To find solutions and improve methods of therapy for patients suffering from type III spinal muscular atrophy (SMA-III) by way of a combination of conventional treatment (medicines, physiotherapy) and fetal stem cells (FSCs) transplantation using suspensions extracted from stem cells of fetal liver and brain of human embryos.

Our study included 7 children suffering from SMA-III, including 5 boys and 2 girls, aged from 3 to 12 years who were allocated into the main group (MG). SMA children in the MG were administered fetal stem cell preparations, mainly suspensions which contained fetal cells of human liver and fetal brain along with standard treatment (treatment by use of medicines if required and physiotherapy). The control group (CG) included 6 children – 3 boys and 3 girls aged from 3.5 to 13 years.

The authors proved effectiveness and safety of treatment using FSCs for patients with SMA-III. Improvement of forced vital capacity of lungs and forced expiratory volume per 1 second was characteristic for the patients of the MG, starting from the period of observation at 6 months after FSCs transplantation. In patients from the CG, such values revealed characteristic improvement at 12 months after FSCs treatment. Significant reductions of alanina aminotransferase, aspartate aminotransferase, creatine phosphokinase, and lactate dehydrogenase ranges have been recorded in the patients of the MG at 6 months after treatment with FSCs. Such values show a significant reduction at 12 months after treatment.

FSCs use in complex treatment of patients with SMA results in stable disease compensation, improvement of laboratory results, and improved ranges of forced vital capacity and forced expiratory volume in 1 second.

To study the effect of fetal stem cell (FSC) therapy on Grade Ⅰ and Ⅱ respiratory failure in patients with amyotrophic lateral sclerosis (ALS) and muscular dystrophy (MD).

A comparative study was conducted on 41 patients with Grade Ⅰ or Ⅱ respiratory failure (RF) resulting from ALS or MD. The patients were divided into 4 groups according to the underlying disease and the degree of RF. Patients underwent combined treatment, including the experimental application of FSC therapy, and were examined before FSC treatment, and 6 months and 12 months after treatment.

FSC treatment improved both subjective and objective breathing parameters as early as 6 months post-treatment. A significant increase in the forced vital capacity (FVC) and forced expiratory volume in 1 second (FEV₁) was reported by all patients with Grade Ⅰ RF linked to ALS and MD compared to baseline. Patient respiratory improvement was maintained over the next 6 months. Grade Ⅱ RF patients with MD reported a significant improvement in FVC 12 months after treatment.

Evidence for respiratory improvement was observed as early as 6 months in all patients after combined treatment including FSC therapy, and this was maintained for a further 6 months after therapy. In MD patients with Grade Ⅱ RF, treatment resulted in a significant FVC and FEV₁ increase within 6 months and downgrading to Grade Ⅰ RF within a year after FSC treatment.