MULTIPLE WHARTON JELLY MESENCHYMAL STEM CELLS-DERIVED HE-ATMP TRANSPLANTATIONS OVERCOMES DRUG-RESISTANT EPILEPSY IN CHILDREN

Speaker

Milczarek, Olga (Jagiellonia University Medical College Faculty of Medicine, Institute of Pediatrics, Department of Children’s Neurosurgery)

Description

"Introduction
The wide spectrum of brain injuries experienced in neonates and preterm newborns and the potential plasticity of the CNS prompts us to seek solutions in the field of neuroregeneration in this group of patients and to prevent the worst effects of prematurity and perinatal problems. Drug-resistant epilepsy remains one of the biggest problems of prematurity and its consequences. There is a need among patients suffering from drug-resistant epilepsy (DRE) for more efficient and less toxic treatments than long time pharmacotherapy.
Method
The purpose of this study was to evaluate the safety and potential efficacy of multiple administrations of HE-ATMP comprised of 3x107 WJMSCs. A study group was composed of six patients, qualified for the treatment with a diagnosis of chronic hypoxic – ischemic encephalopathy and inflammation (including sepsis, and systemic inflammatory reaction) with diagnosis of DRE. All the patients underwent repeated rounds of HE-ATMP administration to the CSF via LP.
Results
There were no adverse events, and the therapy was safe and feasible over 2 years of follow-up. The therapy resulted in neurological and cognitive improvement in all patients, including a reduction in the number of epileptic seizures (from 40 per day to 2-5 per week) and an absence of status epilepticus episodes (from 4 per week to 0 per week). The number of discharges on the EEG evaluation was decreased, and cognitive improvement was noted with respect to reactions to light and sound, emotions, and motor function.
Conclusion
After two years of follow-up examination, we demonstrated the safety and beneficial effects of WJMSCSs transplantation, including neurological improvements and reduction of functional neurodeficits. We are aware that the samples size of this study is relatively small, therefore data need to be further tested in larger groups."

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