ALS was first identified in the 1860s by French neurologist Jean-Martin Charcot. Although clinical interventions have evolved and improved quality of life for many, there are still no approved treatments that significantly slow disease progression for the vast majority of people living with ALS today.
Over the past few years, however, research progress has accelerated and there is growing optimism among ALS researchers that the tide is turning.
Over the past few years, however, research progress has accelerated and there is growing optimism among ALS researchers that the tide is turning.
Building a Better Understanding
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For decades, researchers knew very little about the biology of ALS and the underlying mechanisms they would need to target with drugs. Now they have a foundational understanding that continues to grow and inform current and future research efforts.
Researchers have realized that there are multiple forms of ALS, and that each form may develop and progress differently. They are now studying treatments for different subtypes of ALS to better understand which approaches might work best for different groups of people. |
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“The last 20 years have been revolutionary in terms of what we understand about the causes of ALS and the actual processes within the brain and nerves that produce damage. Our clinical research efforts are now looking at drugs that impact targets that we didn't even know existed a few years ago.”
- Dr. Jeremy Shefner, Barrow Neurological Institute
- Dr. Jeremy Shefner, Barrow Neurological Institute
Identifying biomarkers (measurable indicators of what is happening inside the body) is a big focus of ALS research. Once validated, biomarkers could be used for purposes such as measuring a drug’s effectiveness in clinical trials and diagnosing ALS with a blood test.
Researchers have also discovered that in almost all cases of ALS, a normally useful protein called TDP-43 stops working properly and becomes toxic. Understanding why this happens could play a role in developing effective treatments for the majority of people living with ALS.
Researchers have also discovered that in almost all cases of ALS, a normally useful protein called TDP-43 stops working properly and becomes toxic. Understanding why this happens could play a role in developing effective treatments for the majority of people living with ALS.
Non-Drug Interventions
Attending a multidisciplinary ALS clinic and using interventions like noninvasive ventilation can help people diagnosed with ALS live longer and have a better quality of life. Learn more
Research Progress on Genetic ALS
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Researchers now know that some people’s ALS has a genetic cause. Since 1993, variants (sometimes called mutations) have been identified in over 40 genes that either cause ALS or increase its likelihood.
Decades of research and discovery have led to meaningful progress in genetic ALS research in recent years. In 2023, the approval of tofersen (Qalsody), the first genetically targeted therapy for ALS in the U.S., marked an important milestone in ALS research. |
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“Tofersen is an example of what I’ve been looking for for my whole career—proof that if we target a subset of ALS, we can have an amazing effect. Their ALS has stopped progressing. Some are actually stronger now. This used to be thought of as impossible.”
- Dr. Richard Bedlack, Duke ALS Clinic
- Dr. Richard Bedlack, Duke ALS Clinic
Tofersen has slowed disease progression for some people with a variant in the SOD1 gene. Researchers are currently trying to replicate this approach in clinical trials with experimental drugs that target variants in other genes.
Even though tofersen is only available for approximately 2% of all people living with ALS in the U.S., results have demonstrated that it is possible to slow ALS progression in a meaningful way for some people.
Even though tofersen is only available for approximately 2% of all people living with ALS in the U.S., results have demonstrated that it is possible to slow ALS progression in a meaningful way for some people.
More Sophisticated Clinical TrialsTo find out if experimental drugs are safe and effective, they must go through clinical trials, which are becoming more targeted as researchers learn more about the disease.
In 2020, the Healey ALS Platform Trial introduced a new approach to ALS clinical trials that shares a placebo group and tests multiple drugs at once. This model reduces the percentage of people on placebo and accelerates the drug testing process. |
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Today there are many more ALS clinical trials than before, and advocates continue to push to make trials more patient friendly.
Increased Funding for ALS Research
Thanks to advocacy efforts by ALS organizations and individuals impacted by ALS, research funding has increased dramatically in recent years. This funding has helped create an infrastructure that can further accelerate progress.
On a federal level, the largest funder of ALS scientific research is the National Institutes of Health (NIH). ALL ALS, the largest ALS observational study to date, is collecting data and biosamples from people living with ALS and those at risk. As part of the Accelerating Medicines Partnership® program (AMP ALS), the ALS Knowledge Portal contains centralized data that is freely available to researchers. Under the direction of the Centers for Disease Control (CDC), the National ALS Registry is working to identify risk factors and trends.
On a federal level, the largest funder of ALS scientific research is the National Institutes of Health (NIH). ALL ALS, the largest ALS observational study to date, is collecting data and biosamples from people living with ALS and those at risk. As part of the Accelerating Medicines Partnership® program (AMP ALS), the ALS Knowledge Portal contains centralized data that is freely available to researchers. Under the direction of the Centers for Disease Control (CDC), the National ALS Registry is working to identify risk factors and trends.
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"ALS advocacy is an essential component of unlocking ALS. Advocates continue to push for increased and impactful research funding at every step of the process. We would not be where we are today without our legislative and agency partners who listen to advocates and put ideas into action. That gives me great hope."
- Andrea Pauls Backman, Founder, ALS Strategy Consulting |
ALS Research Timeline- 1860s ALS is first identified - 1939 Lou Gehrig is diagnosed - 1993 First ALS-related gene is identified - 1995 First drug for ALS is approved - 2010 The National ALS Registry opens - 2014 The Ice Bucket Challenge goes viral - 2017 The second ALS drug is approved - 2021 ACT for ALS becomes law - 2023 Tofersen (Qalsody) is approved - 2024 ALL ALS begins enrolling |
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There are many other longstanding studies, programs, and research efforts that are building knowledge and sharing data. Researchers in the U.S. and around the world collaborate, meet, speak at conferences, and share what they have learned in a collective effort to end ALS.
As more and more information is gathered through observational studies and other research efforts, artificial intelligence may also be able to analyze the data, identify trends, and contribute to the solution. |
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Looking Ahead
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Research progress is still too slow for everyone impacted by ALS. But progress is happening—and it is accelerating.
There are more clinical trials than ever before, and more drugs are in the development pipeline. As researchers learn more about ALS, experimental drugs are becoming more targeted and clinical trials are becoming more sophisticated. |
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As of 2026, the ALS community is awaiting results from multiple clinical trials, including one that is testing another gene-targeted drug and one that is aiming to prevent ALS for certain SOD1 gene carriers who don’t yet have symptoms.
Researchers are hopeful that what they learn about genetic ALS will ultimately lead to breakthroughs for people whose ALS does not have a known genetic cause.
With everyone working together—including people impacted by ALS, nonprofit organizations, advocates, researchers, government agencies, and biotech and pharmaceutical companies—we continue to move closer to slowing, stopping, and ending this devastating disease.
Researchers are hopeful that what they learn about genetic ALS will ultimately lead to breakthroughs for people whose ALS does not have a known genetic cause.
With everyone working together—including people impacted by ALS, nonprofit organizations, advocates, researchers, government agencies, and biotech and pharmaceutical companies—we continue to move closer to slowing, stopping, and ending this devastating disease.
