The ALS Association and coalition partners call for quick review of ACA by SCOTUS

The ALS Association and 23 partners that represent millions of patients across the country released a statement strongly supporting the petitions filed today by state attorneys general and by the U.S. House of Representatives that seek expedited U.S. Supreme Court review of last month’s Texas v. United States ruling. The ruling endangers patient access to adequate, accessible and affordable health care.

Continue reading The ALS Association and coalition partners call for quick review of ACA by SCOTUS

Remembering Pete Frates, Co-Founder of the ALS Ice Bucket Challenge

With heavy hearts, The ALS Association joins the ALS community in celebrating the legacy and mourning the loss of Pete Frates, who died Monday at age 34 after a seven-year battle with ALS.  

Pete lived a Hall of Fame life. 

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GUEST BLOG POST: The Promise of CuATSM for ALS and the Challenges of Moving from Mice to Humans

Note: The ALS Association was recently contacted by Dr. Joe Beckman, Ph.D., who had been following recent conversations online about CuATSM and wished to help provide further context about the compound and potential treatment. 

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. 

For several years, we have been overwhelmed with the patient requests from young mothers and fathers with kids and family members with parents who are anxious to try CuATSM and learn of any possible treatment. It is hard to convey the horror of this disease, and I understand their frustration. A good friend from my time in the Army 40 years ago has developed the disease. After 25 years of work on the disease, I feel I have failed so far. This disease is humiliating in its complexity. The one lesson I have learned is that there are no simple answers or short cuts. 

Continue reading GUEST BLOG POST: The Promise of CuATSM for ALS and the Challenges of Moving from Mice to Humans

Remembering Our Beloved Friend and Leader, Stephen Winthrop, Chair, The ALS Association Board of Trustees

It is with heavy hearts that we share that Stephen Winthrop, Chair of The ALS Association Board of Trustees and person with ALS, died peacefully early Monday morning. Our hearts are with his wife, Jane, and their two daughters.

Stephen was diagnosed with ALS on November 6, 2013. Stephen and Jane immediately began working to help raise awareness and support for the fight against ALS. They set up a Facebook page – WillWinAgainstALS – to “advocate, raise awareness, and fundraise to wipe out ALS!”

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Respiratory Strength Training Deemed Safe and Feasible in ALS Case Study

Dr. Emily Plowman, one of our Clinical Management Grant awardees, recently published a promising case report on a person with early-stage ALS who has been participating in a mild-intensity respiratory strength training program. The individual has been doing the strength training program with Dr. Plowman and her team at the University of Florida in Gainesville, Florida, over the past 24 months.

Dr. Plowman, associate professor in University of Florida’s Department of Speech, Language and Hearing Sciences and Neurology, and her colleague, Dr. James Wymer, set out to explore, for the first time, how both expiratory muscle strength training (EMST) and inspiratory muscle training (IMST) impact a person with ALS. They found that the mild-intensity program was safe and improved function in a person with ALS who was early in disease progression.

ALS is a devastating disease that eventually attacks a person’s bulbar muscles of the head and neck, impacting speech, coughing, and the life sustaining functions of swallowing and breathing. Impairments in breathing and swallowing contribute to fatal aspiration, malnutrition, and respiratory issues that account for 92 percent of ALS mortality.

The mild-intensity training program involves daily exercises for both the inspiratory and expiratory muscles. These muscles are responsible for the vital functions of breathing and ventilation (inspiratory muscles), as well as airway clearance/protection and cough function (expiratory muscles) using a handheld trainer.

In an earlier study, Dr. Plowman found that EMST was well-tolerated in people with ALS and led to improvements in maximum expiratory pressures, airway clearance, and swallowing measures.

We asked Dr. Plowman to share her thoughts on respiratory training and her newly published case study.

Continue reading Respiratory Strength Training Deemed Safe and Feasible in ALS Case Study

Clinical Trial: Orphazyme to Enroll 231 People with ALS in Phase III Arimoclomol Clinical Trial

The ALS Association Supported Research In Arimoclomol Development

Our Every Drop Adds Up campaign builds on the idea that people coming together for a common goal can make the impossible happen, just as it did four summers ago when the ALS Ice Bucket Challenge soaked the world. Every person helped, research project funded, story shared, discovery made, piece of legislation passed, and auction bid placed – it all adds up!

Biopharmaceutical company Orphazyme A/S launched a phase III clinical trial of arimoclomol, giving a dose to a person with ALS for the first time, the company announced recently. The randomized and placebo-controlled trial will enroll 231 people with ALS in North America and Europe to determine efficacy and safety of the oral drug.

Dr. Michael Benatar, executive director of the Kessenich Family ALS Center at the University of Miami and principal investigator of the study, presented data of the successful phase II clinical trial testing arimoclomol in patients carrying SOD-1 mutations at the International ALS MND Symposium in Dublin in December 2016.

The ALS Association, along with the U.S. Food and Drug Administration’s (FDA) Orphan Products Development Program, provided funding for the trial. The study was not powered to determine therapeutic effect; however, the data demonstrated safety and preliminary evidence for efficacy, which led to this new phase III trial. The open access data was later published in Neurology in January 2018.

“On behalf of the patients struck by this devastating disease, I am delighted to see this Phase III trial up and running. The ALS Association invested in the early clinical trials and are pleased to see this moving forward to a Phase III study and to seeing the potential of arimoclomol unfold.” (Dr. Lucie Bruijn, Chief Scientist, The ALS Association)

Continue reading Clinical Trial: Orphazyme to Enroll 231 People with ALS in Phase III Arimoclomol Clinical Trial

Throwback Thursday: 2017 Mission Accomplishments

The ALS Association fights for people with ALS every day, leading cutting-edge research to discover treatments and a cure for ALS, and serving, advocating for, and empowering people with the disease to live their lives to the fullest.

In honor of Throwback Thursday, let’s look back at the advances in our mission areas of Advocacy, Care Services, and Research during 2017.

Continue reading Throwback Thursday: 2017 Mission Accomplishments