The ALS Association is deeply committed to accelerating the development of novel ALS therapies and ensuring they are accessible to those who need them. The Association is announcing today its latest strategic action with the release of Principles for Urgent, Patient-Centered ALS Clinical Trials: a series of recommendations to clinical trial sponsors and regulatory bodies worldwide.
With Congress home for the August recess, The ALS Association is redoubling its efforts to build upon momentum toward achieving a long-sought policy goal – the elimination of a five-month waiting period for Social Security Disability Insurance.
Five years ago, I joined millions of people around the world and took the ALS Ice Bucket Challenge. The Challenge took America by storm and fueled the biggest social media movement in history. At least 17 million people uploaded videos to Facebook to raise awareness and donate to ALS research, and over $115 million was raised for The ALS Association.
AB Science has completed a Phase 2/3 human clinical trial of masitinib in ALS with promising results. The company published the results of its trial, conducted in Spain, in the journal Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. A confirmatory Phase 3 study will be launched later in 2019 with all locations yet to be announced.
To help provide more information about our research program, our spending decisions, our approach to certain experimental therapies, and other matters, we have put together the below Frequently Asked Questions.
One of the most significant results of the ALS Ice Bucket Challenge is the synergy it created in the ALS research community. The increase in funds the Challenge made available to research not only created more fundamental knowledge about ALS, it also spurred many global collaborations that further our capacity to identify targets and advance therapies.
The ALS Association, ALS Finding a Cure (ALSFAC), and MDA announced they have jointly awarded a clinical trial grant totaling more than $2.5 million over two-and-a-half years to leading investigators at the Houston Methodist Neurological Institute and Massachusetts General Hospital. The principal investigator is Stanley Appel, MD, co-director of Houston Methodist Neurological Institute, chair of the Stanley H. Appel Department of Neurology and the Peggy and Gary Edwards Distinguished Chair in ALS at Houston Methodist Hospital, and professor of Neurology at Weill Cornell Medical College.
Orphazyme has fully enrolled its Phase 3 clinical study of arimoclomol in amyotrophic lateral sclerosis (ALS). The Danish biopharma company hit full enrollment ahead of schedule due in large part to the efforts of the global ALS community who put out the word to people living with ALS.
Mallinckrodt is permanently halting its Phase 2b PENNANT Study (MNK14042068) investigating the safety and efficacy of a hormone injection for the treatment of ALS. Acthar Gel is a highly purified preparation of Adrenocorticotropic hormone (ACTH) injected either beneath the skin or into the muscle. It is approved by the FDA for the treatment of 19 indications including acute exacerbations of multiple sclerosis in adults.
On July 15, 2014, a small gesture of support by professional golfer Chris Kennedy on behalf of a family member living with ALS, went unnoticed by all but a few of his social media followers. Calling out his cousin Jeanette Senerchia of Pelham, NY, in support of her husband Anthony, Kennedy dumped a bucket of ice water on his head and challenged his network to either follow suit or make a donation to fight ALS. It was not until months later that the significance of his Facebook post would be credited as the start of the largest movement in medical history: The ALS Ice Bucket Challenge.
Today, CNN featured a profile on Larry Falivena, a person living with ALS since 2017. Larry is visiting all 30 Major League Baseball parks in one season to raise awareness of ALS as part of The ALS Association’s Challenge Me campaign. He began his "Iron Horse tour" on May 30.
Eighty years ago on July 4, Lou Gehrig gave one of the most famous speeches in American history. His speech marked his retirement from baseball because of his recent diagnosis of ALS. Gehrig was honored by many on the field that day, and his number 4 was retired, the first time a player had ever had his jersey retired. The New York Times called it “one of the most touching scenes ever witnessed on a ball field.”
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.
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.
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.
In 2016, my colleagues and I published some remarkable results of a new drug called CuATSM in a mouse model of ALS. Moving CuATSM from mice to humans is a long and difficult road that involves the cooperation and scrutiny of many people. The checks and balances are critical to balance the risks with the benefits of any new therapy.
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.
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.
We launched the Challenge Me campaign last week. Challenge Me picks up where the ALS Ice Bucket Challenge left off. This time, we are challenging the world to do anything and everything they can to help end ALS.