Trials & Treatments

Computer modelling identifies three thymoquinone-like compounds for possible ALS drug development

Mainly of interest to researchers studying Keap1-Nrf2 drug discovery and computational ALS pharmacology.

ALS Nexus 2026: Experimental therapy shows early promise in SOD1-ALS

Relevant to people following emerging treatment options for SOD1-ALS

JP1 improved movement and survival in ALS model mice by targeting oxidative stress and autophagy

Provides mechanistic and efficacy data on JP1, Nrf2 signaling, autophagy and ALS mouse models.

Tiziana Life Sciences to present Phase 2a BANYAN trial data at ALS Nexus 2026

Relevant to researchers following clinical data on investigational ALS therapies.

Review outlines why ALS trials fail and how more personalised treatment could help

Relevant to therapeutic mechanisms, translational barriers and innovative ALS trial methodology.

New laboratory methods measure a proposed ALS drug combination

Mainly of interest to researchers studying analytical validation and ALS combination-therapy development.

Stem cell therapy may slow ALS functional decline, researchers say

Relevant to people considering emerging therapies that may affect functional decline

A review outlines why ALS may need combination treatments

Mainly of interest to researchers studying multi-target ALS pharmacology

Mesenchymal stem cell treatments appear generally safe, but benefits in ALS remain uncertain

Mainly of interest to researchers studying MSC protocols, biomarkers and trial design.

A Canterbury study found inconsistent riluzole prescribing and monitoring in ALS

Relevant to clinicians reviewing riluzole prescribing, monitoring and guideline adherence.

A severe hypersensitivity reaction was reported during tofersen therapy in ALS

Important for clinicians assessing tofersen hypersensitivity and treatment safety

Phase 4 trial of Tofersen closes recruitment

ALS therapy moves closer to Health Canada filing planned for December

Relevant to people awaiting potential access to a new ALS therapy

ALS Untangled does not recommend ivermectin for ALS

Relevant to clinicians advising on ivermectin evidence, safety and lack of ALS trials

A review found preliminary signs that stem cell therapy may slow ALS progression

Mainly of interest to researchers comparing stem-cell trial designs, doses, routes and evidence gaps.

A study mapped SOD1 variants and tofersen access in six Central Eastern European countries

Relevant to clinicians assessing SOD1 variants, genetic testing, counseling, and tofersen access.

Phase 2 trial of Digoxin completes

Review highlights evidence and trial-design gaps for CTx1000, a TDP-43-targeting ALS therapy

Relevant to researchers studying TDP-43 degradation, target engagement and ALS trial methodology.

An antibody fragment reduced ALS-related damage in cells and mice

Directly informs PC-OxPL mechanisms, TDP-43 pathology and AAV-scFv therapeutic development.

Developer to seek approval in Canada for ALS treatment aiding survival

Relevant to people monitoring potential new treatments that may affect survival.

Tofersen was linked to slower decline in one person with ALS and an uncertain SOD1 variant

Relevant to clinicians assessing tofersen for SOD1 variants of uncertain significance and monitoring biomarkers.

Phase 2 trial of 50 mg Usnoflast opens to recruitment

A French real-world study links tofersen with slower decline in SOD1-ALS

Relevant to clinicians evaluating real-world tofersen effectiveness, biomarkers, survival and study limitations.

Borsantrazole extended survival in a mouse model of ALS

Mainly of interest to researchers studying oxidative-stress therapeutics, proteomics and ALS models.

Stem cell therapy for ALS still available to patients in Asian nation

Relevant to people considering access to an unproven stem cell treatment option.

Trial of plasmapheresis in ALS/MND registered (PALADIN2)

Platelet factor 4 improved disease measures in SOD1 ALS mice

Mainly of interest to researchers studying PF4, OPTN-dependent autophagy and SOD1 pathology

Therapeutic plasma exchange was not linked to significant functional improvement across a mixed group that included ALS

Provides retrospective data and a rationale for prospective study of plasma exchange in ALS and related disorders.

Early ALS trial finds therapy well tolerated and lowers disease markers

Relevant to clinicians evaluating early safety and biomarker evidence for an investigational therapy.

Phase 1 trial of TCD601 terminated: “The Sponsor has decided to terminate study early due to significant enrolment challenge…”

Real-world study sheds new light on safety of approved ALS drug

Relevant to clinicians assessing real-world drug safety

An editorial warns that consumer AI can mislead people interpreting ALS test results

Relevant to clinicians managing AI-mediated self-diagnoses and diagnostic communication

Respiratory muscle training may modestly improve some breathing measures in ALS

Relevant to clinicians evaluating evidence for respiratory muscle training in ALS care.

Heavy metals and ALS: what the evidence does (and doesn't) mean for you

Relevant to clinicians counselling patients about metal exposure evidence and chelation.

Macrophage inclusions were more common in the cerebrospinal fluid of patients receiving tofersen than nusinersen

Relevant to clinicians interpreting macrophage inclusions, inflammation and treatment response in CSF.

A publication describes TREM2-activating compounds proposed for ALS treatment

Relevant to researchers studying TREM2 agonists and neurodegenerative disease mechanisms.

Oral ALS treatment lowers toxic protein levels, extends survival

Relevant to people considering a treatment that may affect toxic protein levels and survival

New ALS treatment enters human testing in global program

Relevant to people tracking emerging treatment options entering clinical testing

Phase 1/2 trial of TRCN-1023 opens to recruitment

Amylyx advancing ALS treatment in trial after early safety data is positive

Relevant to people following emerging ALS treatment options

New European trial testing safety of ALS therapy targeting fat metabolism

Relevant to people following emerging ALS treatment options and trial participation.

Nura raises $74M to support ALS trial, new nerve-protecting therapy

Relevant to people following development of a potential nerve-protecting ALS treatment

Phase 1/2 trial of ALN-SOD opens recruiting sites in Germany, Poland

REM-suppressing antidepressant prescriptions were associated with modestly longer survival in an ALS database study

Mainly of interest to researchers studying REM sleep, antidepressants and ALS survival confounding

A case report describes foslevodopa/foscarbidopa infusion in POLG-associated Parkinson’s disease and ALS overlap

Mainly of interest to researchers studying POLG variants and ALS-parkinsonism overlap

Results posted for phase 1 trial of Autologous T-regulatory lymphocytes

Masitinib linked to higher 5-year survival rate in ALS trial

Relevant to clinicians assessing long-term evidence for masitinib in ALS.

A U.S. center reports delivering tofersen to 11 people with SOD1 ALS before approval

Relevant to clinicians implementing tofersen expanded access and assessing operational requirements

A fatty acid amide hydrolase inhibitor improved disease-related measures in ALS models

Mainly of interest to researchers studying endocannabinoid metabolism, microglia and motor-neuron degeneration.

FDA grants orphan status to new ALS drug candidate PAS-004

Relevant to people following emerging treatment options for ALS.

NUZ-001 arm in HEALEY ALS trial expands to 240 participants

Relevant to people following recruitment and access to an investigational ALS treatment.

Netherlands approves trial of brain implant for ALS communication

Mainly of interest to researchers following ALS brain-computer interface trials

Triumeq briefly improved motor function in a TDP-43 ALS mouse model

Relevant to researchers studying TDP-43 pathology, endogenous retroviruses and repurposed antiretroviral mechanisms.

Outsee wins award to support AI search for ALS treatments

Relevant to researchers interested in AI-enabled therapeutic discovery

Cholesterol-lowering statins don’t affect ALS progression: Study

Relevant to clinicians assessing statin use and evidence about ALS progression

Neurizon gets OK to test liquid form of ALS medication in Australia

Relevant to people seeking new or more accessible ALS treatment options

RNA therapy shows promise for addressing key driver of ALS

Mainly of interest to researchers studying RNA therapeutics and ALS drivers.

ALS and the gut microbiome, part two: What can we actually do with this science?

Relevant to clinicians counselling patients about evidence and risks of gut-focused interventions

FDA puts injection therapy COYA 302 on fast track for treating ALS

Relevant to clinicians monitoring regulatory progress for an investigational ALS therapy.

New AI matchmaker pairs ALS patients with clinical trials in seconds

Relevant to people seeking suitable clinical trial opportunities

ALS treatment tazbentetol gets FDA fast track status

Relevant to people monitoring emerging ALS treatment options.

Acurastem wins $7.5M to advance ALS treatment to clinical trials

Relevant to people following development of a potential new ALS treatment

Clene plans CNM-Au8 filing for accelerated approval in ALS

Relevant to people tracking potential new ALS treatment options

Dazucorilant shows significant survival benefit in Phase 2 ALS trial

Relevant to people following potential treatments that may extend survival

Choline in real life: food, testing, supplements, and the questions that actually matter

Mainly of interest to researchers following choline metabolism, genetic modifiers and biomarker limitations.

A phase 2b trial found PrimeC was well tolerated and showed longer-term signals of benefit in ALS

Relevant to clinicians assessing PrimeC safety, efficacy signals, and confirmatory-trial implications

Developer gets OK to expand first-in-human trial of ALS therapy to Europe

Relevant to people following access to an experimental ALS therapy

A trial will compare outpatient and hospital initiation of ventilation for people with ALS

Relevant to clinicians evaluating outpatient NIV initiation, adherence and telemonitoring.

A screen of ALS patient-derived motor neurons identified compounds that raise neurofilament light chain levels

Mainly of interest to researchers studying iPSC disease models, neurofilament biology and high-throughput screening

ALS Untangled says clenbuterol needs more study before it can be recommended for ALS

Relevant to clinicians assessing limited efficacy evidence, dosing and adverse cardiac effects

ALS, carbs and ketones: what happens when the science points in two directions at once

Relevant to counselling patients about nutritional evidence and its observational limitations.

ALSUntangled does not support N-acetylcysteine for slowing ALS progression

Relevant to clinicians counselling patients about NAC efficacy and possible bulbar risks

A review suggests red and near-infrared light could be studied as an ALS treatment

Mainly of interest to researchers studying multimodal neuroprotection and mitochondrial mechanisms

GPNMB tracked PIKfyve inhibitor activity in laboratory studies and healthy volunteers

Mainly of interest to researchers studying PIKfyve inhibition, GPNMB biomarkers, and ALS drug development.

MitoQ and coenzyme Q10: what the evidence shows in ALS and MND

Relevant to clinicians counselling patients about mitochondrial therapies and evidence quality

Lion’s Mane is not proven to treat ALS/MND

Relevant to clinicians counselling patients about evidence, product variability and supplement safety.

Urolithin A and ALS: Pulling Mitochondrial Levers

Relevant to researchers tracking mitophagy mechanisms and translational evidence.

Creatine did not slow ALS progression in clinical trials, despite its role in cell energy

Relevant to clinicians reviewing creatine evidence, outcomes and adverse effects in ALS.

Most U.S. neuromuscular therapy policies had restrictions beyond FDA labels

Relevant to clinicians managing prior authorization, reauthorization and treatment continuity.

Developing a new gene therapy

Relevant to researchers studying UNC13A, TDP-43 and antisense oligonucleotide development

Exploring new treatments through clinical trials

Relevant to people considering emerging clinical-trial treatment options.

Finding new treatments for MND

Mainly of interest to researchers following TDP-43, stem-cell, machine-learning and trial-design research.

The ketogenic diet and neurodegeneration: lifeline, landmine… or something in between?

Relevant to clinicians counselling patients about evidence and safety of ketogenic diets.

The Dark Side of Supplements and Off-Label Treatments

Relevant to people weighing supplements and off-label treatments safely

ALS Untangled does not support pyridostigmine to slow ALS progression

Relevant to clinicians advising on pyridostigmine, evidence gaps and risks.

A trial is testing intermittent low-oxygen breathing with istradefylline in ALS

Mainly of interest to researchers studying respiratory neuroplasticity and purinergic signaling

High-dose vitamin B12 slowed functional decline in an early ALS trial

Relevant to people considering high-dose methylcobalamin and its evidence in early ALS.

Dr. Bedlack and the 64 ALS Reversals

Mainly of interest to researchers studying ALS heterogeneity, protective biology and replication trials.

Podcast explores systems biology and nutrition in neurodegenerative disease

Relevant to researchers interested in systems biology and nutrition-related mechanisms in neurodegeneration.

Red light therapy and ALS: hype, hope or both

Relevant to people considering red-light therapy or other complementary ALS treatments

How a gym supplement ended up in my ALS research notes

Mainly of interest to researchers following IGFBP7, IGF-1 and HMB hypothesis development

Cannabis, ALS, and the fight for relief

Relevant to people considering cannabis for pain, spasticity, anxiety, or comfort.

ALS Untangled says evidence is insufficient to support regular ISRIB use in ALS

Relevant to clinicians assessing evidence, tolerability, bioavailability, and lack of human trials.

How can biomarkers be used in clinical trials in MND?

Mainly of interest to researchers studying biomarkers and trial outcome measures.

What are clinical trials?

Relevant to people wanting to understand how potential treatments are tested and when they may become available

Evidence remains insufficient to show alpha lipoic acid slows ALS progression

Relevant to clinicians counselling patients about evidence, safety and efficacy of alpha lipoic acid

MIROCALS: breaking down the trial results

Relevant to clinicians interpreting MIROCALS efficacy, subgroup findings and safety signals.

A review describes possible protective effects of ginseng compounds in ALS models

Mainly of interest to researchers studying ginsenosides, oxidative stress, and neuroprotection in ALS models.

ALS Untangled does not endorse zinc supplementation as an ALS treatment

Relevant to clinicians counselling patients about zinc evidence, dosing and copper-related risks

International clinical trial updates to look out for in 2025

Relevant to clinicians monitoring upcoming efficacy and safety evidence

UK clinical trial updates to look out for in 2025

Relevant to clinicians monitoring trial results that may influence MND treatment discussions

ALS Untangled finds no evidence that psilocybin slows ALS progression

Relevant to clinicians counselling patients about evidence and safety of psilocybin

Genomic analysis identifies acamprosate as a candidate treatment for C9orf72-related ALS and FTD

Mainly of interest to researchers studying genomic drug repurposing and C9orf72 disease biology.

ALS Untangled does not endorse the Wahls Protocol for slowing ALS

Relevant to clinicians evaluating evidence and nutritional risks of an unproven ALS intervention

Advancing Psychological Support for People with MND: The COMMEND Study and iDeliver

Relevant to clinicians evaluating evidence for integrating Acceptance and Commitment Therapy into MND care.

ALS Untangled finds no evidence that PoNS slows ALS progression

Relevant to clinicians evaluating an unproven neuromodulation device for ALS.

A review found mixed effects of neuromodulation in ALS studies

Mainly of interest to researchers comparing neuromodulation methods, outcomes and study limitations.

Risk of death – clinical trial statistics explained

Relevant to critically assessing mortality outcomes and meaningful patient benefit.

ALS Untangled says ashwagandha merits clinical testing in ALS

Relevant to researchers evaluating preclinical mechanisms and the ongoing ALS trial.

7 MND clinical trial updates to look out for in 2024

Relevant to clinicians monitoring trial evidence that may change treatment practice.

ALS Untangled finds no evidence to support lion’s mane for ALS

Relevant to clinicians advising on evidence and safety of Lion’s Mane use.

ALS Untangled does not endorse insulin treatment for ALS

Relevant to clinicians counselling patients about insulin safety and evidence

ALS Untangled says Nuedexta may help bulbar function in ALS

Relevant to clinicians assessing Nuedexta efficacy, safety, monitoring and patient selection.

ALS Untangled does not endorse caffeine as an ALS treatment

Relevant to clinicians counselling patients about caffeine safety and evidence.

Astaxanthin has not been tested in clinical trials for ALS

Relevant to clinicians counselling patients about astaxanthin's limited ALS evidence.

ALS Untangled advises against rituximab for ALS treatment

Relevant to counselling patients about rituximab evidence and safety

Expert review finds no evidence that corticosteroids slow ALS progression

Relevant to clinicians evaluating evidence for corticosteroid use in ALS

ALS Untangled does not support widespread use of antimycobacterial antibiotics for ALS

Relevant to clinicians assessing atypical ALS features and avoiding potentially toxic unsupported treatment

ALS Untangled supports testing butyrates in people with ALS

Relevant to clinicians evaluating evidence for sodium phenylbutyrate and TUDCA

A paper examines how deaths should be handled in ALS clinical-trial results

Relevant to estimand choices and methods for handling death in randomized ALS trials.

Researchers proposed a patient-preference endpoint for ALS trials

Relevant to researchers developing and validating preference-weighted composite endpoints for ALS trials

ALSUntangled does not advise ketogenic diets as an ALS treatment

Relevant to clinicians counseling patients about limited evidence and risks

ALS Untangled advises against using vitamin C to treat ALS

Relevant to counselling patients about evidence and vitamin C use.

ALS Untangled finds no proven benefit for melatonin in ALS

Relevant to clinicians evaluating limited evidence, dosing uncertainty and treatment recommendations.

Light therapy has not been convincingly shown to help people with ALS

Relevant to clinicians advising on evidence, safety and uncertainty around light therapy

ALS Untangled says tamoxifen is not yet supported as an ALS treatment

Relevant to clinicians evaluating limited human evidence and treatment safety.

Expert review advises against azathioprine for ALS treatment

Relevant to clinicians counseling patients about evidence, monitoring and serious azathioprine risks

ALS Untangled reviewed L-carnitine supplements for ALS

Relevant to people considering carnitine supplements and treatment options.

ALS Untangled does not recommend LEAP2BFIT for ALS

Relevant to people considering an unproven supplement for ALS.

ALS Untangled does not recommend vitamin E to treat ALS

Relevant to counseling patients about evidence and lack of clinical efficacy

ALS Untangled supports further study of vinpocetine

Relevant to clinicians assessing evidence and safety of vinpocetine in ALS

ALS Untangled advises against antifungal drugs for ALS

Relevant to clinicians advising against unsupported antifungal use and potential medication harm.

Antibodies reduced disease features in mice with C9orf72-related ALS and FTD

Mainly of interest to researchers studying RAN proteins, antibody delivery and C9orf72 disease models.

ALS Untangled does not advise glutathione supplements for ALS

Relevant to people evaluating glutathione or cysteine-containing supplements for ALS.

ALS Untangled does not recommend using RCH4

Relevant to clinicians advising patients about unverified efficacy, safety and regulatory oversight.

ALS Untangled does not recommend resveratrol as an ALS treatment

Relevant to clinicians counselling patients about the limited evidence and safety of resveratrol.

ALS Untangled does not recommend off-label perampanel for ALS

Relevant to clinicians evaluating off-label perampanel against limited ALS evidence and serious safety concerns.

An expanded access program is underway for RT001 in some people with ALS

Relevant to people considering access to RT001 through an expanded access program.

ALS Untangled does not recommend antiretrovirals as an ALS treatment

Relevant to clinicians assessing evidence and counselling patients about antiretroviral use.

ALS Untangled reviewed curcumin as a possible ALS treatment

Relevant to clinicians evaluating limited evidence for curcumin in ALS

ALS Untangled does not recommend CuATSM for ALS outside clinical trials

Relevant to clinicians advising on CuATSM evidence, dosing uncertainty and safety.

ALSUntangled does not recommend Basis for treating ALS

Relevant to clinicians advising patients about the limited evidence and safety of Basis.

ALS Untangled found no evidence that the Eric is Winning regimen changed ALS progression

Relevant to clinicians counselling patients about evidence, risks and unvalidated regimens.

ALS Untangled does not endorse ayahuasca for ALS

Relevant to people considering ayahuasca or other unproven ALS treatments and their safety risks

ALS Untangled does not endorse Acuscope for slowing ALS progression

Relevant to people evaluating Acuscope as an unproven treatment option

ALS Untangled reviewed L-serine as a possible treatment for some people with ALS

Relevant to clinicians assessing evidence for an unproven ALS therapy.

ALSUntangled does not recommend hyperbaric oxygen therapy for ALS

Relevant to people considering hyperbaric oxygen as an unproven ALS treatment.

ALSUntangled does not recommend Accilion for ALS

Relevant to clinicians assessing evidence and treatment recommendations for Accilion

ALSUntangled found no independently verifiable evidence that GM604 is effective or safe

Relevant to counseling patients about evidence, safety, and expanded access.

ALSUntangled says Precision Stem Cell protocols need clearer evidence and oversight

Relevant to clinicians advising patients about MSC protocols, safety monitoring and evidence quality.

ALS Untangled does not recommend Endotherapia for ALS

Relevant to people considering Endotherapia as an unproven ALS treatment

ALS Untangled found weak evidence linking gluten-related disorders with ALS

Relevant to clinicians assessing possible gluten-related disease and dietary management in ALS.

ALS Untangled says Protandim warrants further study despite limited evidence

Relevant to clinicians assessing the limited evidence, dosing uncertainty, and conflicts of interest around Protandim.

Methylcobalamin did not show an overall benefit in the strongest clinical trial reviewed

Relevant to clinicians evaluating methylcobalamin evidence, possible responder subgroups and dosing safety.

ALS Untangled says MitoQ merits a small pilot trial

Mainly of interest to researchers evaluating MitoQ’s ALS models and need for controlled clinical study.

ALS Untangled says acupuncture needs better studies before testing in a phase II trial

Relevant to clinicians assessing whether acupuncture has sufficient evidence for ALS care.

ALS Untangled says evidence is insufficient to recommend lunasin for ALS

Relevant to clinicians assessing lunasin's limited evidence, safety and need for controlled study.

ALSUntangled does not recommend ursodiol for ALS at 300 mg twice daily

Relevant to clinicians advising on ursodiol safety and lack of demonstrated benefit

Vitamin D deficiency screening is considered reasonable in people with ALS

Relevant to clinicians assessing vitamin D deficiency while noting uncertain ALS-specific benefits.

ALSUntangled does not support using Rife machines for ALS

Relevant to clinicians advising patients about an unsupported complementary treatment.

ALSUntangled advises against off-label propofol use in ALS

Relevant to clinicians assessing evidence against off-label propofol use in ALS

ALSUntangled does not recommend fecal microbiota transplantation for ALS

Relevant to researchers investigating the gut microbiome and ALS mechanisms.

ALSUntangled does not recommend the Deanna Protocol for amyotrophic lateral sclerosis

Relevant to people considering the Deanna Protocol as an unproven treatment option.

ALSUntangled supports further study of sodium chlorite formulations but warns against oral use

Relevant to clinicians advising on NP001, WF10 and oral sodium chlorite risks

ALSUntangled does not recommend apoaequorin for ALS

Relevant to clinicians evaluating evidence and adverse-event concerns around apoaequorin.

ALSUntangled supports further study of cannabis and cannabinoids in ALS

Relevant to clinicians evaluating the limited evidence and need for controlled cannabinoid studies in ALS.

ALSUntangled supports further study of coconut oil in ALS

Relevant to people considering coconut oil as a complementary treatment option.

ALS Untangled does not support using Mototab for motor neuron disease

Relevant to decisions about avoiding an unsupported and potentially unsafe product

ALSUntangled does not support bee venom use outside a study

Relevant to people considering bee venom or other complementary treatments for ALS

ALSUntangled does not support Dean Kraft’s energy healing for ALS

Relevant to clinicians counselling patients about evidence and alternative therapies

ALSUntangled found little objective evidence to support NuTech Mediworld’s stem cell treatment

Relevant to people considering an unproven and potentially costly stem-cell treatment

ALSUntangled does not support luteolin supplements for ALS

Relevant to people considering luteolin supplements and weighing uncertain benefit against possible risks

ALS Untangled does not support spirulina use for ALS

Relevant to people considering spirulina or complementary treatments for ALS

ALS Untangled does not recommend low-dose naltrexone for ALS

Relevant to clinicians assessing evidence and safety of an off-label treatment.

ALS Untangled calls for trials of Aimspro before further use

Relevant to clinicians assessing evidence for Aimspro and advising against unsupported use.

ALSUntangled recommends avoiding the Stowe/Morales ALS Protocol

Relevant to people weighing an expensive and potentially harmful ALS treatment protocol

ALS Untangled questioned the evidence behind the XCell-Center protocol

Relevant to clinicians assessing weak pilot evidence and advising patients about treatment safety

ALS Untangled found insufficient evidence to support stem cell transplants at a Mexican clinic

Relevant to clinicians assessing safety and efficacy evidence for stem-cell interventions

ALSUntangled begins investigating treatment requests from people with ALS

Relevant to clinicians evaluating evidence for Iplex in ALS