Measurable functional plateau or decelerated decline becomes apparent at 3–6 months post-treatment. Peak benefit (longest preserved ALSFRS-R stability) typically extends through 9–15 months. Thereafter, disease progression typically resumes at baseline rate. Some centres recommend repeat intrathecal infusions every 6–12 months; optimal dosing and re-treatment intervals remain experimental. Long-term survival data beyond 2 years post-treatment are sparse.
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.
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Medizinisch geprüft vom Redaktionsteam von StemCellAtlas zusammen mit Kiian Nadiia, MD, PhD (Paediatric Neurologist · Medical Director, CSM Clinic Network · 12+ yrs in Autism Spectrum Disorders) der Partnerklinik Stem Plus (Sofia), gemäß ISSCR-, FDA- und EMA-Leitlinien. Educational information, not medical advice; figures indicative.
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