Stem cell transplantation in amyotrophic lateral sclerosis patients: methodological approach, safety, and feasibility.

Amyotrophic lateral sclerosis is characterized by the selective death of motor neurons. Stem cells have been proposed as a potential therapeutic strategy. The safety of stem cell transplantation into the frontal motor cortex to improve upper motor neuron function is described. Sixty-seven patients with definite amyotrophic lateral sclerosis were included. After giving their informed consent, the patients underwent magnetic resonance imaging, functional rating, pulmonary function test, and laboratory tests. Their bone marrow was stimulated with daily filgrastim (300 µg) given subcutaneously for 3 days. Peripheral blood mononuclear cells were obtained by leukapheresis. Isolated CD133(+) stem cells were suspended in 300 µl of the patient's cerebrospinal fluid and implanted into the motor cortex. Adverse events were recorded at each step of the procedure and were classified according to the Common Terminology Criteria for Adverse Events v3.0. The survival at 1 year was 90% after transplantation. with a mean long-term survival rate of 40.17 months from diagnosis. The most common adverse events were in grades I-II and involved transient skin pain (19.5% of patients) attributed to the insertion of the Mahurkar catheter into the subclavian vein, minor scalp pain (15.9%), and headache (12.2%) from the surgical procedure. Several patients (1.5 - 4.5%) reported diverse grade I adverse events. There were two deaths, one considered to be associated with the procedure (1.5%) and the other associated with the disease. Autologous stem cell transplantation into the frontal motor cortex is safe and tolerated well by patients. Further controlled studies are required to define the efficacy of this procedure.

Survival and clinical features in Hispanic amyotrophic lateral sclerosis patients.

The demography, survival, and motor phenotypes of amyotrophic lateral sclerosis (ALS) patients have been rarely described in Hispanic countries. The clinical characteristics and survival of a series of Mexican ALS patients are described. Mexican patients with definite ALS were included in a five-year retrospective longitudinal study. Their demographic and clinical features, cumulative survival rates, and independent predictive factors for survival were analysed. Sixty-one definite ALS patients were included. The median follow-up period was 35 months (range 12-108 months). Males were predominant (1.8: 1), the mean age at onset was 47.5 ± 10.5 years, and the median interval from onset to diagnosis was 12 months. Spinal onset occurred in 66% of patients. Upper motor neuron phenotype was predominant in 53% of patients. The overall mean survival from onset was 68.6 months, and from diagnosis was 57.8 months. Longer survival was determined in patients aged ≤ 40 years (54.7 months) compared with other age groups (p = 0.006). In conclusion, the clinical heterogeneity, male predominance, and survival rates in our sample are consistent with those of other studies. Patients in this series had a younger age at onset and a clear trend toward longer survival compared with those of other population studies.