How to Prevent ALS Someday: Current Research and Strategies

What Makes 2025-2026 a Turning Point for ALS Prevention and Treatment

Why 2025 marks a shift from symptom management to disease modification

For more than three decades after riluzole's approval, ALS treatment focused primarily on slowing symptoms rather than changing the disease's course. That is now changing: tofersen, approved by the FDA in 2023 for [SOD1-linked ALS](https://alsunited.org/blog/familial-als-inheritance-pattern), is the first therapy to demonstrate real disease modification, with roughly one-quarter of patients showing stabilization or functional improvement over long-term follow-up [1]. A 2025 clinical trial also found that adding low-dose interleukin-2 to standard treatment reduced the risk of death by over 40% in a subset of patients, identifying neuroinflammation as another viable therapeutic target [2]. With over 160 ALS trials currently underway worldwide, ALS treatment options in 2025 signal a field now oriented toward changing outcomes, not just managing them [3].

FDA-approved treatments now available that slow progression: Riluzole, Edaravone, and Sodium Phenylbutyrate-Taurursodiol

Three FDA-approved drugs target ALS disease progression: riluzole, edaravone, and -- until its 2024 market withdrawal -- sodium phenylbutyrate-taurursodiol (PB-TURSO). Riluzole, approved in 1995, extends survival by roughly 60 to 90 days by reducing glutamate toxicity to motor neurons, with no measurable effect on physical function. [4] Edaravone, a free radical scavenger available as an [oral therapy since 2022](https://alsunited.org/blog/radicava-a-complete-guide-to-its-purpose-and-value-for-als-patients), may slow functional decline by 33% in patients with early-stage disease, though recent long-term data have not consistently confirmed that benefit across broader populations. [4] PB-TURSO (Relyvrio) was approved in 2022 after Phase 2 results showed slowed functional decline and improved survival, but Amylyx voluntarily withdrew it from the U.S. market in April 2024 after a 664-patient Phase 3 trial failed to meet its primary endpoints -- leaving riluzole and edaravone as the current FDA-approved options for slowing ALS progression. [5]

How ALS United connects you to the latest approved therapies through our medical resources and clinic finder

Knowing which therapies exist matters less if you can't access a specialist who can prescribe and monitor them. Multidisciplinary ALS clinics provide coordinated care across neurology, nutrition, respiratory therapy, and speech pathology in a single visit, and clinical guidelines consistently support their role in improving survival and quality of life for people with ALS. [6] Our [care services and clinic finder](https://alsunited.org/blog/our-care-services) connect you directly to accredited ALS centers where current therapies -- including riluzole and edaravone -- can be evaluated, prescribed, and managed in one place. [7] We are here for you with updated medical resources, insurance navigation support, and direct referrals to specialists who understand the full landscape of ALS treatment options in 2025 and beyond.

Most Promising Clinical Trials and Emerging Therapies for ALS in 2025-2026

Gene therapy approaches showing early success in SOD1 and C9orf72-linked ALS

Tofersen works as an antisense oligonucleotide (ASO) -- a precision gene therapy that binds directly to SOD1 messenger RNA and blocks production of the toxic, misfolded protein that accumulates in and damages motor neurons. [8] Its FDA accelerated approval in 2023 was based on a roughly 60% reduction in plasma neurofilament light chain, a measurable biomarker of neuronal injury, even after the phase 3 VALOR trial did not meet its primary functional endpoint. [9] For C9orf72-linked ALS -- the most common genetic subtype, found in about 40% of [familial ALS](https://alsunited.org/blog/does-als-run-in-families/) cases and 10% of sporadic cases -- similar ASO approaches like WVE-004 are now in active clinical trials, and a 2025 study found that anti-inflammatory T cell responses targeting C9orf72 correlate with longer predicted survival, suggesting immunotherapy could complement gene silencing strategies. [10]

Stem cell and neuroinflammation research: How immunotherapy may slow neuronal decline

ALS Research Timeline & Comparison Table: Track FDA-approved treatments, clinical trials, and emerging therapies from 2023-2026 with efficacy data and accessibility through ALS United's clinic finder The table below tracks FDA-approved ALS drugs and active trial therapies together, showing mechanism, eligibility, current status, and how to access each option through our clinic network. Two drugs are currently approved for slowing ALS progression: riluzole, which reduces glutamate toxicity to motor neurons, and edaravone, which targets oxidative stress -- though edaravone's benefit was demonstrated in a selected early-stage subpopulation and has not consistently generalized to broader populations.[13] Tofersen, approved in April 2023, applies only to patients with a confirmed SOD1 gene mutation and is delivered by intrathecal injection into the spinal fluid.[13] Sodium phenylbutyrate-taurursodiol (Relyvrio) was voluntarily withdrawn from the U.S. market in April 2024 after the 664-patient PHOENIX Phase 3 trial failed to change disease progression on the ALSFRS-R or meet any secondary endpoints.[5]

| Treatment | Mechanism of Action | FDA Status | Patient Eligibility | Trial Status (2023-2026) | Access via Our Network | |---|---|---|---|---|---| | **Riluzole** | Reduces glutamate toxicity; blocks voltage-dependent sodium channels | Approved (1995) | All ALS patients | Standard of care; included as background therapy in most active trials | Available at ALS United-affiliated clinics | | **Edaravone (Radicava / Radicava ORS)** | Free radical scavenger; reduces oxidative stress | Approved (IV 2017; oral 2022) | Early-stage ALS meeting specific functional criteria; benefit not confirmed in broader populations | Standard of care for eligible patients | Available at ALS United-affiliated clinics | | **Tofersen (Qalsody)** | Antisense oligonucleotide (ASO) binds SOD1 mRNA, reducing toxic protein production | Approved April 2023 (accelerated) | Confirmed SOD1 gene mutation required | Ongoing open-label extension; biomarker follow-up studies active | Accessible via ALS United centers with genetic testing capability | | **PB-TURSO (Relyvrio / AMX0035)** | ER stress inhibition + mitochondrial apoptosis protection | Withdrawn April 4, 2024 | No longer available to new patients | PHOENIX Phase 3 failed (ALSFRS-R p=0.667); PHOENIX OLE available to prior enrollees only | Not available for new starts; prior patients may transition to PHOENIX OLE | | **ASO / Gene Therapies** (e.g., C9orf72-targeted, TDP-43-targeted) | Downregulate mutant gene expression via RNA silencing (ASO or RNAi) | Investigational | Genetically confirmed ALS subtypes; varies by trial | Multiple Phase 1/2 trials active in 2025-2026 | Ask your ALS United care team about genetic trial eligibility | | **Cell Therapies** (MSC, hNSC) | Neuroprotective and anti-inflammatory support via cell delivery | Investigational | Varies by trial protocol; intrathecal delivery most studied | Multiple trials ongoing; optimal cell type and delivery route under evaluation | Ask your ALS United care team about trial eligibility | | **Immunotherapy** (IL-2 / Treg expansion) | Expands regulatory T cells to reduce neuroinflammation and motor neuron loss | Investigational | Varies; neurofilament biomarker stratification being studied for patient selection | Phase 2 data published; biomarker-stratified follow-up trials planned for 2025-2026 | Ask your ALS United care team about immunotherapy trial access |

With over 160 ALS studies planned or ongoing across gene therapies, cell therapies, immunotherapies, and novel small molecules, the breadth of ALS research in 2025-2026 is wider than at any previous point.[14] Eligibility criteria differ significantly across these trials -- genetic subtype, disease stage, baseline neurofilament levels, and functional score all factor into which options apply to any given person. Our care team can help you navigate this table in the context of your specific situation, connecting you to the right specialists through our clinic finder so no eligible option goes unexplored.

Preventive Strategies and Risk Reduction While We Wait for a Cure

Lifestyle factors being studied: Exercise, diet, and cognitive engagement in ALS progression

Exercise intensity matters in ALS: moderate, low-impact activity such as swimming, walking, or stationary cycling supports muscle function and quality of life, while high-intensity endurance training has accelerated symptom onset in animal models and produced inconsistent results in patient trials. [15] A 2024 review found that swimming-based protocols extended median survival in SOD1 mice by roughly 25 days by shifting muscle energy metabolism toward glucose use -- and [structured physical therapy exercises](https://alsunited.org/blog/upper-body-physical-therapy-exercises-for-als) offer a practical starting point for applying this principle to daily care. [15] Diet is equally relevant: ALS disrupts energy metabolism early, and high-calorie, lipid-rich diets have shown neuroprotective effects in mouse models while caloric restriction worsened motor symptoms -- making nutrition another modifiable factor to discuss with your ALS care team. [15]

Early screening and genetic counseling for at-risk families: What the research shows

Genetic mutations account for fewer than 20% of ALS cases, but for families with a known variant in genes such as SOD1, C9orf72, FUS, or TARDBP, early screening creates clinical pathways that don't exist without a confirmed diagnosis. [17] The Pre-fALS study, an ongoing longitudinal study of presymptomatic mutation carriers, is building the biomarker framework needed to detect ALS before symptoms emerge -- including neurofilament light chain levels, which rise measurably in the presymptomatic phase. [16] Genetic counseling helps at-risk individuals understand their [personal risk factors](https://alsunited.org/blog/als-risk-factors-what-you-need-to-know/), navigate family planning decisions, and qualify for presymptomatic intervention trials -- including prevention-focused studies targeting SOD1 and C9orf72 carriers before motor symptoms develop. [17] A 2025 consensus guidance document recommends structured neurological monitoring at regular intervals for confirmed pathogenic variant carriers, formalizing a proactive care model for people who are currently asymptomatic. [16]

How ALS United's counseling and support services help you manage uncertainty and build resilience

ALS creates significant psychological weight for both patients and caregivers -- depression affects 10% to 45% of people with ALS, and roughly 70% of family caregivers experience significant caregiving burden.[18] Research shows that emotional support and open dialogue are the elements most valued across therapy approaches, with Acceptance and Commitment Therapy selected most often by ALS patients in clinical studies for its focus on building psychological flexibility rather than eliminating distress.[18] Our [support groups and counseling services](https://alsunited.org/blog/als-support-groups-connecting-with-others-facing-the-disease/) match the format to the person: individual sessions for caregivers who need tailored coping strategies, and peer group options for patients who benefit from shared experience.[19] We are here for you at every stage, with referrals to ALS-familiar therapists, facilitated connection groups, and mindfulness tools available regardless of insurance status.[19]

Taking Action Today: How to Access Treatments and Join the Prevention Movement

Finding clinical trials near you: Using ALS United's clinic finder and trial databases

ClinicalTrials.gov lists every registered ALS study in the United States, and you can filter results by location, disease phase, enrollment status, and genetic subtype to narrow the list to trials that match your situation. [20] The ALS Trial Navigator adds a second layer of matching by cross-referencing your medical history and disease stage against current eligibility criteria. [21] Once you identify a relevant trial, the next step is contacting the listed research coordinator -- typically a nurse or trained clinical staff member who handles enrollment, scheduling, and baseline assessments. [20] Our care team can help you interpret trial listings, confirm eligibility, and connect you with [ALS neurologists and specialists](https://alsunited.org/blog/what-type-of-doctor-treats-als-understanding-als-medical-care) who can evaluate your options and explain how a trial fits into your overall care plan. [22]

What to discuss with your neurologist about 2025 treatment options and eligibility

Three eligibility factors determine which ALS treatment options in 2025 apply to you: confirmed genetic subtype, current disease stage, and your ALSFRS-R functional score, all of which your neurologist can assess as part of a structured [ALS diagnosis evaluation](https://alsunited.org/blog/als-diagnosis-steps-to-confirming-the-condition/). [6] Ask specifically whether genetic testing has confirmed or ruled out mutations like SOD1 -- tofersen is available only to patients with a confirmed SOD1 variant, and active trials for C9orf72-linked ALS require genotype confirmation before enrollment. [6] Edaravone eligibility is restricted to early-stage patients meeting specific functional criteria, so the timing of that conversation affects your options directly. [6] Your neurologist can also review neurofilament light chain levels, which now factor into biomarker-stratified trial eligibility and provide a measurable baseline for tracking treatment response. [6]

Join ALS United's advocacy and research community: How your participation accelerates prevention breakthroughs

Advocacy participation by ALS patients and families has produced concrete policy results: the elimination of the five-month SSDI waiting period for people with ALS, increased NIH and CDMRP research funding, and the ACT for ALS Act, which created expanded access grants for those ineligible for clinical trials and launched the AMP ALS public-private research partnership. [23] An advocacy survey of over 550 participants in 2025 directly shaped the public policy agenda ALS United brought to Congress, demonstrating that community input has documented influence on research funding priorities. [23] You can add your voice through our [advocacy action alerts](https://www.alsunited.org/advocacy-action-alerts), where each alert connects to a specific policy ask our team is actively pursuing -- because together in the fight, sustained community engagement is what moves prevention research forward. [23]