Cellular transplants in amyotrophic lateral sclerosis patients: an observational study.
OBJECTIVE
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.
Gamez
Josep
J
Neurology Department, Hospital Universitari Vall d'Hebron, Autonomous University of Barcelona, Barcelona, Spain. 12784jgc@comb.es
Carmona
Francesc
F
Raguer
Nuria
N
Ferrer-Sancho
Jaume
J
Martín-Henao
Gregorio A
GA
Martí-Beltrán
Sergi
S
Badia
Merce
M
Gratacós
Margarita
M
Rodriguez-Gónzalez
Esther
E
Seoane
Jose Luis
JL
Pallero-Castillo
Mercedes
M
Burgos
Rosa
R
Puiggros
Carolina
C
Pasarin
Alejandro
A
Bori-Fortuny
Inmaculada
I
D000328:Adult; D000690:Amyotrophic Lateral Sclerosis / Q000473:pathology / Q000503:physiopathology / Q000628:therapy*; D018952:Antigens, CD34 / Q000096:biosynthesis; D001853:Bone Marrow / Q000473:pathology; D002478:Cells, Cultured; D002986:Clinical Trials as Topic; D018450:Disease Progression; D005260:Female; D033042:Fetal Research; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D057193:Medical Tourism; D045164:Mesenchymal Stem Cell Transplantation / Q000009:adverse effects / Q000941:ethics; D059630:Mesenchymal Stem Cells / Q000378:metabolism* / Q000473:pathology; D008875:Middle Aged; D009457:Neuroglia / Q000473:pathology / Q000637:transplantation; D009830:Olfactory Bulb / Q000473:pathology; D013030:Spain; D017154:Stromal Cells / Q000473:pathology / Q000637:transplantation
D018952:Antigens, CD34