No stem-cell therapy for ALS holds FDA approval. Investigational trials in the USA operate under IND protocols; major trials enrol at ALS centres of excellence (Cleveland Clinic, Massachusetts General, Johns Hopkins, UC San Diego). FDA expanded-access pathways exist for eligible patients with rapidly progressive disease. European trials are registered with national authorities; compassionate-use access is variable.
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.
Съм ли кандидат? → · ALS (Motor Neurone Disease): full overview → · Цена ALS (Странична амиотрофна склероза) → · Cost →
Медицински преглед от редакционния екип на StemCellAtlas с Kiian Nadiia, MD, PhD (Paediatric Neurologist · Medical Director, CSM Clinic Network · 12+ yrs in Autism Spectrum Disorders) на партньорската клиника Stem Plus (София), спрямо ISSCR, FDA и EMA указания. Educational information, not medical advice; figures indicative.
GMP-сертифицирана регенеративна медицина в сърцето на ЕС — от 3 000–8 000 €, част от цените в САЩ или Германия. Персонализирани протоколи за пациенти от над 50 държави.
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