Journal ArticleDev Med Child Neurol · December 2022
AIM: To evaluate safety and motor function after treatment with allogeneic umbilical cord blood (AlloCB) or umbilical cord tissue-derived mesenchymal stromal cells (hCT-MSC) in children with cerebral palsy (CP). METHOD: Ninety-one children (52 males, 39 fe ...
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Journal ArticleStem Cells Transl Med · September 2021
Preclinical and early phase clinical studies suggest that an appropriately dosed umbilical cord blood (CB) infusion has the potential to help improve motor function in young children with cerebral palsy (CP). As many children with CP do not have their own ...
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Journal ArticleDev Med Child Neurol · May 2021
Across disciplines, there is great anticipation that evolving cell therapies may finally provide a therapeutic option for conditions in dire need. These conditions are typically complex and their pathophysiology incompletely understood, hindering the devel ...
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Chapter · January 1, 2021
Autism spectrum disorder (ASD) is a neurodevelopmental disorder that presents in childhood and is characterized by restricted, repetitive behaviors and impairments in social communication. The exact cause in unknown, but genetic and immunologic factors are ...
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Journal ArticleStem Cells Transl Med · October 2020
Ongoing neuroinflammation may contribute to symptoms of autism spectrum disorder (ASD) in at least a portion of affected individuals. Mesenchymal stromal cells (MSCs) have demonstrated the capacity to modulate neuroinflammation, but safety and feasibility ...
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Journal ArticleJ Pediatr · July 2020
OBJECTIVE: To evaluate whether umbilical cord blood (CB) infusion is safe and associated with improved social and communication abilities in children with autism spectrum disorder (ASD). STUDY DESIGN: This prospective, randomized, placebo-controlled, doubl ...
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Chapter · January 1, 2020
Biologic and cell-based therapies are increasingly being developed for a multitude of diseases. They are being explored in the treatment of neurologic conditions, as traditional pharmacologic agents typically cannot fully address the pathologic complexity ...
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Journal ArticleStem Cells Transl Med · February 2019
Autism spectrum disorder (ASD) is a heterogeneous neurodevelopmental disorder characterized by social communication deficits and the presence of restricted interests and repetitive behaviors. We have previously reported significant improvements in behavior ...
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Journal ArticleBMJ Case Rep · January 20, 2019
Pleuropulmonary blastomas (PPB) are rare aggressive paediatric lung malignancies associated with DICER1 variants. We present two cases, a 2-year-old girl with upper respiratory tract symptoms as well as a 6-month-old girl sibling undergoing screening due t ...
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Journal ArticleStem Cells Transl Med · November 2018
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 diagn ...
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Journal ArticlePediatr Res · January 2018
The concept of utilizing human cells for the treatment of medical conditions is not new. In its simplest form, blood product transfusion as treatment of severe hemorrhage has been practiced since the 1800s. The advent of hematopoietic stem cell transplanta ...
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Chapter · January 1, 2018
Cord blood (CB) has been established as a viable source of donor cells for hematopoietic reconstitution in allogeneic transplantation for all of the clinical indications treated with adult hematopoietic stem cells. CB is the only hematopoietic stem cell so ...
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Journal ArticleStem Cells Transl Med · December 2017
Cerebral palsy (CP) is a condition affecting young children that causes lifelong disabilities. Umbilical cord blood cells improve motor function in experimental systems via paracrine signaling. After demonstrating safety, we conducted a phase II trial of a ...
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Journal ArticleStem Cells Transl Med · May 2017
Despite advances in early diagnosis and behavioral therapies, more effective treatments for children with autism spectrum disorder (ASD) are needed. We hypothesized that umbilical cord blood-derived cell therapies may have potential in alleviating ASD symp ...
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Chapter · January 1, 2016
Umbilical cord blood (CB), as a source of hematopoietic stem and progenitor cells for hematopoietic reconstitution, has been used for over two decades in allogeneic transplantation to treat patients with hematological malignancies, bone marrow failure, imm ...
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Journal ArticlePediatr Res · December 2015
BACKGROUND: Babies with congenital hydrocephalus often experience developmental disabilities due to brain injury associated with prolonged increased pressure on the developing brain parenchyma. Umbilical cord blood (CB) infusion has favorable effects in an ...
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Journal ArticleCytotherapy · June 2015
Recovery from neurological injuries is typically incomplete and often results in significant and permanent disabilities. Currently, most available therapies are limited to supportive or palliative measures, aimed at managing the symptoms of the condition. ...
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Journal ArticleNeuroimage Clin · 2015
Cerebral Palsy (CP) refers to a heterogeneous group of permanent but non-progressive movement disorders caused by injury to the developing fetal or infant brain (Bax et al., 2005). Because of its serious long-term consequences, effective interventions that ...
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Chapter · January 1, 2015
Regenerative medicine is dedicated to the study of repairing, replacing, or regenerating damaged human cells, tissues, or organs to restore or establish normal function, and it is predicted to develop into numerous applications to treat a wide variety of c ...
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Journal ArticleTransfusion · February 2014
BACKGROUND: Banked unrelated donor umbilical cord blood (CB) has improved access to hematopoietic stem cell transplantation for patients without a suitably matched donor. In a resource-limited environment, ensuring that the public inventory is enriched wit ...
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Journal ArticleNeuroimage Clin · 2013
Cerebral palsy (CP) is a heterogeneous group of non-progressive motor disorders caused by injury to the developing fetal or infant brain. Although the defining feature of CP is motor impairment, numerous other neurodevelopmental disabilities are associated ...
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Journal ArticleTransfusion · September 2010
BACKGROUND: A pilot study was conducted to determine the safety and feasibility of intravenous administration of autologous umbilical cord blood (CB) in young children with acquired neurologic disorders. Most CB units (CBUs) were electively stored in priva ...
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Chapter · January 1, 2010
Overview. Over the past several decades, hematopoietic stem cell transplantation has emerged as an effective approach to curative therapy for both pediatric and adult patients with aggressive or recurrent malignancies, congenital immunodeficiency diseases, ...
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Journal ArticleArch Pediatr Adolesc Med · December 2009
OBJECTIVES: To quantify the frequency and type of new safety information arising from studies performed under the auspices of the Pediatric Exclusivity Program, to describe the dissemination of these findings in the peer-reviewed literature and compare thi ...
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Journal ArticleAm Heart J · October 2008
BACKGROUND: Congress has authorized the United States Food and Drug Administration (FDA) to provide industry sponsors with a 6-month extension of drug marketing rights under the Pediatric Exclusivity Provision if FDA-requested pediatric drug trials are con ...
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