Cerebral palsy (CP) is a disorder of movement, muscle tone and/or posture that begins at or around birth. It is the most common physical disability in childhood affecting up to 1 in 500 newborns and over 17 million people worldwide.
Physical disability of CP is diverse and can range from muscle weakness to complete lack of movement. In addition to physical disability, children with CP are likely to have other medical conditions and impairments. Intellectual impairment, behavior disorders, sleep disorders and others are common.
Although CP is a life-long condition without a known cure, early treatment and intervention can improve symptoms and the quality of life of those who have the condition. The conventional approach includes physical therapy, occupational therapy, massage therapy, speech therapy, behavioral/cognitive therapies, surgery, etc. Medications are also prescribed to CP patients in order to reduce symptoms and treat other medical conditions. Despite these various treatment options, the conventional approach to treating CP is limited and often does not produce significant improvement in symptoms or quality of life. The search for more effective treatment options continues.
Studies have shown stem cells were effective for children with neurological disorders including CP. Several studies have demonstrated stem cells improved gross motor function, muscle tone, spasticity, development and quality of life in patients with CP, and have also shown to reduce brain inflammation, increase whole brain connectivity and activate motor regions of the brain.
Based on early human clinical trials there appears to be a strong argument for the safe and effective use of stem cell therapy in the treatment of CP. While we wait for phase II/III trials the current data is encouraging and supports use of stem cell therapy in the clinical setting.
|Year||Author||No. of Patients||Delivery and Cell Type||Follow-up||Safety Outcome||Efficacy Outcome|
|2018||Huang et al||27||IV UC-MSCs||24 months||No adverse immunological response and no SAEs. Common AEs included diarrhea and URI. No adverse changes in lab tests.||Improvements in gross motor and comprehensive functions measured by GMFM-88 and CFA. Changes in electrophysiology of less diffuse slow waves on MRI.|
|2017||Sun et al||63||IV UCB||12 months||No SAEs. One patient experienced infusion related hives and low grade fever to both placebo and UCB treatment.||UCB above the median cell dose demonstrated statistically significant and clinically meaningful improvement in GMFM-66 and PDMS-2 after 1 year post treatment. MRI showed significant improvement in whole brain connectivity.|
|2015||Romanov et al||80||IV UCB||36 months||No SAEs. No acute or delayed AES reported.||Significant improvements in neurological status, physical capacity and cognitive functions in patients who received >4 infusions. Overall improvements in neurological, physical activity and/or intellectual development were seen in approximately 70% patients.|
|2015||Kang et al||36||IV UCB||6 months||No SAEs reported.||Significant Improvements in muscle strength and gross motor skills. F-FDG PET scans showed increased metabolism in motor regions of the brain after UCB infusion and indicate an activation of motor function.|
|2013||Min et al||96||IV UCB||6 months||Incidence of SAEs did not differ between groups. Pneumonia and irritability were most frequent AEs in treatment group.||Improvements in motor, social and cognitive functions measured by GMPM, BSID-II, GMFM., WeeFIM. The pUCB group showed greater motor function improvements, greater cognitive outcomes and had increased FA on MRI.|
|2012||Lee et al||20||IV CB-MNCs||6 months||Minimal adverse events which were controlled with medication and intravenous hydration.||25% of children had partial improvements in neurodevelopment evaluations tests after a single IV of CB-MNCs. Only 10% of patients demonstrated improved perfusion. Neurodevelopment improvements were significant in patients with hemiplegia or diplegia compared to quadriplegia.|
* IV = intravenous, UCB = umbilical cord blood, CB-MNCs = cord blood derived mononuclear cells, AEs= adverse events, SAEs = serious adverse events, GMPM = gross motor performance measure, GMFM-88 = gross motor function measure, CFA = comprehensive functional assessment, PDMS-2 = Peabody development motor scales, F-FDG PET = Fluorodeoxyglucose positron emission tomography, BSID-II = Bayley scales of infant development, WeeFIM =Wee functional independence measure, FA = fractional anisotropy.
Early Human Clinical Trials of Stem Cells in Cerebral Palsy (CP)
- Improvement in gross motor abilities in lying and rolling, sitting, crawling and kneeling, standing and walking, running, and jumping. (Huang et al, Sun et al, Novak et al, Min et al)
- Improvement in comprehensive function in cognizance, language competence, self-care, motor function, and social adaptability.(Huang et al)
- Improvement in muscle tone and muscle strength. (Romanov et al, Kang et al)
- Improvement in neurological and physical status.(Romanov et al)
- Improvement in intellectual development and mental status.(Romanov et al)
- Decreased brain inflammation and inflammatory markers in blood. (Kang et al)
- Increased whole brain connectivity and metabolism in motor regions of brain. (Sun et al, Kang et al)
Specific Conclusions From Selected Studies:
“The results of the study indicated that hUCB-MSC infusion with basic rehabilitation was safe and effective in improving gross motor and comprehensive functions in children with CP.”
- A Randomized, Placebo-Controlled Trial of Human Umbilical Cord Blood Mesenchymal Stem Cell Infusion for Children With Cerebral Palsy (Huang et al 2018)
“Results of this study suggest that appropriately dosed autologous cord blood (ACB) infusion improves brain connectivity and gross motor function in young children with CP.”
- Effect of Autologous Cord Blood Infusion on Motor Function and Brain Connectivity in Young Children with Cerebral Palsy: A Randomized, Placebo-Controlled Trial (Sun et al 2017)
“Stem cells appeared to induce short-term improvements in motor skills.
- Concise Review: Stem Cell Interventions for People with Cerebral Palsy: Systematic Review with Meta-Analysis (Novak et al 2016)
“The results confirm that multiple intravenous infusions of allogeneic AB0/Rh-identical UCB cells may be a safe and effective procedure and could be included in treatment and rehabilitation programs for juvenile patients with cerebral palsy.”
- Human allogeneic AB0/Rh-identical umbilical cord blood cells in the treatment of juvenile patients with cerebral palsy (Romanov et al 2015)
“In this trial, treatment with UCB alone improved motor outcomes and induced systemic immune reactions and anti-inflammatory changes in the brain. Generally, motor outcomes were positively correlated with the number of UCB cells administered: a higher number of cells resulted in better outcomes.”
- Involvement of Immune Responses in the Efficacy of Cord Blood Cell Therapy for Cerebral Palsy (Kang et al 2015)
“In conclusion, UCB treatment ameliorated motor and cognitive dysfunction in children with CP undergoing active rehabilitation, accompanied by structural and metabolic changes in the brain.”
- Umbilical cord blood therapy potentiated with erythropoietin for children with cerebral palsy: A double-blind, randomized, placebo-controlled trial (Min et al 2013)
“Autologous CB infusion is safe and feasible, and has yielded potential benefits in children with CP.”
- Safety and feasibility of countering neurological impairment by intravenous administration of autologous cord blood in cerebral palsy. (Lee et al 2012)
Brought to you by:
Ahvie Herskowitz, MD, President of ACAM
Director of Anatara Medicine
Clinical Professor of Medicine at UC San Francisco (2014)
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