Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy

Phase I/II Study of Stem Cell Therapy in Patients With Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD), an X-linked recessive genetic disease always progressed slowly，tends to leading proximal skeletal muscle atrophy and weakness of limbs, as well as impaired respiratory muscle and cardiac muscle. To a large extent, patients always lose motor function gradually and die for heart failure or severe infection at the end stage of DMD. At present, the treatment strategy relies on heteropathy accompanied with rehabilitation training. However, the therapeutic effect remains extremely limited.

Human umbilical cord mesenchymal stem cells (hUC-MSCs) have been evidenced to improve motor function, increase muscle strength and reduce abnormal levels of related enzymes, such as creatine kinase (CK), lactate dehydrogenase (LDH), alanine aminotransferase (ALT) and aspartate aminotransferase (AST). This study is aimed to explore the safety and efficacy of hUC-MSCs transplantation for DMD.

This study is designed to investigate the safety and efficacy of human umbilical cord mesenchymal stem cells transplantation in patients with progressive muscular dystrophy.

Inclusion Criteria:

• Aged 5-12 years
• Clinical manifestation, enzymology, electromyogram, gene type confirmed the diagnose of Duchenne muscular dystrophy
• Sign the consent form and follow the clinic trail procedure

Exclusion Criteria:

• Not Duchenne muscular dystrophy
• Any history of hypersensitivity to serum products,or other know drug and food allergy
• Combined Pneumonia or other Severe systemic bacteria infection
• HIV+, TPPA +， patients diagnosed as HBV or HCV
• Tumor Markers +
• Severe psychotic patients, cognitive dysfunction
• Coagulation disorders
• Uncontrolled hypertension after treatment，blood pressure≥180mmHg/110 mmHg
• Other severe systemic or organic disease
• Enrollment in other trials in the last 3 months
• Received any stem cell therapy in past 6 months
• Other criteria that investigator consider improper for inclusion