1) Objectives: Currently there is no treatment for Werdnig Hoffman patients. Stem cell transplantation is a strategy that is actively ongoing in research and development to protect neurons. Non-neural cells could be used to prevent the death of motor neurons. Some studies on hematopoietic stem cells, microglia cells and mesenchymal stem cells (MSC) by injection into Cerebro-Spinal Fluid (CSF) have considered effective in protecting motor neurons. So perhaps the only way for such patients depends on using stem cells that could deliver trophic factors to the dying cells.
2) Design: In this study we will discuss a non-randomize trial for the efficiency of MSCs on the possible phenotypic changes in Werdnig Hoffman patients.
3) Setting & Conduct:
Processing of adipose tissue and isolation of MSCs under GMP condition, Proliferation of MSCs, Conducting quality control tests, Performing electromyogram (EMG) before any injection, The first injection of MSCs via intrathecal route at the dose of 1 million cells, The second injection after 2 weeks at the dose of 3 million cells, The third injection after 4 weeks at the dose of 5 million cells, Sampling of CSF to detect the injected cells, EMG again after the last intervention;
4) Participant including major eligibility criteria:
Inclusion criteria: Age under 12 month; Weak muscle tone; Patient sit is not fully nerve guided; Existence of home senses; Normal brain function;
Exclusion criteria: Age up to 12 month; Other types of spinal muscular atrophies; Patient with sensory loss; Brain abnormalities;
5) Intervention:
Overall 10 patients will be enrolled, of which 5 patients will be injected with MSCs; Allogeneic adipose derived MSCs will be injected (3 times) intrathecally, Sampling from CSF to detect the injected cells;
6) Main outcome measures:
Improve in disease phenotype, Probable increased longevity.