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
Yesterday, MediciNova, Inc. announced that the U.S. Food and Drug Administration (FDA) relayed positive feedback regarding its phase III clinical trial plan to test MN-166 (ibudilast) in a broad population of people with ALS. No safety issues were raised by the FDA and safety will be revisited when results are available in the phase III trial.
The ALS Association helped support the phase II trial through funding the PBR28 PET (positron emission tomography) imaging biomarker study that was conducted by Dr. Nazem Atassi at Massachusetts General Hospital in parallel to the trial. PBR28 is a biomarker that measures inflammation in the brain, which can track inflammation over time and evaluate the effects of ibudilast on brain inflammation.
Continue reading MediciNova Announces Positive FDA Feedback to Start Phase III Plan for MN-166 (ibudilast) to Treat ALS
An amazing group of people came together for Climb to Defeat ALS earlier this month. The first Team Challenge ALS team summited Mt. Elbert in Colorado on September 7 and has raised over $65,000 for The ALS Association, so far.
We asked a few team members to tell us about their experiences.
Continue reading Team Challenge ALS Climbs to Defeat ALS
The ALS community recently presented its recommendations to the U.S. Food and Drug Administration (FDA) regarding the Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment Guidance for Industry at a day-long event, called ALS Community Workshop: Therapy Development and Regulatory Pathways, which was held in Washington, D.C., on July 12. Over 90 people attended in person, with many more tuning in online.
Throughout the day, there were opportunities to make comments to the FDA and industry representatives, both in-person and online. The Association was proud to host the day to bring many ALS stakeholders together to provide targeted feedback and information to further inform the FDA’s Draft Guidance on ALS Drug Development.
The FDA’s Draft Guidance provides drug developers with recommendations for all aspects of clinical trials for ALS, from study design to risk-benefit considerations to patient selection. For many years, The ALS Association has worked closely with the FDA and members of the ALS community in the development of a comprehensive ALS Guidance to make clinical trials faster, shorter, and more responsive to special considerations inherent to people with ALS and their caregivers.
The Workshop was an opportunity to provide targeted feedback and information from the ALS community to further inform the FDA’s Draft Guidance on ALS Drug Development. After the Workshop, the Association composed a report to the FDA to address key points discussed at the Workshop and to make recommendations on how to improve the FDA’s final Guidance (listed below).
“The purpose today is to build a strong guidance together,” said Calaneet Balas, president and CEO of The ALS Association. “We will be taking the great ideas from this meeting and presenting them to the FDA for their consideration in developing the final Guidance document. We look forward to that document, and to seeing it used widely for making clinical trials faster, more effective, and more aligned with the needs of patients and caregivers. We believe that is the route to the fastest development of new treatments for ALS.”
Continue reading Members of ALS Community Gather with Representatives from FDA and Industry to Inform FDA Draft Guidance on ALS
“When someone you love becomes a memory…that memory becomes a treasure,” said Christine Caron, a participant in the Western Massachusetts Walk to Defeat ALS.
Each year, tens of thousands of people come together at Walk to Defeat ALS® events across the country with one common goal: to remember treasured loved ones and honor those who are fighting ALS every day.
The ALS Association launched Unlock ALS at Walk events this fall to provide participants with a tangible way to recognize the real reasons we Walk to Defeat ALS. At each Walk event, participants select a lanyard in one of four colors to represent their connection to ALS, receive a branded key, and join in a meaningful opening ceremony.
“Seeing all the different colored lanyards made me feel like we were one big family as we’re all going through this together,” said Roxane Baillargeon, a Western Massachusetts Walk participant who is living with ALS.
Continue reading Unlock ALS Honors the Real Reasons We Walk to Defeat ALS
By Mary Johnson, Caregiver – Western Pennsylvania
Note: Under current law, people disabled with ALS who qualify for Social Security Disability Insurance (SSDI) must wait five months before receiving SSDI benefits. Every person must wait, regardless of the level of disability or how fast the Social Security Administration (SSA) approves their claim.
The ALS Disability Insurance Access Act (S.379/H.R.1171) would eliminate that five-month waiting period for people with ALS to receive SSDI. People with ALS would receive their SSDI benefits immediately after being approved by the SSA.
The five-month waiting period for Social Security Disability Insurance (SSDI) after an ALS diagnosis has severely impacted my family and I’m mad as hell about it. There’s absolutely no reason people with ALS shouldn’t be getting both SSDI and Medicare benefits immediately.
I can tell you from my own experience – we don’t have five months to wait.
My family has the genetic form of ALS (familial ALS). As of June 2018, I’ve lost 14 family members — siblings, aunts and uncles, nieces and nephews, and cousins – to this horrific disease.
My 25-year-old niece, Amanda, died four months after her ALS diagnosis – before the SSDI waiting period was met. The same situation occurred for my niece, Shannon, who died at age 34. She was diagnosed in January 2013 and died just two months later.
Continue reading Guest Post: We Don’t Have Five Months to Wait
Yesterday, ITF Pharma, Inc. announced that Tiglutik™, the first and only thickened liquid form of riluzole, was approved by the FDA for the treatment of ALS. This formulation contrasts with the oral pill form of riluzole that has been on the market for ALS for more than 20 years.
This thickened liquid form of riluzole should help individuals with swallowing difficulties. Approximately, 80 percent of people with ALS develop difficulty swallowing because of gradual weakness and paralysis in the muscles of the face and throat, called bulbar muscles.
In ALS, muscle weakness in the face and throat can lead to swallowing issues called dysphagia, along with problems with chewing, salivation, talking, and drinking. This often results in unwanted weight loss, issues taking oral medications, and in some cases, choking and aspiration, which is when food or liquid go down the wrong tube and into the lungs.
To counteract difficulties swallowing, many people with ALS end up crushing their pills. When medication is not taken as prescribed, its effect may decrease. Tiglutik provides an alternative as a thickened liquid, which can be administered orally twice-daily via a syringe. Its most common side effects are in line with established side effects observed in oral riluzole.
Continue reading Tiglutik – Thickened Liquid Riluzole – Receives FDA Approval to Treat ALS