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    ALS research tracker: treatments, trials and milestones

    ALS treatment has moved from broad drugs with modest effects to gene-targeted antisense medicines: tofersen for SOD1-ALS won FDA accelerated approval in 2023, and ulefnersen for FUS-ALS met its phase 3 primary endpoint in September 2026.[1][2] No treatment stops or reverses ALS as of October 2026.[3]

    Editor reviewedStrict sourcingUpdated DevelopingHealth and medicine, Life sciences, Neuroscience and brain-computer interfaces

    What we know

    • No approved treatment stops or reverses ALS[3]
    • Riluzole probably extends median survival by about two to three months (Cochrane, 2012)[19]
    • Tofersen has FDA accelerated approval (2023) and EU approval under exceptional circumstances (2024) for SOD1-ALS[15]
    • Relyvrio was withdrawn in 2024 after a larger trial failed to confirm benefit[6]
    • Ulefnersen met its phase 3 primary endpoint in FUS-ALS (company topline, September 2026)[2]
    • Ulefnersen is not approved by any regulator as of October 2026[12]
    • The ATLAS trial of tofersen in presymptomatic SOD1 carriers expects primary completion in August 2027[14]

    What we don't know yet

    • The full FUSION data, including effect sizes, which have not yet been presented or peer-reviewed.
    • Whether and when the FDA or other regulators will accept a filing for ulefnersen, and on what timetable.
    • Whether starting tofersen before symptoms delays or prevents ALS in SOD1 carriers (ATLAS).
    • Whether the antisense approach can be extended to the common C9orf72 form or to sporadic ALS.
    Story status

    Story status

    State
    developing
    Started
    2012-03-14
    Timeline entries
    11
    Last entry

    Timeline data (JSON)

    This tracker follows treatment and research milestones in amyotrophic lateral sclerosis (ALS), a progressive disease of the motor neurons, with the newest first in the timeline.[4] For background, start with ALS explained. For drug-by-drug evidence, see the ALS treatments guide. This page is not medical advice; talk to a neurologist or ALS care team about treatment.

    Where things stand

    As of October 2026, FDA-approved drugs may prolong survival, slow decline or manage symptoms, but none stops or reverses ALS.[3] NINDS counts four disease-modifying approvals and describes their impact as modest.[5] One of them, Relyvrio, was withdrawn in 2024.[6] The main recent progress has come in rare genetic forms, which antisense drugs can target directly.[7][2]

    The FUSION result

    On 22 September 2026, Otsuka and Ionis said ulefnersen met the primary endpoint of the phase 3 FUSION trial in FUS-ALS. The endpoint was a joint-rank measure of function and survival through day 505, with p=0.0005 against placebo.[2] They reported that secondary endpoints, including neurofilament light, also favoured the drug, and that most adverse events were mild or moderate.[8] The trial enrolled 89 people.[9] FUS-ALS makes up an estimated 0.6% of all ALS but about half of juvenile cases.[10] The ALS Association called it the second antisense therapy, after tofersen, to show it can change the course of a genetic form of ALS.[11] These are company-reported topline results. Detailed data are due at a medical congress and in a peer-reviewed journal, and the drug is not approved.[12]

    Tofersen and the presymptomatic question

    Tofersen won accelerated approval in the US in 2023 on a biomarker. In its phase 3 trial it missed the primary functional endpoint.[1][13] The ATLAS trial is testing whether starting it in SOD1 carriers with elevated neurofilament, before symptoms appear, makes a difference. It enrolled 158 people and expects primary completion in August 2027.[14] The EU approval requires the company to study people without symptoms.[15]

    Living with ALS

    Research also targets communication. A speech neuroprosthesis let one man with ALS converse with 97.5% accuracy over 8.4 months; see speech-neuroprostheses and the neurotech tracker.[16] The ALS community also lost one of its most visible advocates in October 2026. Brooke Eby, who founded the ALStogether peer community, died aged 37.[17][18]

    Timeline

    11 confirmed

    1. confirmed

      ALS Network announces the death of patient-advocate Brooke Eby, aged 37[17]

    2. confirmed

      Ulefnersen meets phase 3 primary endpoint in FUS-ALS, companies report[2][8][11]

      Otsuka and Ionis reported p=0.0005 on a joint-rank measure of function and survival through day 505; full data are to be presented and published later.

    3. confirmed

      FUSION trial reaches primary completion[9]

    4. confirmed

      ALS Network names Brooke Eby its 2026 Advocate of the Year[25][18]

    5. confirmed

      Speech neuroprosthesis lets a man with ALS converse at 97.5% accuracy (NEJM)[16][24]

    6. confirmed

      EU authorises tofersen under exceptional circumstances[15]

    7. confirmed

      FDA grants accelerated approval to tofersen (Qalsody) for SOD1-ALS[1][7]

      The approval rested on reduced plasma neurofilament light; a confirmatory trial in presymptomatic carriers continues.

    8. confirmed

      Tofersen phase 3 VALOR results published (NEJM)[13][23]

      Tofersen lowered SOD1 and neurofilament light but missed its primary functional endpoint at 28 weeks.

    9. confirmed

      FUSION phase 1-3 trial of ulefnersen in FUS-ALS begins[9]

    10. confirmed

      Edaravone phase 3 trial in selected early-stage patients published (Lancet Neurology)[21][22]

      Functional decline over 24 weeks was smaller with edaravone than placebo in a subgroup defined by an earlier post-hoc analysis.

    11. confirmed

      Cochrane review finds riluzole probably adds two to three months of median survival[19][20]

      The review pooled randomised trials with 974 riluzole-treated and 503 placebo-treated patients.

    Health & medicine

    Brooke Eby

    Brooke Eby was an ALS patient-advocate who documented life with the disease online and founded the ALStogether community. She died in October 2026, aged 37.

    More in Health & medicine

    Sources

    · 25 claims from 17 publishers

    Each numbered claim is a statement we checked against the sources listed with it. Tap a number in the text to see its sources in place.

    1. [1]

      The FDA granted accelerated approval to Qalsody (tofersen) for ALS associated with SOD1 mutations in April 2023, based on a reduction in plasma neurofilament light, a blood marker of nerve injury. confirmedas of 2023-04-25

    2. [2]

      On 22 September 2026 Otsuka and Ionis announced that the phase 3 FUSION trial of ulefnersen in FUS-ALS met its primary endpoint, a joint-rank measure of function and survival through day 505 (p=0.0005 versus placebo). confirmedas of 2026-09-22

    3. [3]

      There is no treatment that stops or reverses ALS; approved drugs may prolong survival, slow decline or manage symptoms. confirmedas of 2026-10-11

    4. [4]

      Amyotrophic lateral sclerosis (ALS), formerly known as Lou Gehrig's disease, is a progressive neurological disorder affecting motor neurons, the nerve cells in the brain and spinal cord that control voluntary muscle movement and breathing. confirmedas of 2026-10-11

    5. [5]

      NINDS says the FDA has approved one symptom-managing and four disease-modifying ALS therapies, but the impact of the disease-modifying therapies is modest. confirmedas of 2026-10-11

    6. [6]

      The FDA approved sodium phenylbutyrate/taurursodiol (Relyvrio) in September 2022 based on a single smaller trial; a larger trial failed to confirm the results and the manufacturer withdrew it from the market in 2024. confirmedas of 2026-10-11

    7. [7]

      Tofersen is an antisense oligonucleotide that targets SOD1 messenger RNA to reduce production of SOD1 protein, and it is given by spinal (intrathecal) injection. confirmedas of 2023-04-25

    8. [8]

      The companies reported that secondary endpoints, including serum neurofilament light, also favoured ulefnersen, and that most adverse events were mild or moderate. reportedas of 2026-09-22

    9. [9]

      FUSION is a randomised, double-blind, placebo-controlled phase 1-3 trial of intrathecal ulefnersen that started in June 2021, enrolled 89 people and reached primary completion in June 2026, with a 72-week blinded period followed by an open-label extension. confirmedas of 2026-10-11

    10. [10]

      FUS-ALS, caused by mutations in the fused in sarcoma (FUS) gene, accounts for an estimated 0.6% of ALS cases overall but 43-52% of juvenile and pediatric cases, and early-onset cases can be fatal within 1-2 years of symptom onset. confirmedas of 2026-09-22

    11. [11]

      The ALS Association called ulefnersen the second antisense therapy, after tofersen, to show it can change the course of a genetic form of ALS, and noted the drug was first named jacifusen after patient-advocate Jaci Hermstad, who died in 2020. confirmedas of 2026-09-22

    12. [12]

      As of October 2026 ulefnersen is not approved anywhere; Otsuka and Ionis plan to discuss expedited submission with the FDA and to present detailed results at a medical congress and in a peer-reviewed journal. confirmedas of 2026-09-22

    13. [13]

      In the phase 3 VALOR trial (108 adults with SOD1-ALS), tofersen lowered SOD1 in spinal fluid and neurofilament light in plasma over 28 weeks but did not significantly improve the primary functional endpoint. confirmedas of 2022-09-22

    14. [14]

      The ATLAS confirmatory trial is testing tofersen in 158 presymptomatic adult SOD1 mutation carriers with elevated neurofilament; as of October 2026 it is active but not recruiting, with primary completion estimated for August 2027. confirmedas of 2026-10-11

    15. [15]

      Qalsody received an EU marketing authorisation under exceptional circumstances on 29 May 2024, with a requirement to provide long-term data and study people who do not yet have symptoms. confirmedas of 2024-06-14

    16. [16]

      The UC Davis neuroprosthesis reached 99.6 percent accuracy on a 50-word vocabulary on its first day of use and sustained 97.5 percent accuracy over 8.4 months, with the participant conversing at about 32 words per minute for more than 248 hours. confirmedas of 2024-08-15

    17. [17]

      ALS patient-advocate Brooke Eby died aged 37; the ALS Network announced her death on 1 October 2026. confirmedas of 2026-10-02

    18. [18]

      Eby founded ALStogether, an online Slack peer community for people with ALS and caregivers, which joined the ALS Network in 2026 and had more than 1,700 members as of March 2026. confirmedas of 2026-10-01

    19. [19]

      A 2012 Cochrane review of randomised trials (974 riluzole-treated and 503 placebo-treated patients) concluded riluzole is reasonably safe and probably prolongs median survival by about two to three months. confirmedas of 2012-03-14

    20. [20]

      In the Cochrane analysis, one-year survival was 58% with riluzole versus 49% with placebo, median survival rose from 11.8 to 14.8 months, and raised liver enzymes were more frequent with riluzole. confirmedas of 2012-03-14

    21. [21]

      In a 24-week Japanese phase 3 trial of 137 randomised patients with early-stage ALS chosen by criteria from an earlier post-hoc analysis, functional scores (ALSFRS-R) fell by 5.01 points with intravenous edaravone versus 7.50 with placebo. confirmedas of 2017-05-15

    22. [22]

      The edaravone trial authors said the drug showed efficacy in a small subset of people with ALS, after an earlier phase 3 study had found no significant difference in the full population. confirmedas of 2017-05-15

    23. [23]

      At 52 weeks in VALOR's open-label extension, functional scores fell by 6.0 points in participants who started tofersen early versus 9.5 in those who started 28 weeks later; neurologic serious adverse events occurred in 7% of tofersen recipients. confirmedas of 2022-09-22

    24. [24]

      In the UC Davis speech neuroprosthesis study, a 45-year-old man with ALS received four microelectrode arrays with 256 electrodes in his left ventral precentral gyrus. confirmedas of 2024-08-15

    25. [25]

      The ALS Network named Eby, whom it called one of the most visible ALS advocates, its 2026 Dean and Kathleen Rasmussen Advocate of the Year in March 2026 and honoured her in June 2026. confirmedas of 2026-10-01

    Revision history (1)
    1. Page created.

    Created Oct 11, 2026. Last reviewed by an editor on Oct 11, 2026. Next scheduled review: Dec 11, 2026.

    Cite this page

    "ALS research tracker: treatments, trials and milestones." ContentLora, updated Oct 11, 2026. https://contentlora.com/events/als-research-tracker

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