Challenge Me to Halt the Progression of ALS

By Stanley Appel, M.D.
Co-director, Houston Methodist Neurological Institute
Chair, Stanley H. Appel Department of Neurology

I call ALS “the nice guys’ disease.” For, as indiscriminate as it appears, ALS certainly has a way of finding the best and most courageous people to attack. Watching people endure the wasting of their muscle control and seeing them defiant in the face of the sure and steady loss of communication and independence, makes me defiant too. People with ALS fight the disease with great courage. Seeing the fire and determination of my patients inspires me every day. Our team puts that inspiration to work in the clinic and in my lab, where we are on a promising path to finding effective therapies to slow ALS.

The money you generated by accepting the ALS Ice Bucket Challenge has changed the treatment and care landscape for people living with ALS. It has funded groundbreaking research, giving the medical and pharmaceutical communities more insight into how ALS progresses. These discoveries help us narrow treatment targets that we can test in our labs – and in more clinical trials – in more places across the country. In fact, The ALS Association has been able to increase the number of Certified Treatment Centers of Excellence by 29 and Recognized Treatment Centers by 20. This makes better care more accessible to more people fighting ALS.

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Challenge Me to Find More ALS Genes

By John Landers, Ph.D.
University of Massachusetts Medical School

Furthering our understanding of disease and the creation of effective therapies won’t happen in a vacuum. My peers and colleagues in the medical research community know first-hand the necessity of global collaborations that bring diverse specialties into the process of understanding complex medical mysteries such as ALS.

A few months before the ALS Ice Bucket Challenge, my colleagues and I approached The ALS Association about investing in the work of Project MinE, a global research consortium focused on understanding the genetics of ALS. Although the Association had a deep interest in advancing work to understand the genetic factors associated with ALS, resources to participate in a significant way were constrained. Genetics research is expensive, and there simply was not sufficient budget to establish a Project MinE branch in the United States.

But gallons upon gallons of ice water later, everything changed.

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Challenge Me to Make the Impossible Possible

By Dexter Ang
Co-founder & CEO, Pison Technology

Watching my mother struggle to interact with the world as her ALS progressed was extremely difficult for our family, and for me personally. Her struggle inspired me to find solutions that could help people like my mother better interact with computers and their environment, even after they have lost almost all of their ability to move. I had the motivation to advance technology in a way that would bring a new quality of life to people living with ALS under Steve Saling’s motto that until medicine proves otherwise, technology is the cure.

Thanks to everyone who contributed to the Ice Bucket Challenge, funds were made available to my company when we won The ALS Association’s Assistive Technology Challenge in 2016. These funds were used to launch my company, Pison Technology, and take my ideas for assistive technology from concept into development. I am grateful every day for the millions of people who participated in the Ice Bucket Challenge and created a whole new environment for those of us working to empower people living with ALS to live more fulfilling lives.

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Understanding the Impact of the Ice Bucket Challenge on The ALS Association’s Finances

The ALS Association has always been committed to transparency, including providing complete and accurate financial information on our website. We continue to receive the highest ratings from charity watchdog groups tasked with reviewing our spending, fundraising, and management.

The Ice Bucket Challenge generated $115 million for the national office of The ALS Association in 2014, which spurred a massive increase in the Association’s capacity to invest in promising research, the development of assistive technologies, and increased access to care and services for people with ALS. Since 2014, we have committed more than $131 million toward our mission, including over $89 million committed specifically to worldwide research collaborations.

Since that time, the Association has devoted a page on our website to provide a list of how those funds are being spent. You can always find that information by clicking on the “Ice Bucket Challenge Progress” button on our homepage, or you can view those investments here.

After the Ice Bucket Challenge in 2014, The ALS Association’s Board of Trustees determined that the best use of these proceeds was to immediately put them to work by increasing funding in our core mission areas. As a result, we doubled our operating budget, with most of the increase allocated to research and care service initiatives.

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Challenge Me to Revolutionize the Treatment of ALS

By Don Cleveland, Ph.D.
Ludwig Institute for Cancer Research, University of California, San Diego

There was a lot of skepticism in the research community when we proposed development of designer DNA drugs as a way to treat ALS. This therapy is an approach that uses DNA-based designer drugs to “silence” genes that are known to cause a particular disease. The ALS Association was the first funding organization to invest in designer DNA technology research and development for therapy in neurodegenerative disease.

The ALS Association, from the beginning to the present, has been the boldest supporter of designer DNA drug technology and its support has been transformative for the development of promising new therapies. With ALS Ice Bucket Challenge donations, generated by people like you, the Association has been able to double down on its investment in designer DNA drugs. These include trials underway for the two most common genetic causes of ALS, mutation in the SOD1 or C9orf72 genes. Designer DNA drugs to silence either of those genes are now in clinical trials and we’re encouraged by the data gathered thus far. Ice Bucket support also enabled identification of new gene targets that are affected in essentially all ALS patients and that are ideal targets for designer DNA drugs.

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Meet Jorge Gomez-Deza: A 2018 Milton Safenowitz Postdoctoral Fellow

Our Milton Safenowitz Postdoctoral Fellowship Program continues to support young scientists and is the only program of its kind specifically funding early ALS postdoctoral fellows.

The awards were founded in memory of Mr. Safenowitz by the Safenowitz family – through The ALS Association Greater New York Chapter. The program encourages young scientists to enter and, importantly, to remain in the ALS field.

We are proud that 76 percent of the postdoctoral fellows we fund go on to start their own labs to continue studying ALS and mentor other young ALS researchers. The rest of our Safenowitz fellowship program graduates go on to careers in the biomedical industry, nonprofits, and medical writing, with many still staying in the ALS space.

This year, we are supporting five new postdoctoral fellows out of a highly competitive applicant pool. Over the next few weeks, we will highlight each fellow – their dedication and unique contributions to ALS research, as well as their interests outside of the lab.

We recently talked with Dr. Jorge Gomez-Deza with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)/National Institutes of Health (NIH) to learn about his unique research project, which has the ultimate goal of identifying a druggable target that prevents neuronal death.

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FAQ: Biogen is Now Enrolling in Phase 3 VALOR Clinical Trial to Test Safety and Efficacy of Tofersen

Biogen has initiated a phase 3 clinical trial evaluating tofersen (previously called BIIB067), an antisense oligonucleotide (ASO), a type of antisense drug, targeting superoxide dismutase (SOD1), for the potential treatment of ALS. The trial is now enrolling and aims to enroll approximately 60 people with SOD1 ALS

At this week’s 2019 American Academy of Neurology (AAN) Annual Meeting in Philadelphia, Biogen presented promising results of their phase 1/2 SOD1-ALS trial (tofersen – BIIB067). They were encouraged by the initial findings of the study, so they are moving into a larger phase 3 trial to further test the safety and efficacy of tofersen. While this news is promising, it is important to note that the drug (tofersen) is still experimental.

Continue reading FAQ: Biogen is Now Enrolling in Phase 3 VALOR Clinical Trial to Test Safety and Efficacy of Tofersen