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New Research Update for Proposed ALS Drug Treatment—AMX0035

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We’re excited to share another milestone in the search for better drug treatments for ALS. Amylyx announced today that the company will soon submit a New Drug Application (NDA) for AMX0035 to the FDA. The ALS Association has been closely following the development of this treatment, even providing serious funding to propel its development.

Supporting the Development of a New Treatment

Many of these donations were received during the record-breaking ALS Ice Bucket Challenge in 2014. The ALS Association provided a grant of $750,000 for a clinical trial pilot in June 2016. The next month, a $1.46 million grant was provided to the Northeast ALS Consortium (NEALS) to fund the phase 2 CENTAUR clinical trial of AMX0035.

In September 2020, the concluded trial reports were released. They showed promise for this new drug. Those who received this AMX0035 experienced a significant slower progression of ALS than those who received the placebo. In terms of side effects, the drug presented itself as well-tolerated with a better benefit to risk balance. Read more about the CENTAUR phase 2 results here.

The Crucial Role of ALS Advocates

As soon as the results of phase 2 were released for AMX0035, ALS advocates jumped into action. In a team effort with I AM ALS, the ALS Association issued a petition. It urged the FDA and the drug company to make the treatment widely available as soon as possible. When the average lifespan is just 2 – 5 years after an ALS diagnosis, we can’t wait!

That’s just what ALS advocates communicated to the FDA just earlier this year in May. Eight ALS representatives from across the United States hosted a We Can’t Wait Action Meeting with the FDA. The meeting urged the FDA to act swiftly when it comes to approving new drug therapies for those living with ALS.

Our advocates play just as much as a role in the development and approval of new drugs as does the funding provided by generous supporters. With both these functions working in tandem, we can only hope for speedy approvals of future drug treatments.

What Comes Next?

Amylyx is currently conducting a third clinical trial (PHOENIX) of AMX0035; however, they can concurrently submit the NDA. It’s estimated that the drug company will submit this application sometime in the Fall 2021.

Following the application submission, the FDA will determine if they want to grant the new treatment approval. Or, contrarily, the FDA could require an additional study. This review process will take several months from the time of submission. In addition to the United States, Amylyx has also filed for approval of AMX0035 in Canada and the European Union.

In the meantime, the ALS Association plans to put pressure on the FDA to approve this treatment as soon as possible.

Read the full press release

View the we can't wait action meeting

Read the FDA's response to the we can't wait action meeting

Advancing Research: The HEALEY ALS Platform Trial

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In 2020, the ALS Association, in partnership with Massachusetts General Hospital, launched an innovative approach to research, the HEALEY ALS Platform Trial. In most clinical trials, a single drug is tested, and participants cannot be enrolled in more than one trial at a time. The HEALEY ALS Platform Trial enables us to test multiple proposed drug treatments at once. This is a model that has been successful in cancer research. It accelerates our search for effective treatments for people living with ALS by allowing investigators to test more drugs, increase patient access to trials, and reduce costs by quickly and efficiently evaluating the effectiveness of multiple therapies. There are now 54 test sites nationwide, three of which are in Texas.

Texas Neurology was one of the first five initial sites for the new model. “This is big. I’ve been doing trials since the early nineties. The average trial takes 8 years. Going through platform methodology shortens it to 2-3 years,” states Dr. Daragh Heitzman, clinical director at Texas Neurology. Dr. Heitzman was recently appointed to the HEALEY ALS Platform Trial Executive Committee, as the first physician investigator to join an elite group of scientists.

“What’s different about this trial is that the placebo group is being shared for all five drugs, such that the number of participants who actually get a drug is dramatically increased. So, it’s a lot quicker to get to an answer, more patients are getting drugs, and less getting a placebo.”

Trial participants will have the option to continue treatment for at least a year, and if the drug shows promise during the six-month trial, the FDA could potentially fast-track its approval, getting a crucial treatment to people living with ALS.

Texas HEALEY ALS Platform Trial Sites

  • The ALS Clinic at Texas Neurology in Dallas
  • Houston Methodist ALS Clinic
  • The ALS Clinic at UT Health San Antonio

Read more about Dr. Heitzman and the clinic at Texas Neurology

How ALS Patients Can Help with Research

ALS Drug Shows Further Promise, Slowing Progression and Increasing Survivability

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Muscle and Nerve has published the results of the open label extension study of AMX0035, a promising new drug therapy for people with ALS developed by Amylyx. The study showed that people who received the drug lived about 6.5 months longer than people who received the placebo during the trial. This finding is in addition to the findings from the phase 2 trial, showing that people with ALS receiving this drug experienced a significantly slower decline in disease progression, compared to those on a placebo.

As quoted in the New York Times article, Neil Thakur, chief mission officer of the ALS Association, states, “That is a very meaningful benefit for people affected by this devastating, fatal disease.”

These findings further show that the treatment should be made available to people with ALS as soon as possible. We urge the FDA and Amylyx to work together to bring this treatment to market as soon as possible and we stand ready to help ensure that happens. You can help by signing our petition to the FDA and Amylyx at als.org/petition.

ALS Drug Slows Progression, Petition for FDA Approval

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The New England Journal of Medicine (NEJM) has published the results of a phase 2 trial (known as CENTAUR) of the compound AMX0035, produced by Amylyx, a Massachusetts-based pharmaceutical company. The results of the trial were very promising – people with ALS receiving this drug experienced a significantly slower decline in disease progression, compared to those on a placebo. Additionally, this drug shows to be safe and well-tolerated indicating a positive benefit and risk consideration for people with ALS.

The ALS Association and I AM ALS have now issued a petition calling on the drug company and the FDA to make the treatment widely available as soon as possible. The ALS Association and I AM ALS are asking that Amylyx and the FDA move with urgency to ensure this drug is a treatment option for all people living with ALS.

Click to expand

The ALS Association committed $750,000 to Amylyx in funding for this research, and $1.4M to the NEALS clinical trial consortium to support the trial. These grants were made possible through the ALS Ice Bucket Challenge. The trial included 137 people with ALS and was conducted across 25 top medical centers through the Northeast ALS (NEALS) consortium. All of the participants enrolled in the trial were diagnosed with sporadic or familial ALS within the previous eight months and had a rapid progression of the disease.

With a typical survival time of between 2 to 5 years, people with ALS cannot wait for the full experimental process of this drug to continue. In light of the substantive data of AMX0035’s effectiveness and safety, it has been requested that the FDA and Amylyx to work together to bring this to people with ALS as soon as possible by approving the drug prior to a Phase 3 trial. To access and sign the petition, visit www.als.org/petition.

Sign the Petition Today!

The ALS Association, I AM ALS Award BrainStorm Cell Therapeutics $500,000 for ALS Biomarker Study

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The ALS Association and I AM ALS announced today that the organizations have awarded a combined grant of $500,000 to BrainStorm Cell Therapeutics (NASDAQ: BCLI), a biotechnology company, to support this amyotrophic lateral sclerosis (ALS) biomarker research study. The grant will be used to draw insights from data and samples collected from patients enrolled in BrainStorm’s ongoing phase 3 clinical trial of its NurOwn® treatment, to further understanding of critical biomarkers associated with treatment response for people with ALS.

The study is designed to evaluate how NurOwn interacts with its targets in the brain and spinal cord and to explore the changes in the biomarkers that may correlate with response to the drug treatment. Biomarkers are any measurable substance in the body that change over time (such as cholesterol or blood pressure) and that correlate with disease processes or treatment response. If successful, this study will help confirm that the ALS treatment NurOwn works in the way it is intended and will help inform our larger understanding of ALS biomarkers.

“This grant to BrainStorm marks an important step forward in establishing how exactly NurOwn works in the body,” said Calaneet Balas, President and CEO of The ALS Association. “This research is also important to our overall pursuit of identification and validation of ALS biomarkers. We hope NurOwn is ultimately proven effective in treating ALS and we stand ready to support BrainStorm in its plan to apply for a biologics license for NurOwn.”

Danielle Carnival, CEO of I AM ALS commented, “We need to move with urgency in all of our efforts to deliver treatments and cures for ALS. This biomarker research will help us more expeditiously understand the effectiveness of NurOwn, while possibly unlocking discoveries that provide clues for other promising treatments. We are at a pivotal time for ALS research in pursuit of treatment solutions and will do whatever we can, together, to drive new answers and new hope for patients.”

The joint award to BrainStorm consists of a $400,000 grant from The ALS Association and a $100,000 grant from I AM ALS. As part of this agreement, BrainStorm has agreed to share data and samples with the ALS community so that the results can be independently validated and to advance other ALS research.

Chaim Lebovits, CEO of BrainStorm stated, “We sincerely appreciate the scientific recognition and generous support from The ALS Association and I AM ALS and we are excited through this study to further an already strong partnership between The ALS Association, I AM ALS and Brainstorm. This critical research study involves one of the largest and most robust clinical trial collections of CSF biomarkers. Data generated from this study will increase our understanding of how NurOwn® therapy impacts ALS disease progression and may identify patients who benefit the most from this form of therapy. We also hope that this research study will benefit the broader ALS scientific community as we collectively advance towards our shared goal of delivering much needed treatments.”

About NurOwn®
NurOwn® (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently received U.S. FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began in March 2019.

About The ALS Association
The ALS Association is the largest private funder of ALS research in the world. The Association funds global research collaborations, provides assistance for people with ALS and their families through our nationwide network of chapters and certified clinical care centers, and advocates for better public policies for people with ALS. The ALS Association builds hope and enhances quality of life while urgently searching for new treatments and a cure. For more information about The ALS Association, visit our website atals.org.

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 cures, 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: iamals.org.

About BrainStorm Cell Therapeutics Inc.
BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn® technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six U.S. sites supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently received U.S. FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive Multiple Sclerosis. The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment in March 2019. For more information, visit the company’s website at brainstorm-cell.com.

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.

Original Press Release was posted here.

The ALS Association Continues to Push Development of Novel Treatments

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The ALS Association is excited to announce $2.5 million in grants to help develop promising new treatments for people living with ALS.

The grants are supported by the Association’s Lawrence and Isabel Barnett Drug Development Program, which supports drug discovery research in both academia and industry to develop new drug therapies and test them in a preclinical setting while moving those therapies closer to clinical use.

“This program funds preclinical projects aimed at developing treatments for ALS,” said Kuldip Dave, Ph.D., vice president of research at The ALS Association. “We anticipate that the agents and data generated from these projects will lead to the advancement of new promising therapies for ALS.”

While we continue to add new projects to this portfolio, these latest grant awards are showing a great deal of promise and hope for people living with ALS.

INmuneBio was awarded $500,000 allocated over two years to support proof of concept testing of XPro1595 for use in ALS therapy. XPro1595 has advanced to clinical testing for Alzheimer’s disease, but its use in ALS is novel. It targets immune dysfunction and inflammation by blocking the development of bad soluble TNF, a cell-signaling protein. The principal investigator for the study is CJ Barnum, Ph.D., director of neuroscience at INmune Bio. For more information, click here.

Peter Crouch of the University of Melbourne was awarded $495,176 to support animal testing of the novel use of copper-ATSM combined with follistatin. The proof of concept testing builds on the theory that effective ALS treatment could require drug cocktails, or the combination of multiple drugs and will occur concurrently to clinical testing already underway to test the efficacy of copper ATSM more generally. The ALS Association Oregon and SW Washington Chapter contributed $300,000 to the project.

Neuropore Therapies, Inc. was awarded $500,000 in support of their preclinical evaluations of NPT1220-312, a small molecule Toll-Like 2 receptor (TLR2) antagonist, as a potential ALS treatment targeting inflammation and the immune system. Early work in the area of TLR2 antagonists has been promising. This research will provide key efficacy and translational biomarker data, which will be used to drive a go/no go decision to transition NPT1220-312 to clinical development as a disease-modifying ALS therapeutic. The principal investigator for the study is Diane Price. For more information, click here.

Bloom Science Inc. was awarded a $500,000 grant to explore preclinical development of a Live Biotherapeutic Product used in the treatment of ALS. Bloom is leveraging its GOLD platform (Genetically Optimized Living Drugs) in combination with strong preclinical data to advance a new generation of therapeutics aimed at managing microbiota, or the spectrum of bacteria that naturally populate the human gut. They propose that the gut microbiome plays an important role in metabolizing the diet to liberate factors that ultimately protect against neurological damage that contributes to ALS pathology. The principal investigator of the study is Christopher Reyes. For more information, click here.

ImmunoBrain Checkpoint was awarded $499,413 to further studies targeting immune-checkpoint pathways, another therapeutic approach for ALS. The immune system, beyond its immediate role in fighting pathogens, is involved in supporting brain function, in health and in disease. In neurodegenerative conditions, and specifically in ALS, immune system failure is characterized along with disease progression. Currently, in advance stages of developing this approach for Alzheimer’s disease, these studies aim to collect biomarkers of the evoked immune response following treatment, which they believe are key for clinical translation of the results. The principal investigator on the study is Kuti Baruch.

Click here to learn more about The Association’s research commitments and the many grants made possible by the kindness and generosity of donors to the Lawrence and Isabel Barnett Drug Development Program.

Original blog post was published here.

ALS Association Makes Multi-Year Commitment to HEALEY ALS Platform Trial

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Photo provided by Texas Tech University Health Sciences Center in El Paso

This week a $3 million commitment was made by The ALS Association to the first platform trial for ALS. The platform trial will be taking place at the Sean M. Healey & AMG Center for ALS at Mass General in collaboration with the Barrow Neurological Institute and the Northeast ALS Consortium (NEALS). A “platform trial” is a clinical trial in which multiple treatments are tested and evaluated simultaneously. The Association’s commitment is $1 million per year for 3 years.

“This platform trial will significantly speed efficacy trials for ALS research, and is a major advancement for ALS research,” said Calaneet Balas, President, and CEO of The ALS Association. “In addition to supporting the HEALEY ALS Platform Trial, we will be working to ensure the rest of the research pipeline is moving fast enough to keep up. We are delighted to be partnering with Dr. Merit Cudkowicz and her team on this project.”

The platform trial model will greatly accelerate therapy development, allowing investigators to test more drugs, increase patient access to trials, and reduce the cost by quickly and efficiently evaluate the effectiveness of multiple therapies. This trial mode has already been proven successful in the cancer field. New treatments will be added to the trial as they become available which will decrease the gap in time from identification of an exciting therapy to testing. Having a shared infrastructure, common data & sample collecting processes, and centralized governance within the platform trial will help save time and money and led to more operational efficiencies.

“Working closely together, we will bring new therapies forward faster for people with ALS,” said Cudkowicz. “The platform trial builds on previous ALS Association supported research on many new ALS targets and trial innovations. The HEALEY ALS Platform Trial initiative can only succeed with the support and involvement of the entire ALS community. We look forward to ongoing partnerships with people and families with ALS, all ALS foundations, our colleagues in the NEALS Consortium, industry partners and regulatory agencies,” Cudkowicz added.

This funding from The ALS Association will directly support numerous parts of the platform trial. The following will directly benefit from this funding: the Clinical and Data Coordination Center activities at Mass General, the Biomarker Development Endpoint Engine, and the Monitoring and Outcome Measures Core at Barrow Neurological Institute.

The ALS Association will become an integral part of the Foundations and Benefactors Committee responsible for advising on study progress, data sharing, budget matters, fundraising, patient recruitment/retention and variety of other scientific and operational topics. In addition, the trial will be supported by the enrollment of patients at ALS Association Certified Centers of Excellence, Recognized Treatment Centers, and other affiliated centers.

This award to the Healey Center builds on a long relationship The ALS Association has had with Massachusetts General Hospital, Dr. Cudkowicz, and other researchers. The Association has committed over $9 million since 2010 to ALS researchers at Massachusetts General Hospital.

For more information, please email healeycenterforals@mgh.harvard.edu and visit the center’s website www.massgeneral.org/als/. To read the full press release, visit www.alsa.org/.

Houston Neurologist Discovers Treatment That Could Halt the Progression of ALS

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Research Study Information and Photos Provided by Methodist Houston Hospital

Dr. Stanley Appel, neurologist and co-director of the Houston Methodist Hospital Neurology Institute, has been leading the ALS Clinic at Houston Methodist Hospital since its founding in 1982. This was the first multi-disciplinary clinic dedicated to the treatment of and research on ALS. More than 35 years later, he continues to innovate in the field.

His latest research? An immunotherapy treatment that could stop the progression of ALS in its tracks. [Read more…]

Closer to a Cure: Researchers Open Enrollment for Tofersen Phase 3 Clinical Trial

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Our “closer to a cure” segment will highlight research news and important milestones made by researchers as they work towards finding a cure for ALS.

Exciting news! Neuroscience collective Biogen has initiated a phase 3 clinical trial to evaluate Tofersen (previously called BIIB067), a type of antisense drug meant to target superoxide dismutase (SOD1), for the potential treatment of ALS. 

It’s approximated that two percent of people living with ALS have a mutation in the SOD1 gene- the second most common cause of genetic ALS. An SOD1 mutation leads to abnormal protein, which is likely toxic to cells and could lead to the death of motor neurons- the cells that die in ALS. Tofersen has been designed to reduce levels of SOD1 protein in people with ALS caused by an SOD1 gene mutation.

The trial is currently enrolling with 60 spots available for people with SOD1 ALS.

Interested in enrolling and helping researchers test the safety and efficacy of Tofersen? The ALS Association has worked with Biogen to create a FAQ surrounding the trial. The FAQ covers how to enroll, where to get more information, study design and endpoints, expanded access, and more. See the FAQ here.

For more information about the clinical trial and its currently enrolling locations, visit: https://clinicaltrials.gov/ct2/show/NCT02623699.

Closer to a Cure: What is the KIF5A ALS gene & why is it important

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Our “closer to a cure” segment will highlight research news and important milestones made by researchers as they work towards finding a cure for ALS.

While ALS is currently a disease with no cure and limited treatment options, the ALS Association continues to fund groundbreaking research to bring us closer to a cure. Last March, researchers from collaborative initiatives funded by The ALS Association, with money raised through the ALS Ice Bucket Challenge, announced the discovery of a new ALS gene- KIF5A.

You may have heard the news in the past year, but do you truly understand what the KIF5A gene is and why it’s important? If your answer is “no” that’s ok! That’s what we’re here for.

What is the KIF5A ALS Gene?

The KIF5A ALS gene was discovered through a unique collaborative effort of over 250 researchers, led by Dr. John Landers at University of Massachusetts Medical School and Dr. Bryan Traynor at the NIH. Researchers analyzed genetic data from over 101,000 samples to discover the KIF5A gene and it’s actually the fifth new ALS gene discovered since the 2014 Ice Bucket Challenge.

KIF5A, or kinesin family member 5A, is a motor protein involved in the protein cargo transportation in cells. Kinesins play an important role in transporting along a motor neuron axon, which is crucial to motor neuron health. Mutations identified in KIF5A may cause disease by interrupting that axonal transport.

Why is the KIF5A ALS Gene Important?

Identifying common pathways leading to ALS is an important part of creating new treatments to slow or reverse the progression of ALS. The more we understand about the mechanics of the disease, the closer we get to finding a cure.

ALS is a disease that’s incurable for now, but with more research we have hope that a cure will be discovered.