Investigational Amyotrophic Lateral Sclerosis Treatments to Watch in 2026
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative condition in which motor neurons in the brain and spinal cord gradually lose function and die. These neurons control voluntary muscle movement, including walking, speaking, swallowing, and breathing. As motor neurons deteriorate, muscles weaken and waste away over time, eventually leading to severe physical disability.
ALS is difficult to treat because multiple biological processes appear to contribute to neuron damage, including inflammation, impaired protein handling, cellular stress responses, and immune dysfunction. Existing treatments may slow disease progression for some individuals, but they do not stop or reverse neuron loss. As a result, current research focuses on experimental approaches that attempt to intervene in these underlying disease mechanisms rather than solely managing symptoms. The complexity of ALS biology means that many experimental treatments target different pathways, and long-term effectiveness and safety remain key unanswered questions.
Important: This article is for informational purposes only. Being listed here does not mean a therapy works or is recommended. Clinical trial participation should always be discussed with a healthcare professional.
Below are five ALS clinical trials involving experimental treatments that represent different research approaches.
1. FHND1002 Experimental Drug Study
What is this study evaluating?
This study is testing FHND1002, an experimental pill designed specifically for people with ALS. Participants take the medication at set doses over many months so researchers can see how the body handles the drug when it is used regularly, not just once or twice. The study also looks for changes in biological signals linked to nerve cell damage, helping researchers understand whether the drug is interacting with processes involved in ALS.
How could this approach change the way ALS is treated?
Most ALS treatments focus on managing symptoms after nerve cells have already been damaged. This study is asking whether a pill can directly interact with disease-related processes earlier, inside the nervous system. If the drug shows consistent biological activity, it could support future treatments that target specific disease pathways rather than only slowing symptoms.
Study link:
https://clinicaltrials.gov/study/NCT07138014 ClinicalTrials.gov
2. XT-150 Gene-Based Therapy Study
What is this study evaluating?
This study is evaluating XT-150, an experimental gene-based therapy designed to deliver instructions that allow cells in the nervous system to produce a modified form of interleukin-10 (IL-10), a naturally occurring anti-inflammatory signaling molecule. Rather than acting like a traditional drug, XT-150 is delivered via intrathecal injection into the spinal canal and aims to influence inflammation by changing how cells produce specific signals internally.
How could this approach change the way ALS is treated?
Inflammation is thought to contribute to motor neuron damage in ALS, but broadly suppressing the immune system can cause serious side effects. This study is addressing whether localized genetic delivery can safely influence inflammatory signaling within the nervous system. The key question is whether this approach can be administered safely and tolerated in people with ALS, which is a necessary step before determining whether modifying inflammatory pathways could influence disease progression.
Study link:
https://clinicaltrials.gov/study/NCT06704347 ClinicalTrials.gov
3. Digoxin Repurposing Study in ALS
What is this study evaluating?
This study is evaluating digoxin, a medication already approved for certain heart conditions, in people with ALS. Researchers are examining whether digoxin affects biological markers and clinical outcomes associated with ALS when given at carefully monitored doses.
How could this approach change the way ALS is treated?
Repurposing existing medications allows researchers to focus on biological effects rather than early safety development, since much is already known about the drug’s general safety profile. This study is exploring whether digoxin interacts with molecular pathways relevant to ALS. The central question is whether those effects are consistent and meaningful enough to justify further ALS-specific investigation.
Study link:
https://clinicaltrials.gov/study/NCT07047209 ClinicalTrials.gov
4. Dazucorilant (CORT113176) Study
What is this study evaluating?
This study is evaluating dazucorilant (CORT113176), an investigational oral drug designed to target the body’s stress-response signaling system. Chronic cellular stress is believed to contribute to neuron vulnerability in ALS. Researchers examined whether the drug could influence disease-related processes while remaining safe and tolerable.
How could this approach change the way ALS is treated?
Most ALS therapies do not directly target stress-response signaling. Although the primary functional endpoint of this study was not met, longer-term analyses reported by the sponsor suggested differences in survival at certain dose levels. The unresolved question is whether modifying stress-response pathways can influence disease course over longer timeframes and how future studies should be designed to evaluate that possibility.
Study link:
https://clinicaltrials.gov/study/NCT05357950 (link to trial listing) ClinicalTrials.gov
5. RAPA-501 Stem Cell-Based Research Protocol
What is this study evaluating?
This study is evaluating RAPA-501, a personalized stem cell-based research protocol that uses a participant’s own regulatory immune cells. These cells are collected, expanded, and modified outside the body before being returned. The study focuses on how immune regulation may relate to motor neuron damage in ALS.
How could this approach change the way ALS is treated?
Immune system abnormalities have been observed in ALS, but their role in disease progression remains unclear. This study is examining whether a personalized immune-based approach can be delivered repeatedly and safely, and how the body responds over time. The key question is whether altering immune regulation can provide insights into ALS biology and inform whether immune-focused strategies warrant further investigation.
Study link:
https://clinicaltrials.gov/study/NCT04220190 ClinicalTrials.gov
Final Takeaway
These ALS studies are focused on learning whether different processes in the body — such as inflammation, stress inside cells, immune activity, and specific molecular signals — can be safely adjusted in people with ALS. What researchers learn will help decide which treatment ideas should be studied further and how future ALS trials are designed.
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Disclaimer
This article is for informational purposes only. The information shared here summarizes publicly available research, clinical trials, and scientific developments and no guarantees are made regarding accuracy, completeness, or current relevance. Always consult a qualified healthcare professional regarding any health-related decisions.