Amyotrophic Lateral Sclerosis News and Research

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Australian-developed MND therapy advances to Phase 2 clinical trial

An Australian-developed therapy aimed at halting motor neurone disease progression and restoring function will advance to a national Phase 2 clinical trial.

9 Nov 2026

Genetic family mapping uncovers hidden links across ALS and FTD

Researchers at the Macquarie University Brain Institute and Clinic have helped accelerate the search for disease-causing genes by building new genetic 'families'.

3 Nov 2026

Scientists may have underestimated how much genes contribute to Alzheimer's risk

A large multi-ancestry genetic analysis identified 127 genomic loci associated with Alzheimer's disease, including 48 previously unreported loci, and implicated microglial and neuronal gene-expression patterns in disease susceptibility. The researchers estimated that common genetic variants account for 16% of Alzheimer's disease liability in European-ancestry populations, substantially higher than previous estimates, while demonstrating improved polygenic risk prediction that remained less reliable in non-European populations.

2 Nov 2026

Brain-computer interface enables unresponsive ICU patients to control sounds

Unresponsive ICU patients in a new Columbia study were able to raise and lower the frequency of a beeping tone with their minds, via noninvasive EEG electrodes that recorded and translated their brainwaves.

23 Oct 2026

Stem cell therapies may need new protein quality control standards

Current Good Manufacturing Practice (GMP) standards for cell therapies assess identity, viability, sterility, and genetic stability. For extracellular vesicle (EV) products, Minimal Information for Studies of Extracellular Vesicles (MISEV2023) guidance adds particle size, concentration, markers, morphology, contaminants, endotoxin, and assays.

22 Oct 2026

Alzheimer’s biomarkers may help predict disease progression in ALS

Amyotrophic lateral sclerosis (ALS) is a serious neurodegenerative disease in which the nerve cells that control the body's muscles break down.

20 Oct 2026

Scientists discover natural cellular defense mechanism against ALS progression

ALS is a disease where, in most cases, a protein forms aggregates in neurons, which leads to the rapid loss of motor control and death.

20 Oct 2026

Buck Institute and Georgetown lead effort mapping harmful protein changes before disease begins

A research "super team" co-led by theBuck Institute for Research on Aging and Georgetown University has received an award of up to $51.7 million from the Advanced Research Projects Agency for Health (ARPA-H) to predict how disease-causing proteins misfold before irreversible damage occurs and use those predictions to develop novel diagnostics and treatments.

12 Oct 2026

Advances in chemistry and delivery are taking oligonucleotide medicines beyond rare diseases

A review in Cell Reports Medicine examines how advances in oligonucleotide chemistry and delivery have established ASOs and RNA interference therapies as a class of genetic medicines, with 21 oligonucleotides approved at the time of writing. It also identifies extrahepatic delivery, safety, target validation, individualized treatment, and data quality as priorities as the field expands into common diseases and explores AI-assisted development.

24 Sep 2026

Experimental drug reverses multiple sclerosis damage in mouse model

A drug developed by a UVA Health scientist has not only stopped but reversed paralysis and vision loss in a mouse model of multiple sclerosis.

23 Sep 2026

Genetic system converts scar-forming cells into neurons to restore movement after spinal cord injury

What if cells that form scars after a spinal cord injury could become part of the repair process? Researchers have developed a genetic system that converts certain scar-forming cells into neurons, improving movement in mice and rats with spinal cord injuries.

23 Sep 2026

New discovery overturns a prevailing model of brain development

For centuries, scientists have thought of the brain as a single, unified organ. But new research led by Stanford Medicine reveals that what we call the brain is two distinct organs that evolved independently over hundreds of millions of years.

18 Sep 2026

One brain implant could give people with paralysis a more expressive way to communicate

Researchers showed that a single high-density ECoG implant could decode attempted speech and upper-body gestures in parallel in people with severe paralysis, allowing two participants to control a personalized full-body avatar. Training the decoders on both isolated and concurrent behaviors improved performance across contexts and reduced false activations, offering proof-of-concept evidence for more expressive multimodal BCIs.

16 Sep 2026

Study uncovers mechanism for reading extra-long neuronal genes

Neurons in humans and other mammals express many exceptionally long genes, ranging from more than 100 kb to over 2 Mb, that are important for forming synapses and neural circuits.

15 Sep 2026

AI platform uncovers new molecular targets for Alzheimer's disease

Groundbreaking study published in Alzheimer's & Dementia reveals the crucial links between the NAD⁺-mitophagy axis and neurodegeneration.

1 Sep 2026

Compound PF-04457845 slows motor decline in ALS mouse models

Amyotrophic lateral sclerosis (ALS) is a severe neurological disease that destroys nerve cells responsible for muscle movement.

28 Aug 2026

Shared protein pathology redefines treatment for multiple neurodegenerative diseases

On the surface, amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD) and limbic-predominant age-related TDP-43 encephalopathy (LATE) might seem like unrelated diseases.

24 Aug 2026

Disrupted brain-immune signaling may help drive neurodegeneration

A Cell perspective argues that neurodegeneration can emerge from disrupted communication between neural and immune networks, with peripheral immunity, microglia, and gene-regulatory programs all shaping disease processes. The authors propose that restoring immune homeostasis and recalibrating neuroimmune circuits could create new therapeutic and diagnostic opportunities, while emphasizing that many mechanisms remain unresolved in humans.

24 Aug 2026

A single blood test could offer a glimpse of disease risk years in advance

Machine learning-derived cellular aging clocks mapped more than 7,000 plasma proteins to over 40 cell types, revealing marked differences in how individual cell populations age. These blood-based signatures were associated with future risks of Alzheimer’s disease, ALS, lung cancer, type 2 diabetes, and mortality across follow-up extending to 15 years.

19 Aug 2026

New review urges proteostatic fitness standards for stem cell therapies

Stem cell therapies have expanded rapidly, with more than 115 clinical trials involving over 1,200 patients approved by late 2024. The U.S. Food and Drug Administration (FDA) recently approved Ryoncil (remestemcel‑L) for children with steroid‑refractory acute graft‑versus‑host disease, signaling a major shift toward routine clinical use.

11 Aug 2026