Functional decline slowing (≤2-point ALSFRS-R deterioration per month, preserved muscle strength in treated regions for ≥6 months) is reported in 35–50% of treated cohorts. Complete halting of disease progression is rare; survival extension beyond historical 2–3 year median remains unproven. Responders tend to be early-stage (symptom onset <18 months), younger (<60 years), and non-bulbar-onset phenotypes; rapidly progressive and bulbar-onset variants show lower response rates.
Clinical trial safety data across >500 ALS recipients shows no treatment-limiting adverse events from intrathecal or intravenous cell infusion. Efficacy signals emerge from small phase II cohorts: decline in ALSFRS-R (disease rating scale) is slowed by 30–50% over 12 months in 30–45% of treated patients versus untreated historical controls; some maintain muscle strength for 6–12 months longer than expected disease trajectory. Markers of neuroinflammation (CSF cytokine levels, microglial activation on PET imaging) decline in responder subgroups. No completed pivotal trial has demonstrated survival prolongation; two large multi-centre efficacy trials are recruiting.
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Medically reviewed by StemCellAtlas’s editorial team with Kiian Nadiia, MD, PhD (Paediatric Neurologist · Medical Director, CSM Clinic Network · 12+ yrs in Autism Spectrum Disorders) of partner clinic Stem Plus (Sofia), against ISSCR, FDA & EMA guidance. Educational information, not medical advice; figures indicative.
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