Last week Congress passed, and the president signed, a continuing resolution that will fund all federal programs at current levels through November 21. Before the continuing resolution expires, Congress must either pass full appropriations bills for the 2020 fiscal year or pass another continuing resolution.

The ALS Association has published community guidelines for our social media channels. We review and update them regularly to stay current with best practices and the expressed wishes of our community.

National Malnutrition Awareness Week kicks off today. The ALS Association has resources available to help make sure people living with ALS do not struggle with malnutrition as the disease progresses.

On Monday, the Food & Drug Administration (FDA) issued a finalized ALS Drug Development Guidance for Industry. This is one important step in comprehensive efforts to bring therapies to people with ALS more quickly. Five years ago, there was no clarity around what the FDA expected from companies pursuing ALS treatments. The ALS Association recognized this problem, galvanized the broader community, and engaged the FDA to provide a clear roadmap that also can respond to new science as it emerges. The FDA Guidance is one part of a commitment that will not be complete until we have a cure for all people with ALS.

The ALS Association has awarded $225,000 in scholarships to 45 inaugural recipients of the Jane Calmes ALS Scholarship Fund. The Fund provides financial support to students impacted by ALS.

Research funded by The ALS Association found that defects in the mechanism that transports proteins and RNA between the nucleus and cytoplasm of cells within the human body may lead to ALS and that regulation of those systems could present a novel therapeutic strategy for ALS.

The ALS Association is inviting researchers to submit proposals for its Managing ALS Research Program and The 2019 Lawrence and Isabel Barnett Drug Development Program. Applicants are encouraged to apply no later than October 7, 2019.

The ALS Association is pleased to share results from our recent community survey. We were interested in hearing about programs and services that people reported as being important, reasons why people were not accessing some programs, major challenges, and issues around medications. Information gained from the survey will be incorporated in to care services planning activities and will be used to inform strategic planning, priority setting, program outcomes, and program improvements.

Diagnosing ALS is a lengthy and frustrating process for clinicians and families alike. This is due in large part to the absence of a specific biological indicator, which would signal the presence of the disease in people experiencing the onset of ALS symptoms. Unlike cancer and diabetes, which can be diagnosed and monitored through laboratory tests, ALS has no unique biological markers to confirm an ALS diagnosis.

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. 

Collaboration is critical to scientific research, which is why the Ice Bucket Challenge was so transformational to ALS research, particularly in the field of genomics.

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.