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!”

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

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

Remembering Stephen Hawking, Who Inspired the ALS Community and the World

The ALS Association mourns the loss of Dr. Stephen Hawking and commemorates his life and legacy.

Hawking, a renowned physicist and best-selling author who famously studied black holes and whose life was depicted in the 2014 film The Theory of Everything, was diagnosed with ALS, also known as Lou Gehrig’s Disease, in 1963. The average life expectancy of person living with ALS is approximately two to five years after diagnosis and only 10 percent of people survive for more than 10 years.

He served as an inspiration for many in all walks of life, including people living with ALS around the world.

“Be curious, and however difficult life may seem, there is always something you can do, and succeed at. It matters that you don’t just give up,” Hawking once said.

ALS Association Board of Trustees Chair Stephen Winthrop spoke of Hawking’s impact.

“Most people describe ALS as a disease that, bit by bit, robs a patient’s ability to move and function. But Stephen Hawking has inspired me to focus on what I can do, rather than what my body can no longer do,” Winthrop said.

Guest Post: Patient Voices Matter

This guest post is from Shelly Hoover, EdD, a member of the Patient & Caregiver Advisory Committee, and does not necessarily reflect the opinions of the Association staff, its Board of Trustees, or its chapters.

By guest blogger Shelly Hoover

Patient voices matter, yet the collective ALS patient community has been too patient and too quiet for too long.

The ALS Association led an unprecedented effort to develop an FDA Guidance Document for Industry. Here’s a quote from the introduction:

“In February of 2015, The ALS Association launched this effort to develop the first-ever community-driven drug development guidance for ALS with funding from the ALS Ice Bucket Challenge, bringing together over 100 participants including people living with ALS, caregivers, researchers, clinicians, and industry experts from across the world to contribute their expertise and experience. In parallel with this effort, a committee was formed to update the ALS clinical trial guidelines that were developed more than 15 years ago. The guidance and guidelines have different audiences and different goals, yet are meant to be consistent. This guidance is intended to represent the Agency’s interpretation of, or policy on, evaluating new ALS therapies for approval in order to assist industry in navigating the regulatory process. The clinical trial guidelines incorporate stakeholder views across all phases of drug development, from preclinical to market approval, and serve as “best practices” for clinical trial design and are used by researchers and industry to provide structure and direction for the design and conduct of clinical trials in ALS. The goal of clinical trial guidelines is to lead to more effective and efficient trials, but they do not directly impact the FDA regulatory process.”

This guidance and the subsequent FDA document ALS: Developing Drugs for Treatment; Draft Guidance for Industry, are conservative and uphold the FDA’s gold standards for drug trial design and approval. That is great news for patients who will be diagnosed decades from now. Patients who are living with ALS today will not benefit in any significant way. As a person living with ALS, I find this conservative approach unacceptable.

I applaud The ALS Association ’s efforts. However, as a member of the patient advisory committee, I feel the patient voice was drowned out by louder voices in the development of the FDA Guidance. Read the last sentence in the introductory paragraph (bold emphasis mine). Are minor tweaks in trial design going to get potential treatments to patients now? And, why aren’t we pushing for changes to the archaic FDA regulatory process?

Here are two changes that the patient community can demand today:

  1.  Use historical controls instead of placebos until we have a widely effective treatment.
  2. Treatments in Phase 2 must be made available all patients. This will require changes to the FDA Expanded Access Program that currently does not incentivise nor protect drug manufacturers

You can make a difference by forming your own opinion and leaving a public comment here. Please do it today.

Let me be perfectly clear — this is not an ALS Association problem. The Association has multiple stakeholders, and the patient community played nice and our quiet voice got buried in the last pages of the The ALS Association Guidance Document (see Appendix C, of ALSA’s document). This is not an FDA problem. The FDA is upholding their gold standards of drug safety and approval. Why would they change?

This problem lies squarely on the shoulders of the patient community. We have the responsibility to speak out, speak loudly, and speak now. We need 1,000 public comments to get the FDA’s attention (101 comments have been submitted at the time of this writing). Then,  we must demand changes and do whatever it takes to save our own lives. If we don’t act now, we will die waiting for someone else to act for us.

About the Author

Shelly Hoover, EdD was diagnosed with ALS five years ago at age 47. She is an ALS Advocate, retired public school administrator, and US Navy Veteran. Shelly lives with her husband, Steve, in Northern California.