Amyotrophic lateral sclerosis (ALS) is characterised as the loss of voluntary muscle movement control due to a neurological debilitation. This may manifest as the inability to chew, walk, or talk and these symptoms worsen over time, eventually leading to incapacitation, meaning the inability to control all muscle movement. In the early stages of ALS, the disease manifests itself in muscle twitches, muscle cramps, muscle stiffness and muscle weakness, though it will increasingly progress to slurred speech, difficulty in eating, and the other more severe symptoms described.
In the United States in 2016, the Centre for Disease Control and Prevention placed an estimate of 14,000 to 15,000 Americans affected with ALS, though the disease is rather common worldwide. Age plays an important role in targeting ALS as those within the ages of 55 to 75 are more at risk of developing the disease. Men are also slightly more likely to develop the disease though to a much lesser extent.
The cause of the disease is not well known, but a small percentage of ALS is caused by genes carried within families. Even so, no test can accurately diagnose the disease. To date, there is no cure for the disease, and treatments for the disease are limited in efficacy with regards to halting or even slowing disease progression.
In light of this, recent research efforts have been poured into regenerative medicine, and the use of stem cells to reverse the progression of neuronal loss in the brain. This will enable new neurons to be regenerated and restore the mobility loss caused by ALS. In order to further elucidate stem cell effects on ALS, Unique Access Medical is now welcoming patients with ALS to participate in a study of stem cells efficacy in ALS. The stem cell product use will be a new innovation cultivated from a patient’s own blood. This will provide an eventual method of treating ALS.
Autologous Multi-lineage Potential Cell Private Trial Scope
Primary Outcome Measures
To evaluate the incidence of adverse events (AE) in the study population of intrathecal and intravenous administration of Autologous Multi-lineage Potential Cells (AMPC) in patients with ALS. [ Time Frame: 48hours ]
- Fever (>38℃): Evaluation the rate of fever 48 hours after cell transplantation
- Unconsciousness: Evaluation the rate of unconsciousness 48hours after cell transplantation.
- Vomiting: Evaluation the rate of vomiting 48hours after cell transplantation.
- Skin Rash: Evaluation the severity of skin rash 48 hours after cell transplantation
Secondary Outcome Measures
To evaluation the overall response rate (ORR) of intrathecal and intravenous administration of Autologous Multi-lineage Potential Cells (AMPC) in patients with ALS. [ Time Frame: 6months]
- ALSFRS-R: Evaluation the improvement of ALSFRS-R by questionnaire before, 1 month, 3 months and 6months after cell transplantation.
- FVC: Evaluation the improvement of FVC by spectrometry in patients before, 1 month, 3 months and 6months after cell transplantation.
- EMG: Evaluation the improvement of EMG by electromyograph before, 3 months and 6months after cell transplantation.
ESR and hs-CRP: Evaluation the change of ESR and hs-CRP by haematological testing before, 1 month, 3 months and 6months after cell transplantation.
Studying the incidence of adverse events and overall response rate of intrathecal and intravenous administration of Autologous Multi-lineage Potential Cells (AMPC) in patients with ALS.
Brief Summary A private trial for evaluating the incidence of adverse events and overall response rate contributed by AMPC in ALS.
Detailed Description Study Drug Administration:
On day -7 prior to the intervention, medical histories of possible candidates will be examined for suitability for AMPC treatment
On day -5, the patient will undergo a pathology test for the following test items:
- Complete blood count (CBC)
- Hepatitis B/C
- Human T lymphocytic virus type I and II (HTLV-I/II)
- Syphilis serology
- Mycoplasma serology
- Electrolyte/liver function test (E/LFT)
- Fasting glucose
- Triglyceride (TG)
- Erythrocyte sedimentation rate (ESR)
- High sensitivity C-reactive protein (hs-CRP)
- Cogulation test: Prothrombin time, INR and APTT
On day 0, patient will be interviewed with the medical consultant for his/her medical history, including the medication taken recently and history of drug and anaesthesia allergy. Also the patient will be given a general physical exam for the baseline information, then our staff will provide candidates with a consent form and agreement for the administration of AMPC. Peripheral blood will then be collected, ranging from 250mL to 400mL depending on candidate fitness. The blood is collected into a sterile blood bag and sealed. Subsequent processes will be conducted in the blood bag within a closed-system to minimise contamination risks.
On day 0 to day 3, the collected blood will be sent to the laboratory for stem cell culture, and a sample of the collected blood will be sent to a third-party laboratory for contamination testing of the following parameters:
- Bacterial endotoxin
- Total viable aerobic count
- Total viable count
- Microbial growth
- Mycoplasma real-time PCR test
On day 4, the biotest results will be released and must reflect the safety of the sample taken before the cultured stem cells may be released for treatment
On day 5, candidates will receive an intravenous infusion and intrathecal injection of the cultured stem cells. Prior to the administration, the treating doctor will first conduct an allergy skin test to determine suitability for treatment. The cultured stem cells are then reinfused intravenously into the candidate in a process that could take up to 2 hours and rest for half hour.
Then the candidate will be given the intrathecal injection of 3ml-5ml AMPC from point L2-L3 or L3-L4 after local anaesthesia is done.
(Note: the local anaesthesia requires the patient to stay NPO for 4 hours.)
After the intrathecal injection, 4-6 hours lying flat in bed is required for wound recovering.
The candidates participation will take place on day 0 and day 5 for peripheral blood collection and stem cell reinfusion and injection through intravenous infusion and lumbar puncture respectively; with 6 month follow up after treatment
1 month post-treatment follow-up: ALSFRS-R, FCV, ESR and hs-CRP
3 month post-treatment follow-up: ALSFRS-R, FCV, ESR, hs-CRP and EMG
6 month post-treatment follow-up: ALSFRS-R, FCV, ESR, hs-CRP and EMG
Intervention Model: Single group assignment
Masking: No masking (open label)
Primary purpose: Treatment
Amyotrophic Lateral Sclerosis
Biological: Autologous Multi-lineage Potential Cells
AMPC are autologous cells cultured from peripheral blood of the recipient and will be reinfused intravenously on day 5 of the treatment
Experimental: Pilot study
Sample size: 10
To evaluate the safety and the effects of AMPC on ALS by monitoring the incidence of AE and overall response rate.
- Both gender
- Duration of disease40% ALS-FRS>26
- Neurological and psychiatric concomitant disease different from ALS
- Concomitant systemic disease
- Neoplasms or other diseases reducing life expectancy
- Treatment with corticosteroid,Ig,immunosuppressive during 12 months.
- Involvement in other clinical trials
- Patients unable to understand informed consent form and study aims.
- Women who are pregnant or childbearing potential for the duration of the study
- Health volunteer
Male and Female
18 to 65 only
Accepts Healthy Volunteers?
Apply for Trial
If you meet the above eligibility criteria, please feel free to contact us with the relevant medical records and physician notes.
Non-Eligible or Unsure
If you do not fit the above criteria, or are unsure of eligibility, do also contact us by leaving your contact information and our team will get back to you as soon as we can. Alternatively, you may subscribe to the email list to stay up to date regarding the trial.
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