Guest Post: Putting the Brakes on ALS – The Power of Community

By Kaisha Johnson and Cliff Whitlock, The ALS Association Golden West Chapter

More than 1,200 participants, volunteers, and members of the community came together for a family-friendly day of celebration, exercise, education, awareness, music, food, and family fun at The ALS Association Golden West Chapter’s 14th annual Napa Valley Ride to Defeat ALS and Walk on Saturday, September 22.

Together, these dedicated individuals raised more than $920,000 on event day toward a $1 million goal to fund the mission priorities of the Golden West Chapter in care services, advocacy, and research.

Held at the California Veterans Home in Yountville, the annual Fall event brings incredible energy and an outpouring of support for those affected by ALS in California and around the world. Many participants said it was the best ride they’ve attended.

There was an incredible outpouring of community support, including more than 140 volunteers who set up, checked in participants, served food and drinks, supported rest stops and the six bike routes, cheered on all of our incredible riders and walkers as they crossed the finish line, and so much more.

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Certified Treatment Center of Excellence Spotlight: ALS Treatment and Research Center at UCSF

Our national network of 63 Certified Treatment Centers of Excellence (CTCEs) provides robust, expert multidisciplinary ALS care and services in a supportive atmosphere, emphasizing hope and quality of life. Studies show attending a multidisciplinary clinic can extend survival, increase quality of life, and improve access to potential therapies.

CTCEs bring together a team of health care professionals specially trained to address the needs of people living with ALS, allowing them to receive care from each discipline during a single visit.

Teams typically include a neurologist, a physical therapist, an occupational therapist, a respiratory therapist, a nurse, a dietitian, a speech-language pathologist, a social worker, a mental health professional, and a liaison from The ALS Association.

Thanks to donations from the ALS Ice Bucket Challenge, The ALS Association has increased the number of CTCEs, as well as the size of its annual grants to each one. This is the third in an ongoing series of CTCE spotlights.

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Meet Dr. James Shorter: ALS Researcher Dedicated to Understanding What Causes ALS

Meet Dr. James Shorter, professor of Biochemistry and Biophysics at the Perelman School of Medicine at the University of Pennsylvania in Philadelphia. He and his team work tirelessly in the lab to better understand the causes of ALS, so those causes can be translated into potential therapeutic targets. The ALS Association has proudly funded him since 2014.

Dr. Shorter’s work focuses on protein homeostasis, which is how proteins in the cells of the body fold into specialized structures to perform specific functions. Sometimes protein misfolding occurs, which allows the formation of protein aggregates (clumps of protein).

When protein aggregates are in the form of fibrils and oligomers, they can be harmful to cells. Dr. Shorter and his team’s goal is to understand how protein misfolding occurs and how cells can prevent it from happening.

In ALS disease, it is known that certain RNA-binding proteins, such as TDP-43, FUS, hnRNPA1, and hnRNPA2, mis-localize from the cell’s nucleus into the cytoplasm and accumulate in cytoplasmic aggregates (protein clumps). Mutations in a specific area of these proteins, called prion-like domains, play a part in accelerating fibrillization that causes toxicity in cells.

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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.

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MediciNova Announces Positive FDA Feedback to Start Phase III Plan for MN-166 (ibudilast) to Treat ALS

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.

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Team Challenge ALS Climbs to Defeat 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.

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Members of ALS Community Gather with Representatives from FDA and Industry to Inform FDA Draft Guidance on 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.”

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