Patients with Amyotrophic Lateral Sclerosis (ALS) typically endure a progressive paralysis due to the continued loss of motoneurons that leads them to death in less than 5 years. No treatment has changed its natural history. Intrathecal injection of umbilical cord mesenchymal stem cells can secret trophic factors that keep the motorneurons functional. The investigators have designed a phase I/II clinical trial to check the feasibility of this approach in humans.
A total of 30 diagnosed ALS patients. The patients would got these symptoms such as gait difficulty and tremor, hand incoordination or speech difficulties.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
30
after stem cell prepared, the patients accepted 4 times stem cell transplantations through lumbar puncture, the time is 3-5days between two treatments. The patient would have to be in the bed at least 6 hours and removed the pillow.
Yihua An
Beijing, China
Nerve functional evaluation
1. Medical Research Council scale：To determine the strength of bilateral little finger abductor muscle and anterior tibial. 2. To evaluate the situation of upper motor neuron impairment through the Ellis reflex scale 3. The severity evaluation: NorrisALS score and ALS functional rating scale, ALSFRS 4. Speed of disease progression by the following formula：Progress rate = (40-ALSFRS score) / course 5. Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI) and memory (Mini Mental State Examination)
Time frame: within one week before,1month,6months,12months and 24months after transplantation
Forced vital capacity
vital capacity(VC）、forced vital capacity( FVC）、forced expiratory volune(FEV1）、FEV1/FVC、maximal voluntary ventilation(MVV）、peak expiratory flow(PEF）
Time frame: within one week before,1month,6months,12months and 24months after transplantation
Blood test
white blood cell、neutrophilic granulocyte、leukomonocyte；glutamic pyruvic transaminase(GPT)、glutamic oxalacetic transaminase、lactate dehydrogenase(LDH)、;hydroxybutyrate dehydrogenase(HBDH)、phosphocreatine kinase(CK)；acidum uricum(UA)、creatinine(Cr)、α1- microglobulin、β2- microglobulin；lymphotoxin(LCT). Tumor markers , Lymphocytes classification, cholesterol total、Triglyceride、low density lipoprotein、Glycosylated serum protein glycosylated hemoglobin、Islet function，Na+、K+
Time frame: within one week before,1month,6months,12months and 24months after transplantation
Urinal test
proteinum、akaryocyte、α1- microglobulin、β2- microglobulin. CSF test:：IgA, IgG quantitation
Time frame: within one week before,1month,6months,12months and 24months after transplantation
Electrophysiology examination
motor evoked potential(MEP)、nerve conduction and electromyologram(EMG)
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Time frame: within one week before,1month,6months,12months and 24months after transplantation