Therapeutic Consequence of Allogeneic Mesenchymal Stem Cell for Duchenne Muscular Dystrophy- Case Study
Journal Title: International Journal of Pharmaceutical Science Invention - Year 2018, Vol 7, Issue 3
Abstract
Duchenne muscular dystrophy (DMD) is an inherited disorder caused by mutation in gene that prevents the translation of dystrophin protein, which leads to massive skeletal muscle wasting. Any form of muscular dystrophy is considered as an incurable disease yet there are occupational treatments assisting patients with locomotion strengthening and to further extend the lifespan. Thus, emerging treatment strategy is warranted to improve patients’ conditions from DMD. In the present study, we have evaluated the therapeutic outcome of human umbilical cord derived mesenchymal stem cell (hUC-MSC) in patients with DMD. In light of that, we have produced therapeutically safe (chromosomal stability, stable Immunopheotyping and mesodermal lineage differentiation ability) hUC-MSCs in our GMP compliance laboratory. Patients (24 and 27 years old) were administrated (intravenous [50X106 ] + intramuscular [50X106 ]) one single dose of hUC-MSC and followed up for 12 months. Therapeutic efficacy was measured as based on the level of creatinine phosphokinase (CK) in plasma. Both patients were highly responsive to hUC-MSC and the CK level was significantly dropped from 3 rdmonth of post transplantaton. After 12 months of post transplantation the CK level was slightly increased in both patients as compared to 6 th months. However, the level of CK level was still significantly lower than basal level. In conclusion, one single dose of hUC-MSCs treatment may not be sufficient to give better clinical outcome and also these results suggested that multi-dose of hUC-MSCs may give long term beneficial effects in patient with DMD.
Authors and Affiliations
Sellamuthu Subbannagounder, Kris See Ks, Baskar Subramani
Keywords
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