House and Senate introduce Accelerating Access to Critical Therapies for ALS Act (ACT for ALS)


For Immediate Release: Oct. 29, 2020

Media Contact: James Crotty, [email protected], (402) 570-2708   

Early Access to Promising Treatments Offers New Hope in Fight against ALS and Improved Infrastructure for Neurodegenerative Disease Drug Development

(WASHINGTON, DC)––Congressman Jeff Fortenberry (R-NE), Congressman Mike Quigley (D-IL), Senator Chris Coons (D-DE), and Senator Lisa Murkowski (R-AK) introduced an updated version of ACT for ALS, Accelerating Access to Critical Therapies for ALS Act in the United States House and Senate.

The two new pieces of legislation, H.R.8662 in the House and S.4867 in the Senate, create new pathways for faster and broader access to therapies for those most in need, including those facing a fatal disease like ALS.

“This new legislation represents a monumental shift for those suffering from ALS and other neurodegenerative diseases.  It breaks the paradigm of disease research and creates a new pathway to deliver promising treatments.  With this new law, we can break through faster for those who have suffered so much,” said Congressman Jeff Fortenberry.

“We must do all we can to support the tens of thousands of Americans impacted by ALS, a terrible disease without a cure.  The ACT for ALS will expand early treatment options and support the cutting-edge research needed to improve quality of life and find a cure.  We should take steps to facilitate access to new therapies, drug treatments, and medical breakthroughs to aid Americans who suffer from ALS or other forms of neurodegenerative disease,” said Senator Chris Coons, co-chair of the Senate ALS Caucus. 

“For too long, ALS and diseases like it have gone under researched and under resourced at the federal level.  That changes today.  The new ACT for ALS bill is the product of intense engagement over many months and is our best opportunity in decades to disrupt the status quo, move the ball forward, and expedite treatment options for those living with terminal illnesses like ALS.  I’ve been so proud to work alongside a diverse group of stakeholders—patients, advocates, scientists, researchers and doctors, both in and outside of government— to produce a bold, bipartisan piece of legislation that offers new hope to those suffering from ALS and other neurodegenerative diseases with few treatments and no cures.  I want to thank my partner in this effort, Congressman Jeff Fortenberry, and all those who worked with us to improve this legislation, and I am honored to give voice to the ALS patient community, without whom this effort would simply not be possible,” said Congressman Mike Quigley.

“For years I’ve been working with the ALS community to help increase research opportunities and ensure those living with this deadly disease have the support they need.   We’ve made progress, but even with promising treatments in clinical trials, patients unable to enroll in those research studies unfortunately have very few options for treatment.   Through a new pilot grant program, as well as other additional funding, this bipartisan legislation will help address barriers to utilizing the current expanded access pathway, while also broadly supporting the development of ALS treatments.  As someone whose family has been personally impacted by ALS, I’m proud to help Senator Coons lead this effort,” said Senator Lisa Murkowski.

The revised ACT for ALS addresses several challenges associated with ALS therapeutic development.  First, ALS clinical trials do not always employ expanded access programs that facilitate access to experimental treatments outside of the trial.  This legislation tackles the financial barrier for therapies being tested from small biopharmaceutical companies by creating a grant program that funds access to investigational ALS treatments to patients not participating in the clinical trial. Additionally, the grant will fund a research project as part of providing expanded access to people living with ALS, adding to the understanding of how treatments impact the disease.

The revised legislation also invests in neurodegenerative disease research through a new Food and Drug Administration (FDA) Rare Neurodegenerative Disease Grant Program.  With this investment, Congress prioritizes making progress in the development of treatments and cures for ALS and other rare neurodegenerative diseases.

Finally, ACT for ALS would establish an HHS Collaborative for Neurodegenerative Diseases jointly led by the FDA and the National Institutes of Health (NIH), the first federal effort explicitly charged with the responsibility to speed the development and approval of therapies for neurodegenerative diseases.  This collaborative is charged with coordinating efforts across the public and private sector to accelerate therapeutic development and regulatory review of treatments and cures for ALS and neurodegenerative diseases, including:

  • Coordination among the centers of the Food and Drug Administration to achieve the goals specified in the draft guidance entitled “Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment Guidance for Industry,’’ published in September 2019;
  • Facilitation of access to investigational drugs for ALS;
  • Definition or development of the regulatory and translational pathway for emerging therapeutic categories; and
  • Development and implementation of an ongoing mechanism to share feedback and information and develop strategies with the neurodegenerative disease community, including patients, treating physicians, national organizations that facilitate provision of care services, access and research, researchers, drug sponsors, drug manufacturers and Federal agencies.

The bills have the full support and coordination of three leading ALS organizations in I AM ALS, The ALS Association and the Muscular Dystrophy Association.  The revised ACT for ALS addresses several challenges associated with ALS therapeutic development.

“We made a commitment at the start of 2020 to firmly stand behind this legislation and the drive to make it a reality for ALS patients waiting for critical, needed treatments.  This bill will provide additional opportunities to access promising ALS therapies and create a collaborative focused squarely on expediting development and approval of new ALS treatments and cures.  We now look forward to working with the community and Congressional leaders to take one more step toward changing the course of history as we end ALS and other neurodegenerative diseases,” I AM ALS CEO Danielle Carnival, PhD., said.

“The strengthened Accelerating Access to Critical Therapies (ACT) for ALS Act could be transformative for the ALS community.  It will help provide effective treatments for everyone living with ALS today, and expanded access to experimental ones.  It creates the first federal entity explicitly charged with finding effective treatments for ALS as quickly as possible.  We thank Reps. Jeff Fortenberry (R-NE), Mike Quigley (D-IL) and Sens. Chris Coons (D-DE) and Lisa Murkowski (R-AK) for introducing this bill to meet these goals,” stated Neil Thakur, Ph.D., The ALS Association Chief Mission Officer.

“The introduction of a strengthened ACT for ALS represents a substantial step forward in the effort to bring safe, effective, and innovative new treatments to the ALS community.  We are proud to endorse this bill, and in coordination with the ALS patient community, we call on Congress to pass this legislation as soon as possible,” said Kristin Stephenson, EVP, Chief Advocacy and Care Services Officer, Muscular Dystrophy Association.

Link to House Bill: https://bit.ly/37POqlG

Link to Senate Bill: https://bit.ly/34GW6ou

Congressman Jeff Fortenberry is the Ranking Member of the House Appropriations Subcommittee on Agriculture, Rural Development, and Food and Drug Administration.

Senator Chris Coons is the founder and co-chair of the Senate ALS Caucus and a member of the Senate Committee on Appropriations.

Congressman Mike Quigley is Chairman of the House Appropriations Subcommittee on Financial Services and General Government.

Senator Lisa Murkowski is one of the original members of the Senate ALS Caucus, a member of the Senate Appropriations Committee, and a member of the Senate Health, Education, Labor and Pensions Committee.

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