ABSTRACT
BACKGROUND: Interest in the issue of childhood autism has surged in the recent decades. At the same time, despite the significant progress achieved in understanding the etiological and pathogenetic aspects of the condition, effective ways to treat it have continued to elude us. Stem cell therapy appears to hold great promise in the treatment and rehabilitation of patients with both neurological diseases (cerebral palsy, hydrocephalus) and mental disorders (autism, schizophrenia). METHODS: This article presents a case report describing the use of nucleated cord blood cells in a patient with regressive autism and resistance to standard therapies. The child's condition was assessed before treatment and 6 and 12 months after. RESULTS: Clinical observation, psychometric, and instrumental diagnostic methods led to a significant improvement in the child's condition in the form of perception development, reduction of somatosensory disorders, normalization of emotional status, and a development of social and communication skills. CONCLUSION: We assume that the result obtained may be associated with the normalization of the immunological status of our patient thanks to the cord blood cells therapy and consider it necessary to conduct further studies into the effectiveness of the method, taking the pathogenic mechanisms of autism into account.
ABSTRACT
OBJECTIVE: Phase 1 of the SUBSCI I/IIa (Systemic Umbilical Cord Blood Administration in Patients with Acute Severe Contusion Spinal Cord Injury) study focused on safety and primary efficacy of multiple systemic infusions of allogeneic unrelated human umbilical cord blood mononuclear cells in patients with severe acute spinal cord contusion having severe neurologic deficit. The primary end point was safety. The secondary end point was the restoration of motor and sensory function in lower limbs within a 1-year period. METHODS: Ten patients with acute contusion spinal cord injury (SCI) and American Spinal Injury Association (ASIA) level A/B deficit were enrolled into phase 1. Patients were treated with 4 infusions of group-matched and rhesus-matched cord blood samples after primary surgery within 3 days after SCI. All patients were followed up for 12 months after SCI. Safety was assessed using adverse events classification depending on severity and relation to cell therapy. Primary efficacy was assessed using dynamics of deficit (ASIA scale). RESULTS: The overall number of adverse events reached 419 in 10 patients. Only 2 were estimated as possibly related to cell therapy, and the remaining 417 were definitely unrelated. Both adverse events were mild and clinically insignificant. No signs of immunization were found in participants. Analysis of clinical outcomes also showed that cell therapy promotes significant functional restoration of motor function. CONCLUSIONS: The data obtained suggest that systemic administration of allogeneic, non-human leukocyte antigen-matched human umbilical cord blood is safe and shows primary efficacy in adults with severe acute contusion SCI and ASIA level A/B deficit.
Subject(s)
Contusions , Hematopoietic Stem Cell Transplantation , Spinal Cord Injuries , Contusions/therapy , Fetal Blood , Humans , Leukocyte Count , Spinal Cord Injuries/therapyABSTRACT
BACKGROUND AIMS: The term "cerebral palsy" (CP) encompasses many syndromes that emerge from brain damage at early stages of ontogenesis and manifest as the inability to retain a normal body position or perform controlled movements. Existing methods of CP treatment, including various rehabilitation strategies and surgical and pharmacological interventions, are mostly palliative, and there is no specific therapy focused on restoring injured brain function. METHODS: During a post-registration clinical investigation, the safety and efficacy of intravenous infusion of allogeneic human leukocyte antigen (HLA)-unmatched umbilical cord blood (UCB) cells were studied in 80 pediatric patients with cerebral palsy and associated neurological complications. Patients received up to 6 intravenous infusions of AB0/Rh-identical, red blood cell-depleted UCB cells at an average dose of 250 × 10(6) viable cells per infusion. RESULTS: Patients were followed for 3-36 months, and multiple cell infusions did not cause any adverse effects. In contrast, in most patients who received four or more UCB cell infusions, positive dynamics related to significant improvements in neurological status and/or cognitive functions were observed. CONCLUSIONS: 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.