogeneous patient populations (including, for example, people with C9orf72 repeat expansions), thereby advancing science towards development of effective therapies for patients afflicted with these devastating degenerative disorders. As part of this initiative The ALS Association will support the enhancement of a repository of biosamples collected longitudinally with deep phenotyping of approximately 700 people with ALS. This effort, led by Michael Benatar, M.D., Ph.D., of the University of Miami, will combine detailed clinical data with biosample analysis to search for biomarkers that correlate with clinical characteristics, including patterns of disease spread and the rate of disease progression. Funding by The ALS Association will allow more extensive samples to be collected, stored, and shared with the ALS research community.      

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Cytokinetics, Inc. Phase 3 Trial - $1,500,000

In July 2015, The ALS Association announced $1,500,000 in funding to Cytokinetics, Inc. to support the collection of clinical data and plasma samples to advance the discovery of biomarkers in ALS in VITALITY-ALS, a Phase 3 clinical trial of tirasemtiv in patients with ALS. For the first time, this unique collaboration between Cytokinetics, The ALS Association, and the Barrow Neurological Institute will enable plasma samples collected from patients enrolled in a Phase 3 clinical trial to be added to The Northeastern ALS Consortium (NEALS) Repository, a resource for the academic research community to identify biomarkers that may help to assess disease progression and underlying disease mechanisms in ALS.


 

2015 Annual Research Awards - $11,621,638

In July 2015, The ALS Association announced its support of 58 new research grants totaling $11,621,638 million to find treatments and a cure for ALS. These awards include investigator-initiated grants, drug development contracts, Milton Safenowitz Postdoctoral Fellowships and support of the NEALS/TREAT ALS™ Clinical Trials Network. Descriptions and dollar amounts for each grant can be found here.

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