4.5 Article

Cellular transplants in amyotrophic lateral sclerosis patients: an observational study

Journal

CYTOTHERAPY
Volume 12, Issue 5, Pages 669-677

Publisher

ELSEVIER SCI LTD
DOI: 10.3109/14653241003774037

Keywords

amyotrophic lateral sclerosis; CD133(+); CD34(+); cytotherapy; motor neuron; stem cell

Funding

  1. Spanish Fondo de Investigacion Sanitarias [PI 10/01970]

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Background aims. Cytotherapy is a promising option for neurodegenerative disease treatment. Because of the fatal prognosis and imperative need for effective treatment, amyotrophic lateral sclerosis (ALS) patients request this therapy before its effectiveness has been verified. The increase in clinics offering cytotherapies but providing little scientific information has prompted considerable medical tourism. We present an observational study of Spanish ALS patients receiving cytotherapy, analyzing the experiences arising from the treatment (TX) and considering two progression markers, FVC and ALSFRS-R. Methods. Twelve ALS patients with a mean age of 48.6 years (SD 12.8) received cytotherapy 26.9 months (SD 15.8) after clinical onset. ALSFRS-R and PVC at TX were 32.3 (SD 6.8) and 63.4% (SD 15.3), respectively. TX involved transplants of olfactory ensheathing cells in three patients, and autologous mesenchymal stromal cells in the remainder. Results. One patient died 33 months post-TX after surviving for 49 months. Five required mechanical non-invasive home ventilation 7.4 months post-TX. Two required invasive ventilation 13 months post-TX. Five patients needed gastrostomy feeding 23.3 months post-TX. Survival between clinical onset and the study end date was 50 months (SD 17.2). No significant adverse events or changes in the decline of PVC and ALSFRS-R compared with the disease's natural history were observed. Conclusions. Our observations suggest that these therapies do not halt the course of the disease. Cytotherapy cannot yet be considered a curative treatment for ALS.

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