Electrophysiological Biomarkers Predict Clinical Improvement in an Open-Label Trial Assessing Efficacy of Autologous Umbilical Cord Blood for Treatment of Autism.
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2018-11
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This study was a phase I, single-center, and open-label trial of a single intravenous infusion of autologous umbilical cord blood in young children with autism spectrum disorder (ASD). Twenty-five children between the ages of 2 and 6 with a confirmed diagnosis of ASD and a qualified banked autologous umbilical cord blood unit were enrolled. Safety results and clinical outcomes measured at 6 and 12 months post-infusion have been previously published. The purpose of the present analysis was to explore whether measures of electroencephalography (EEG) theta, alpha, and beta power showed evidence of change after treatment and whether baseline EEG characteristics were predictive of clinical improvement. The primary endpoint was the parent-reported Vineland adaptive behavior scales-II socialization subscale score, collected at baseline, 6- and 12-month visits. In addition, the expressive one word picture vocabulary test 4 and the clinical global impression-improvement scale were administered. Electrophysiological recordings were taken during viewing of dynamic social and nonsocial stimuli at 6 and 12 months post-treatment. Significant changes in EEG spectral characteristics were found by 12 months post-infusion, which were characterized by increased alpha and beta power and decreased EEG theta power. Furthermore, higher baseline posterior EEG beta power was associated with a greater degree of improvement in social communication symptoms, highlighting the potential for an EEG biomarker to predict variation in outcome. Taken together, the results suggest that EEG measures may be useful endpoints for future ASD clinical trials. Stem Cells Translational Medicine 2018;7:783-791.
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Murias, Michael, Samantha Major, Scott Compton, Jessica Buttinger, Jessica M Sun, Joanne Kurtzberg and Geraldine Dawson (2018). Electrophysiological Biomarkers Predict Clinical Improvement in an Open-Label Trial Assessing Efficacy of Autologous Umbilical Cord Blood for Treatment of Autism. Stem cells translational medicine, 7(11). pp. 783–791. 10.1002/sctm.18-0090 Retrieved from https://hdl.handle.net/10161/24595.
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Jessica Muller Sun
Joanne Kurtzberg
Dr. Kurtzberg is an internationally renowned expert in pediatric hematology/oncology, pediatric blood and marrow transplantation, umbilical cord blood banking and transplantation, and novel applications of cord blood and birthing tissues in the emerging fields of cellular therapies and regenerative medicine. Dr. Kurtzberg serves as the Director of the Marcus Center for Cellular Cures (MC3), Director of the Pediatric Transplant and Cellular Therapy Program, Director of the Carolinas Cord Blood Bank, and Co-Director of the Stem Cell Transplant Laboratory at Duke University. The Carolinas Cord Blood Bank is an FDA licensed public cord blood bank distributing unrelated cord blood units for donors for hematopoietic stem cell transplantation (HSCT) through the CW Bill Young Cell Transplantation Program. The Robertson GMP Cell Manufacturing Laboratory supports manufacturing of RETHYMIC (BLA, Enzyvant, 2021), allogeneic cord tissue derived and bone marrow derived mesenchymal stromal cells (MSCs), and DUOC, a microglial/macrophage cell derived from cord blood.
Dr. Kurtzberg’s research in MC3 focuses on translational studies from bench to bedside, seeking to develop transformative clinical therapies using cells, tissues, molecules, genes, and biomaterials to treat diseases and injuries that currently lack effective treatments. Recent areas of investigation in MC3 include clinical trials investigating the safety and efficacy of autologous and allogeneic cord blood in children with neonatal brain injury – hypoxic ischemic encephalopathy (HIE), cerebral palsy (CP), and autism. Clinical trials testing allogeneic cord blood are also being conducted in adults with acute ischemic stroke. Clinical trials optimizing manufacturing and testing the safety and efficacy of cord tissue MSCs in children with autism, CP and HIE and adults with COVID-lung disease are underway. DUOC, given intrathecally, is under study in children with leukodystrophies and adults with primary progressive multiple sclerosis.
In the past, Dr. Kurtzberg has developed novel chemotherapeutic drugs for acute leukemias, assays enumerating ALDH bright cells to predict cord blood unit potency, methods of cord blood expansion, potency assays for targeted cell and tissue based therapies. Dr. Kurtzberg currently holds several INDs for investigational clinical trials from the FDA. She has also trained numerous medical students, residents, clinical and post-doctoral fellows over the course of her career.
Geraldine Dawson
Geraldine Dawson is the William Cleland Distinguished Professor of Psychiatry and Behavioral Sciences at Duke University, where she also is a Professor of Pediatrics and Psychology & Neuroscience. Dawson also is the Founding Director of the Duke Center for Autism and Brain Development, an NIH Autism Center of Excellence, which is an interdisciplinary research program and clinic, aimed to improve the lives of those diagnosed with autism through research, education, clinical services, and policy. Dawson received a Ph.D. in Developmental and Child Clinical Psychology from the University of Washington and completed a clinical internship at the UCLA Neuropsychiatric Institute.
Dawson's work focuses on improving methods for early detection and intervention for autism, understanding brain function in autism, and validation of autism EEG biomarkers. She co-developed the Early Start Denver Model, an empirically-validated early autism intervention that is used worldwide. She collaborates with colleagues in the departments of computer science and engineering, pediatrics, and biostatistics to develop novel digital health approaches to autism screening and outcome monitoring.
Dawson previously served as Director of the Duke Institute for Brain Sciences, President of the International Society for Autism Research, and was appointed by the US Secretary of Health as a member of the NIH Interagency Autism Coordinating Committee (IACC) which develops the federal strategic plan for autism research, services, and policy. Dawson is a member of the American Academy of Arts and Sciences. She was Founding Director of the University of Washington (UW) Autism Center and the Duke Center for Autism and Brain Development. Dawson's awards include the American Psychological Association Distinguished Career Award (Div53); Association for Psychological Science Lifetime Achievement Award; Clarivate Top 1% Cited Researcher Across All Scientific Fields; among others. Dawson is a Fellow of the International Society for Autism Research, the American Psychological Society, and the American Psychological Association.
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