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Early Access to Promising Treatments Provides New Hope in Fight

May 28, 2020  -  I AM ALS

FOR IMMEDIATE RELEASE: May 28th, 2020

CONTACT: Kathleen Rooney, [email protected]

House and Senate Bills Create New Pathways for Treatment Access and Improved Infrastructure for Neurodegenerative Drug Development

WASHINGTON - Today, two new pieces of legislation were announced in the House and the Senate to create real pathways to provide faster and broader access to therapies for those most in need, including those facing a terminal disease like ALS.  

Congressman Jeff Fortenberry (R-NE) and Congressman Mike Quigley (D-IL) announced an intent to introduce a bipartisan bill in the House of Representatives that will create an infrastructure to fund early access to promising therapies discovered through clinical trials for patients suffering from fast-progressing neurodegenerative and terminal diseases like ALS.

Senator Mike Braun (R-IN) announced an intent to introduce a bill that will speed up the regulatory processes for getting drugs showing benefits to the patients who need them. Braun’s bill provides an FDA provisional approval pathway for therapies that treat conditions where the disease progresses rapidly and few to no treatment options exist.

A disease like ALS, which will affect 1 in 300 people in their lifetimes, typically gives patients no more than 3 years to live following diagnosis. Patients don’t have years to wait for new treatment options. Access to promising therapies in a matter of months is a game changer and potentially a lifesaver.

“One month ago, my wife’s little brother died from ALS. He was 37, with a wife and four small children. His suffering, the heartache, the toll on his family is repeated over and over in families throughout America. Let’s do something about it,” says Congressman Fortenberry.

“What many don’t know is that when we find a cure for one of the fastest progressing neurodegenerative diseases, like ALS, we will unlock critical breakthroughs for other diseases like Parkinson’s, Alzheimer’s, Frontotemporal Dementia and beyond, which affect 50 million Americans each year,” says Congressman Quigley. “This legislation not only paves the way to curing a litany of diseases but provides a model for how we speed up our drug treatment pipeline and bring it into the 21st century.”

In the House, the Accelerating Access to Critical Therapies for ALS Act will make $75,000,000 available in FY 2021 and 2022 as part of a pilot to provide grants to support expanded access programs. It will bring treatments for rapidly progressing diseases for which effective therapies don’t already exist to patients beyond the ongoing clinical trial.  The legislation will also establish a Center of Excellence for Neurodegenerative Diseases at the FDA to accelerate the development and approval of therapies for the coming tide of neurodegenerative diseases that will cause a health and economic crisis in the coming decades. The Center will be modeled after FDA’s Center of Excellence for Oncology that has driven forward many monumental discoveries in cancer research. 

“It is estimated that more than 5,000 Americans each year are diagnosed with ALS and told to get their affairs in order. This legislation gets us ever closer to a place where we can say, ‘you have ALS, here is an effective therapy that will allow you to see your children and grandchildren grow up.’ We fight every day to make that day real.” says Danielle Carnival, CEO of I AM ALS.

In the Senate, the Promising Pathway Act will amend the Federal Food, Drug, and Cosmetic Act to establish a time-limited provisional approval pathway for certain drugs and regulated medical products. 

“Americans with life-threatening and terminal diseases don’t have time to waste on a bureaucratic journey through red tape and regulations, only to be denied access,” says Senator Braun.

Both bills will open up pathways to not only move the fight forward for ALS but for many terminal, fast progressing diseases with limited to no treatment options. 

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About I AM ALS

Founded in 2019 by husband-wife team Brian Wallach and Sandra Abrevaya, I AM ALS was born out of their desire to change the future for Brian and the thousands of other ALS patients in the world. I AM ALS brings together patients, advocates, organizations and scientists to deliver critical and innovative resources for patients, empower and mobilize patients and their networks to lead the fight for a cure, and transform the public understanding of ALS with a goal of flooding the research pipeline with new, lifesaving drugs. A cure is possible ... if we work together to re-imagine the fight against ALS. Learn more here: https://iamals.org

About ALS

ALS is a disease that attacks cells in the body that control movement. It makes the brain stop talking to the muscles, causing increased paralysis over time. Ultimately, ALS patients become prisoners within their own bodies, unable to eat, breathe, or move on their own. Their mind, however, often remains sharp so they are aware of what’s happening to them.

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