The ALS Association Fights To Protect Health Care Access After Texas Ruling

The ALS Association joined 37 patient groups opposing a decision from a federal court in Texas that declared the Affordable Care Act (ACA) unconstitutional. The Texas v. United States decision is expected to be appealed to the U.S. Court of Appeals for the Fifth Circuit.

The ACA will remain in place during the appeals process, including:

• Coverage protections for pre-existing conditions like ALS.
• Essential health benefits, including coverage of prescription drugs, chronic disease. management, rehab and habilitative services, and mental health.
• Coverage through Medicaid in states where the program was expanded.
• 2019 health insurance plans purchased through the marketplace (

The ALS Association unequivocally opposes any effort to eliminate protections for people with ALS and urges Congress and the U.S. Court of Appeals for the Fifth Circuit to safeguard protections for people with chronic diseases like ALS.

SOD1 Phase I Antisense Trial Shows Promise and C9orf72 Phase I Antisense Trial Begins

The ALS Association is proud to be the first investor in antisense technology, dating back to 2004 when antisense was just an idea in Dr. Don Cleveland’s lab at University of California San Diego (UCSD). Fast forward to 2018 and we are seeing promising results in antisense drugs targeting the two most common causes of inherited ALS, mutations in the SOD1 and C9orf72 genes.

After years of hard work by The ALS Association-funded investigators, Biogen recently announced promising results of the phase 1 SOD1 antisense trial (BIIB067) that is now moving into the next clinical phase and announced the initiation of a phase 1 antisense trial (BIIB078) targeting C9orf72.

The phase 1 SOD1 antisense trial enrolled 70 people with ALS that demonstrated proof-of-biology and proof-of-concept for the BIIB067. At the highest dose tested in 10 people with ALS over a three-month period, compared to placebo, BIIB067 showed a statistically significant lowering of SOD1 protein in the cerebral spinal fluid and a numerical trend towards the slowing of clinical decline as measured by the ALS Functional Rating Scale-Revised.

With these encouraging interim results, Biogen is urgently moving BIIB067 forward into a pivotal study. This means that Biogen could use the results of the pivotal study, if they are positive, as a basis to file a new drug application to the FDA.

Read more for a primer about antisense technology and how The ALS Association has played an integral part in moving this promising technology from the laboratory into clinical trials.

Below is a statement we would like to share from Biogen that explains the company’s exciting progress in antisense technology.

Dear members of the ALS community,

As we look towards 2019, we wanted to provide an update on our investigational therapies for ALS to those of you around the world who are impacted by this disease.

We have made important progress along with our partner Ionis Pharmaceuticals in moving forward the development of our investigational therapies for familial ALS, BIIB067 and BIIB078. BIIB067 is designed to treat ALS in adults with confirmed mutations in the superoxide dismutase 1 (SOD1) gene. BIIB078 targets the chromosome 9 open reading frame 72 (C9ORF72) mutation (often called a “repeat expansion”), which is the leading familial cause of ALS.

Both of our ongoing studies in ALS are progressing and we wanted to share some important updates with you:

BIIB067 for SOD-1 Related ALS

  • Our Phase 1 study of BIIB067 recently completed enrollment. A total of 70 people enrolled in this effort.
  • An additional study of BIIB067 remains underway, which evaluates outcomes over a longer period of time.
    • On December 6, we announced positive results from an interim analysis of this Phase 1 study, as well as Biogen’s decision to obtain a license to develop and commercialize BIIB067 for SOD1 ALS from Ionis Pharmaceuticals.
    • Results of the interim analysis were encouraging, and Biogen is urgently advancing BIIB067 to a pivotal clinical study, meaning it could potentially serve as a basis for regulatory filing. Study timing and enrollment plans are not yet confirmed but we will communicate these as soon as we are able to.
    • You can learn more about the status of the BIIB067 program in our news release, here.

BIIB078 for C9ORF27 Related ALS

  • Our Phase 1 clinical trial for BIIB078 was initiated this fall, and the first patients have now been dosed. We hope to have 12 U.S. as well as several Canadian and European sites open and enrolling within the next several months to complete enrollment as urgently as possible.

These studies will help us better understand the safety, tolerability and pharmacokinetic profile of the investigational treatments and appropriately plan for future studies.

We’re grateful to those in the ALS community who have chosen or sought to participate in one of our clinical trials and for their belief in our science. Groundbreaking clinical research cannot be accomplished without a commitment from people living with ALS who are willing to join a clinical study.

While we wish we could accommodate every person who wants to join a study, clinical studies at the early stages typically enroll a small number of participants and have a limited number of sites available. We recognize that this can be very frustrating, especially for people who are eager to access investigational compounds when there are few treatment options available.

We are humbled by the overwhelming response for participation in our studies. Please know we are working tirelessly with study sites and investigators to optimize the study participant experience while ensuring that investigators have the resources they need to properly conduct a study and evaluate study data on our behalf.

Our goal is to evaluate the data for both studies as quickly as we can, looking for suitable signals of safety and efficacy that will help move our development programs forward and advance future studies.

In 2019, we will continue to provide quarterly updates on progress in our development plans, including opportunities for participation in Biogen clinical trials. Biogen is committed to finding a treatment for ALS. This commitment extends to individuals participating in our studies, their families, caregivers and the greater ALS community.

For more information or resources on clinical trial enrollment, please reach out to


Motor Neuron Excitability is Reduced in People with ALS Following Ezogabine Treatment

Dr. Brian Wainger from the Massachusetts General Hospital (MGH) presented initial top-level results from a recently completed phase II clinical trial of ezogabine (retigabine) on motor neuron excitability (NCT02450552). The study, supported by The ALS Association, met its main goal of quantifying a reduction in motor neuron excitability in people with ALS following treatment. Results were presented during the 29th International Symposium on ALS/MND in Glasgow, Scotland, last week.

“This novel study provides us with a better understanding of neuron hyperexcitability, an important ALS disease pathway, and we are thrilled to be part of such a powerful, collaborative team,” stated Calaneet Balas, president and CEO of The ALS Association.

This trial, conducted by investigators from the MGH Neurological Clinical Research Institute (NCRI) at Northeast ALS Consortium (NEALS) trial sites, focuses on hyperexcitability of motor neurons. It was previously shown that people living with ALS have motor neurons (both upper and lower) that fire too many signals, meaning they are hyperexcitable. Too much firing leads to motor neuron damage. These “hyperexcitable neurons” are thought to play a role in ALS.

Continue reading Motor Neuron Excitability is Reduced in People with ALS Following Ezogabine Treatment

Guest Post: ‘It Takes A Village’

By Patti O’Reilly

I was diagnosed with ALS in January 2014, at the age of 53.

I was an oncology nurse for 32 years and had just completed my master’s degree in nursing and passed the boards to become a nurse practitioner when I was diagnosed. Being a nurse practitioner had always been a dream of mine, but I was unable to use my master’s degree at all because of ALS.

Continue reading Guest Post: ‘It Takes A Village’

We Helped Support Over 20,000 People with ALS This Year

People living with ALS come first in everything we do. We’re dedicated to providing people fighting ALS and their families and friends with the critical information, support, and resources they need to live full lives and better meet daily challenges.

Our chapters help people living with ALS and their families across the country through our equipment loan closets, support groups, augmentative communication and assistive technology program, caregiver support, and education and outreach.

We also collaborate with the best ALS physicians and clinics in our service areas to help ensure that people living with ALS have access to specialized multidisciplinary care.

Here are just a few ways we provided critical assistance to those living with ALS and their families in 2018:

  • More than $4 million in grants were provided through our Certified Center Program
  • Nearly 9,000 people were served specifically through our Certified Treatment Centers of Excellence and Recognized Treatment Centers
  • More than 2,400 veterans received assistance through our nationwide chapter network

Check out our latest infographic to discover all the ways we made a difference to the more than 20,000 people with ALS we served this year. (You can also view the infographic on our website here.) These efforts are essential in improving the quality of life for people fighting ALS.


We need your help TODAY to continue to provide the best care across the country. Your tax-deductible gift will allow us to continue these crucial services into 2019. We can’t do it without you!

Thank you for your support – for giving hope and making an impact on the lives of people affected by ALS.

People with ALS Should Have Access to the Medicare Home Health Benefits They Deserve

The ALS Association is ramping up its efforts to make sure people with ALS who rely on Medicare have access to home health care benefits.

“Many people in the community, including people with ALS, people within our chapters, and our clinical partners, have told us about the serious and persistent challenges faced by Medicare beneficiaries with ALS,” said Dr. Neil Thakur, executive vice president for mission strategy at The ALS Association. “Many such beneficiaries, despite being fully qualified, are turned down for the home health benefits they deserve. Other beneficiaries are able to receive Medicare home health but deal with inadequate hours of service and inappropriate termination from care.”

Continue reading People with ALS Should Have Access to the Medicare Home Health Benefits They Deserve

A Husband and Caregiver’s ‘ALS Experience’

Ronnie selflessly cared for Linda, his wife of 20 years, throughout her fight with ALS.

“The hardest thing anyone can experience is watching a loved one slowly die [when you] can’t do anything about it,” he said. “It doesn’t matter what disease they have.”

He added, “ALS is [an] ugly disease. It slowly takes away the person we saw being able to take care of themselves and enjoy life. The sad thing is…there is no cure. The person who has ALS just slowly wastes away.”

Linda was diagnosed with ALS on April 16, 2012. She passed away less than 14 months later – on June 8, 2013.

“If it wasn’t for God, I couldn’t have made it through,” Ronnie said. “I pray every day for all the caregivers and have the utmost respect for them.”

He wrote “ALS Experience” after Linda’s death.

Continue reading A Husband and Caregiver’s ‘ALS Experience’