Amyotrophic lateral sclerosis (ALS) is a progressive disease that destroys the nerve cells controlling voluntary muscle movement, leading to complete paralysis while typically sparing cognition. It remains one of the most devastating neurological conditions and one where even modest slowing of progression is meaningful.
What's actually going on in research
Tofersen, an antisense oligonucleotide targeting SOD1, showed it could slow progression in patients with SOD1-ALS and represents the first gene-targeted therapy in this disease. Multiple RNA and gene therapies are now in trials for the most common familial forms including C9orf72, FUS, and TDP-43 mutations. Neuroprotective drugs targeting neuroinflammation and protein aggregation — the hallmarks of ALS pathology — continue to enter early trials.
Antisense oligonucleotides
Tofersen, delivered by spinal injection, silences the mutant SOD1 gene in hereditary ALS. Similar RNA-targeting drugs for C9orf72 and FUS-ALS are in early-phase trials.
Neuroinflammation targeting
Drugs targeting microglia activation and neuroinflammatory cascades are in trials based on evidence that immune cell dysfunction accelerates motor neuron loss in ALS.
TDP-43 protein clearance
Strategies to prevent or clear the abnormal TDP-43 protein aggregates — present in 97% of ALS cases — are a central research focus, including approaches using autophagy enhancement.
What to know before you search
Eligibility depends on ALS subtype and genetic mutation (for gene-targeted trials), disease duration, respiratory function (FVC percentage), and rate of progression.
What types of trials are currently open
- Gene-targeted therapy trials — Testing antisense oligonucleotides and RNA interference for SOD1, C9orf72, and other familial ALS mutations.
- Neuroprotective drug trials — Evaluating anti-neuroinflammatory drugs and protein-targeting agents to slow motor neuron loss.
- Cell therapy trials — Testing stem cell and immune cell infusions for neuroprotective effects in ALS.
- Device and assistive technology trials — Evaluating brain-computer interfaces, communication devices, and ventilatory support strategies.
- Biomarker trials — Validating neurofilament light chain and other markers to track disease progression and treatment response.
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