TY - JOUR
T1 - Cellular transplants in amyotrophic lateral sclerosis patients: An observational study
AU - Gamez, Josep
AU - Carmona, Francesc
AU - Raguer, Nuria
AU - Ferrer-Sancho, Jaume
AU - Martín-Henao, Gregorio A.
AU - Martí-Beltrán, Sergi
AU - Badia, Merce
AU - Gratacós, Margarita
AU - Rodriguez-Gónzalez, Esther
AU - Seoane, Jose Luis
AU - Pallero-Castillo, Mercedes
AU - Burgos, Rosa
AU - Puiggros, Carolina
AU - Pasarin, Alejandro
AU - Bori-Fortuny, Inmaculada
PY - 2010/1/1
Y1 - 2010/1/1
N2 - 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 FVC 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 FVC 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. © 2010 Informa Healthcare.
AB - 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 FVC 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 FVC 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. © 2010 Informa Healthcare.
KW - Amyotrophic lateral sclerosis
KW - Cytotherapy
KW - Motor neuron
KW - Stem cell
U2 - 10.3109/14653241003774037
DO - 10.3109/14653241003774037
M3 - Article
SN - 1465-3249
VL - 12
SP - 669
EP - 677
JO - Cytotherapy
JF - Cytotherapy
IS - 5
ER -