For patients with early-stage, rapidly progressive ALS (symptom onset <18 months, ALSFRS-R decline >1 point/month) and failed conventional therapies (riluzole, edaravone), participation in stem-cell trials offers potential functional-decline mitigation with acceptable risk. Advanced disease (wheelchair-bound, ventilator-dependent) shows lower response likelihood. Decision-making requires frank discussion of realistic outcomes (disease slowing rather than reversal), intrathecal procedural risks (infection, meningitis, cord trauma), and the experimental nature of the intervention. Trial transparency, centre ALS expertise, and patient commitment to long-term monitoring are essential.
Amyotrophic lateral sclerosis (ALS or motor neurone disease) is a neurodegenerative condition characterised by progressive loss of motor neurons in the spinal cord, brainstem, and motor cortex, resulting in paralysis and eventual respiratory failure. Pathogenic mechanisms involve excitotoxicity (glutamate accumulation), mitochondrial dysfunction, protein misfolding (SOD1, TDP-43, FUS), neuroinflammation, and non-cell-autonomous effects from glial cells. Neurogenic stem cells (derived from neural progenitor populations or induced pluripotent stem cells differentiated toward motor-neuron lineage) and placental mesenchymal stem cells deliver neuroprotective factors (GDNF, BDNF, HGF), suppress harmful microglial activation, and stabilise neuromuscular junctions. Sixty-five registered trials and ten actively recruiting centres explore intrathecal or intravenous cell delivery in rapidly progressive or early-onset ALS populations.
Sunt un candidat? → · ALS (Motor Neurone Disease): full overview → · Cost SLA (Scleroza Laterală Amiotrofică) → · Cost →
Revizuit medical de echipa editorială a StemCellAtlas cu Kiian Nadiia, MD, PhD (Paediatric Neurologist · Medical Director, CSM Clinic Network · 12+ yrs in Autism Spectrum Disorders) ale clinicii partenere Stem Plus (Sofia), în funcție de orientările ISSCR, FDA și EMA. Educational information, not medical advice; figures indicative.
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