Understanding ALS research
ALS research is advancing faster than at any point in its history. Scientists understand more about the genetics and biology of ALS, have better tools for measuring disease progression, and are pursuing new approaches to treatment.
Understanding where the field stands today can make the ALS research landscape easier to navigate.
Why do ALS treatments take time?
Developing effective treatments starts with understanding what is happening in the disease. Researchers need to identify the biological processes involved in ALS, determine which of those processes could be targeted by a potential therapy, and develop ways to measure whether a treatment is having its intended effect.
ALS makes this especially complex. Researchers have identified more than 40 causative and risk genetic factors associated with ALS, and multiple biological processes may contribute to how the disease develops and progresses. For many years, researchers also lacked reliable biomarkers to determine whether an experimental treatment was altering the disease’s underlying biology.
That’s beginning to change. Advances in genetics, disease biology, biomarkers, and research tools are giving scientists a clearer picture of ALS and enabling earlier evaluation of promising approaches in the development process.
The current state of research
Where science stands today and where it’s heading. Explore the latest breakthroughs, clinical trials, promising treatments, and the rapid pace of progress.
Where is progress happening in ALS research?
There is still an urgent need for more effective treatments. At the same time, several developments are changing what scientists can study and how quickly they can advance promising ideas.
Understanding the biology of ALS
Researchers are learning more about the biological processes that contribute to ALS. One important example is TDP-43, a protein involved in processing RNA inside cells. Abnormal TDP-43 is found in approximately 97% of ALS cases, making it an important focus for researchers working to identify new drug targets. At the same time, research is revealing a more complex picture of ALS involving the immune system, support cells, blood vessels, and gene regulation. Understanding the underlying biology of ALS is the critical first step to developing biomarkers and novel treatments for the disease.
Better ways to measure disease
Emerging biomarkers are providing researchers with better ways to measure biological processes. Neurofilament light chain (NfL), a marker of nerve cell damage, can help researchers assess whether a potential treatment affects disease biology and make more informed decisions earlier in the research process. Researchers are working to develop a broader suite of biomarkers to help diagnose ALS, track disease progression, and better understand differences among people with the disease.
Treatments designed around disease biology
In 2023, the FDA approved Tofersen, the first gene-targeted treatment for ALS, for people with ALS caused by mutations in the SOD1 gene. Although Tofersen applies to a small subset of people with ALS, its development demonstrates that understanding a specific biological driver of the disease can lead to a therapy designed to target it. Across the field, researchers are working to advance discoveries rooted in fundamental ALS biology toward potential treatments.
How does Target ALS fund research?
Target ALS funds promising research worldwide, with a focus on moving discoveries out of silos and toward effective treatments. Our funding spans the ALS research landscape, from understanding the disease’s fundamental biology and identifying new therapeutic targets to accelerating drug discovery and developing biomarkers.
Our approach includes collaborative grants that bring together multidisciplinary teams from academia and industry, as well as individual investigator grants that support emerging scientists and new ideas in ALS research.
Want to participate in ALS research?
People living with ALS and their families are essential partners in research. Participation can take many forms, including clinical trials, natural history studies, and biosample donation.
Learn more about participating in research
Advancing ALS research relies on the generous participation of people with ALS and healthy volunteers in clinical studies.
Learn more about the ALS Global Research Initiative
Target ALS launched the ALS Global Research Initiative (AGRI) to tackle one of the field’s most urgent challenges: the need for research that reflects the full ALS community.
Learn more about brain and spinal cord donation
Brain and spinal cord donation is one of the most meaningful gifts a person with ALS, or their loved ones, can offer.
The information in this Resource Center is for general educational and informational purposes only and is not intended as medical, health, legal, financial, or insurance advice.
Target ALS is a medical research foundation and does not provide medical care or professional advice. This information should not be used as a substitute for guidance from qualified healthcare providers or other professionals familiar with your individual circumstances. Information about treatments, benefits, insurance coverage, and other resources may change over time. Please consult the appropriate healthcare provider, government agency, insurer, or other qualified professional for information specific to your situation.
If you are experiencing a medical emergency, call 911 or go to your nearest emergency room.
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