To preliminarily evaluate the efficacy and safety of umbilical cord-derived mesenchymal stem cells (UC-MSCs), and compare the efficacy of UC-MSCs administered through the intravenous, intrathecal, and intranasal routes, in the treatment of cerebral palsy in children.
Cerebral palsy refers to a neurological disorder caused by a non-progressive brain injury or malformation that occurs in early childhood. It can result in central motor deficits, developmental retardation, abnormal posture, abnormal muscular strength, abnormal muscle tone and/or dysreflexia. It has a high disability rate. There is currently no effective treatment for cerebral palsy. Conventional treatments for cerebral palsy are often tiresome and expensive and have a slow onset of action. Stem cells have been recently used in the treatment of cerebral palsy. This provides a novel method for the treatment of cerebral palsy. According to the existing clinical studies, neural stem cells, bone marrow-derived mesenchymal stem cells, and umbilical cord-derived mesenchymal stem cells (UC-MSCs) are mainly used as the seed cells for the treatment of cerebral palsy. UC-MSCs are the most promising seed cells for the treatment of cerebral palsy because of the advantages including rich sources, ease of harvesting, short doubling time, low immunogenicity, long-time survival post-transplantation, and no ethical issues. UC-MSCs have been widely used to treat Parkinson's disease, rheumatoid arthritis, traumatic brain injury, aplastic anemia, and decompensated liver disease. However,only two studies, and performed only in China, are reported on the treatment of cerebral palsy with UC-MSCs. One from the Hospital 463 of PLA reported 51 patients with cerebral palsy whose symptoms had not been obviously improved after 1 year of rehabilitative treatment. These patients received intrathecal injection of UC-MSCs for three times, once a week, followed by one injection of UC-MSCs via the peripheral vein in the fourth week. Four UC-MSCs injections, once per week, were considered as one course of treatment. Activities of daily living scale score was compared between before and after treatment to evaluate efficacy. Another study is a case report from China. In this report, a combined intravenous and intrathecal injection of UC-MSCs was used to treat cerebral palsy in a 5-year-old child. 28-month follow-up results revealed that the child's gross motor function, immune function, muscle strength, and language ability improved and adverse reactions were not obvious.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
44
The control group received routine rehabilitation treatments including acupuncture, massage, Bobath therapy, and Vojat therapy.
Injection of umbilical cord-derived mesenchymal stem cells (UC-MSCs) (at least 1×10\^7), once every 2 weeks, one course of treatment including two injections within 4 weeks, a total of two courses of treatment.
The First Affiliated Hospital of Dalian Medical University
Dalian, Liaoning, China
The Gross Motor Function Measure (GMFM)-88 item score
a 4-point scale that is divided into five categories including lying and rolling, sitting, crawling and kneeling, standing, walking, running, and jumping. A score of 0 indicates that the child does not initiate the task; 1 indicates that the child initiates the task (completes \< 10% of the activity); 2 indicates that the child partially completes the task (completes from 10 to 99% of the activity); 3 indicates that the child completes the task (100%). The sum of score of the five categories is used as the evaluation outcome.It is used to evaluate gross motor function.
Time frame: 12 months after 2 courses of treatment with UC-MSCs.
Fine Motor Function Measure scale (FMFM) score
To evaluate fine motor function.The scale is divided into 45 items covering visual tracing (7 items), upper extremity range of motion (8 items), grab ability (8 items), operational ability (10 items), and hand-eye coordination (12 items). The FMFM is a 4-point scale with the score range of 0-3. The original total score is 135. The summed score for evaluating fine motor function measure is 0-100.
Time frame: 1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.
The Modified Ashworth scale (MAS) score
To evaluate muscle tone. The MAS score is divided into six grades: 0, 1, 1+, 2, 3 and 4. Passive full ranges of motion exercises are performed by moving the knee and ankle until the patient feel a slight resistance from hamstrings and plantar flexor muscles (soleus muscle and gastrocnemius muscle). The average score is calculated across three repeated measurements.
Time frame: 1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.
Gesell Development Schedule score
To evaluate neurobehavioral development.
Time frame: 1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.
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Incidence of adverse reactions
Liver and kidney dysfunction, fever, infection, vomiting.
Time frame: 1, 3, 6, 9 and 12 months after 2 courses of treatment with UC-MSCs.