ALS gradually destroys the nerve cells that control voluntary movement, leading to progressive paralysis while mental function typically remains intact. About 5,000 people are diagnosed in the US each year. Treatment options remain limited, with riluzole and edaravone modestly slowing progression and recent approvals like tofersen targeting specific genetic mutations.
What's actually going on in research
Trials are testing drugs that reduce neuroinflammation, protect motor neurons from dying, and address specific genetic causes. Tofersen was approved in 2023 for people with SOD1 mutations, and other antisense oligonucleotides are in development for different ALS genes. Researchers are also studying combination therapies, stem cell approaches, and drugs that target the protein aggregates found in ALS motor neurons.
Gene-targeted therapies
Antisense oligonucleotides can block specific genes that cause inherited ALS. Tofersen for SOD1 mutations is now available, and similar drugs for C9orf72 and FUS mutations are in trials.
Neuroinflammation
Several trials are testing drugs that calm the immune cells in the brain and spinal cord, which may contribute to motor neuron death. This includes drugs that target specific immune pathways and repurposed anti-inflammatory medications.
Stem cell trials
Researchers are testing stem cells injected into the spinal cord to support surviving motor neurons or replace lost cells. Early trials focus on safety and whether the cells survive and function.
What to know before you search
Eligibility typically depends on time since symptom onset, rate of progression, breathing function, and for some trials, specific genetic mutations.
What types of trials are currently open
- Treatment trials — Testing new drugs or drug combinations to slow ALS progression, often measured by changes in muscle strength, breathing function, and ability to perform daily tasks.
- Gene therapy trials — Testing antisense oligonucleotides or gene therapies for people with specific ALS mutations like SOD1, C9orf72, or FUS.
- Stem cell trials — Studies of stem cells delivered to the spinal cord, testing safety and potential benefit.
- Biomarker studies — Research to identify blood or spinal fluid markers that can track disease progression and predict treatment response.
- Natural history studies — Following people with ALS over time to understand how the disease progresses and what factors influence survival.
Recently added ALS trials
Receive emergency blood vessel access during resuscitation
The FEMART-1 Pilot Study is focused on evaluating the feasibility, safety, and procedural timing of prehospital femoral arterial sheath placement in patients with non-traumatic out-of-hospital cardiac arrest (OHCA), both during ongoing cardiopulmonary resuscitation and after return of spontaneous circulation (ROSC). The primary objective of the study is to enable invasive arterial blood pressure monitoring for targeted and continuous hemodynamic management using vasopressor therapy in the prehospital setting. The study evaluates the feasibility, safety, and procedural performance of invasive arterial monitoring with the aim of improving assessment of the patient's hemodynamic status and enabling more accurate titration of vasopressor support. This approach may reduce episodes of hypotension and decrease the risk of recurrent cardiac arrest while allowing safer transport to specialized cardiac arrest centers. Femoral arterial access enables more precise monitoring of perfusion pressure, targeted vasopressor titration, and early recognition of impending circulatory collapse. The intervention may contribute to improved early organ perfusion and could be associated with more favorable neurological and overall clinical outcomes after cardiac arrest. In accordance with the ERC Guidelines 2025, which emphasize active hemodynamic optimization after ROSC and acknowledge the potential role of invasive arterial pressure monitoring during ongoing resuscitation, the study evaluates not only feasibility, safety, and procedural timing, but also the potential clinical benefit of continuous hemodynamic-guided management in the prehospital phase, including (1) early identification of hypotension, (2) targeted vasopressor administration, and (3) prevention of re-arrest. The study is conducted by the Prague Air Rescue Service Kryštof 01 (Prague Emergency Medical Services) in collaboration with the Central Bohemian Emergency Medical Service, the Second Department of Internal Medicine - Cardiology and Angiology of the General University Hospital in Prague and First Faculty of Medicine, Charles University, and the Department of Anesthesiology, Resuscitation and Intensive Care Medicine of the General University Hospital in Prague and First Faculty of Medicine, Charles University. FEMART-1 is designed as a prospective pilot study without external funding.
Complete a hearing test to assess middle ear function
There is a need for a non-behavioral clinical test that provides an assessment of how speech is conducted through the middle ear for the optimization of middle ear implants. These implants utilize the functionality of the human middle ear and require adequate speech conduction through the middle ear chain. Consequently, the clinical test of this degree of speech transfer can contribute to the pre-operative indication for middle ear implants. Wideband Tympanometry (WT) is a non-invasive diagnostic tool for the clinical assessment of the condition of the middle ear. Unlike classical tympanometry, WT tests the impedance of the middle ear system as a function of both pressure and frequencies. Consequently, a WT measurement contains many times more information about the functionality of the middle ear than classical tympanometry and is ideally suited as a diagnostic tool for the clinical assessment of speech transfer through the middle ear. The WT measurements will be correlated with the Speech Intelligibility Index (SII) collected in normally hearing individuals and patients with isolated conductive hearing loss. This index is a measure of speech transfer through the middle ear. Consequently, a correlation of the WT measurements with the SII addresses the need for a non-behavioral clinical test of speech transfer through the middle ear. Additionally, WT measurements will be performed longitudinally in cochlear implant candidates, one of the populations that can benefit from the developed clinical test. These longitudinal WT measurements, which will be performed both pre- and post-operatively, allow for the evaluation of the impact of middle ear surgery on the predicted SII.
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