In every journey of discovery, certain days distinguish themselves as fundamental to achieving the ultimate objective.

Yesterday was one of those days in the fight against ALS, Lou Gehrig’s Disease, with the FDA’s approval of Amylyx’s new drug, Relyvrio, the first new ALS drug in over five years.

ALS (amyotrophic lateral sclerosis) is a devastating disease of the nervous system which robs patients of their muscle control and eventually their ability to breathe on their own.  While rare, there is no known cure, and it famously struck down Lou Gehrig, the legendary Yankee, in the prime of his career.

Founded by two Brown University graduates, Josh Cohen and Justin Klee, Amylyx is a Boston area company that received its funding for the critical initial studies that led to yesterday’s breakthrough announcement from a unique organization called ALS Finding a Cure.

ALS Finding a Cure was the brainchild of the long time CEO of the global beauty company Conair, Leandro (Lee) Rizzuto.  Accustomed to solving complex problems and developing innovative new products, Lee was confounded when his daughter-in-law Christie was diagnosed with ALS.  Lee saw news reports of a new study that the NFL was undertaking in conjunction with GE to address traumatic brain injuries in 2014.  Curious, Lee contacted his old friend from GE, Peter Foss, who was working with GE Healthcare and the NFL on the project to determine if some of the same approaches to traumatic brain injury could apply to ALS.  Peter agreed to work with Lee to set up a new organization which became ALS Finding a Cure, which had a sole mission: find a cure for ALS.

Peter and Lee turned first to Dr. Merit Cudkowicz, the Chair of the Department of Neurology at Massachusetts General Hospital and the Julieanne Dorn Professor of Neurology at the Harvard Medical School.  Dr. Cudkowicz was Christie’s physician and a specialist in the clinical treatment and research of ALS.  They then joined forces with Dr. Stanley H. Appel, the  Chair of the Stanley H. Appel Department of Neurology and Co-Director of the Houston Methodist Neurological Institute, who was a specialist in immunology and the study of inflammation in the brain.  The team also added Dr. Robert H. Brown Jr., the director of the Program in Neurotherapeutics at the University of Massachusetts Medical School, a specialist in genetics, and Dr. Clive Svendsen, the founder and director of the direct the Cedars-Sinai Regenerative Medicine in Los Angeles and a specialist in stem cell research, as well as other leading specialists in the fight against ALS.

Over the next several years, Lee provided more than $40 million of his own money to fund cutting edge research into the fight against ALS, while funding projects that also attracted tens of millions of additional funding from public and private foundations.

One of the early projects which received funding from ALS Finding a Cure was Amylyx.

Josh Cohen and Justin Klee were students at Brown University at the time.  They were in Charleston at the Massachusetts General Hospital Lab facility when they met with Peter Foss and Dr. Cudkowicz.  Their initial idea for the new drug they were developing was to target Alzheimer’s Disease.  Dr. Cudkowicz suggested that ALS might also be a good target for their disease and introduced them to the team at ALS Finding a Cure, which along with Cure AD, provided them with the grant to fund the toxicology studies that would pave the way for subsequent clinical trials.

ALS Finding a Cure then collaborated with the ALS Association (ALSA) to provide the second grant to support the Phase 2 Clinical trial for Amylyx’s Relyvrio ALS drug.  ALSA’s funding came from the famous ALS Bucket Challenge, a viral sensation in 2014 co-founded by Patrick Quinn and Peter Frates (a patient of Dr. Cudkowicz) that raised over $200 million.  Dr. Sabrina Paganoni, Co-Director of the Massachusetts General Hospital Neurological Clinical Research Institute and a member of the ALS Finding a Cure team was the leading research advisor to Amylyx for their studies to develop Relyvrio.

The decision by the FDA on September 29, 2022 approves a drug that demonstrated slowing of the loss of function from ALS and a prolongation of life. It is truly an extraordinary day for people living with ALS.

ALS Finding a Cure, Amylyx, and the entire ALS global community came together to advocate successfully for the FDA approval that will have a profound impact on people suffering from ALS.

The approval of Relyvrio was a milestone event in the long fight against ALS, and one that gets us ever closer to Lee Rizzuto’s bold vision of finding a cure for this dreaded disease.

Links to Articles on Amylyx

Amylyx’s ALS Drug Is Approved by the FDA – WSJ, Wall Street Journal

Amylyx’s ALS drug finally scores FDA approval, but that’s just the tip of the iceberg for ALS research, founders say | Fierce Pharma, Fierce Pharma

FDA greenlights Amylyx’s ALS drug | Reuters, Reuters

Much-debated drug from Amylyx for Lou Gehrig’s disease wins FDA approval | Fox News, Fox News

Cambridge biotech wins approval for much-debated ALS drug – The Boston Globe, The Boston Globe

FDA approves controversial new drug designed to slow the progress of ALS (nbcnews.com), NBC News

Amylyx Pharmaceuticals’ ALS Drug Wins FDA Approval (AMLX) – Bloomberg, Bloomberg

ALS drug approved by FDA in closely watched decision, marking win for patients, developer | BioPharma Dive, BioPharma Dive

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An international team of investigators has discovered that an inorganic polyphosphate released by nerve cells known as astrocytes in people with amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) contributes to the motor neuron death that is the signature of these diseases. The research, by Brigitte van Zundert, PhD, adjunct professor of neurology at UMass Chan and professor at the Universidad Andres Bello in Chile; Robert H. Brown Jr., DPhil, MD, professor of neurology, and colleagues appears this week in Neuron.