scholarly journals Stem Cells for Huntington’s Disease (SC4HD): An International Consortium to Facilitate Stem Cell-Based Therapy for Huntington’s Disease

2021 ◽  
pp. 1-6
Author(s):  
Keyword(s):  
Stem Cells ◽  
Stem Cell ◽  
Huntington's Disease ◽  
Huntington’S Disease ◽  
Cell Therapies ◽  
Cell Based Therapy ◽  
Technological Advances ◽  
Regulatory Standards ◽  
Multiple Challenges ◽  
The Brain

Huntington’s disease (HD) research is entering an exciting phase, with new approaches such as huntingtin lowering strategies and cell therapies on the horizon. Technological advances to direct the differentiation of stem cells to desired neural types have opened new strategies for restoring damaged neuronal circuits in HD. However, challenges remain in the implementation of cell therapy approaches for patients suffering from HD. Cell therapies, together with other invasive approaches including allele specific oligonucleotides (ASOs) and viral delivery of huntingtin-lowering agents, require direct delivery of the therapeutic agents locally into the brain or cerebrospinal fluid. Delivering substances directly into the brain is complex and presents multiple challenges, including those related to regulatory requirements, safety and efficacy, surgical instrumentation, trial design, patient profiles, and selection of suitable and sensitive primary and secondary outcomes. In addition, production of clinical grade cell-based medicinal products also requires adherence to regulatory standards with extensive quality control of the protocols and cell products across different laboratories and production centers. Currently, there is no consensus on how best to address these challenges. Here we describe the formation of Stem Cells For Huntington’s Disease (SC4HD: https://www.sc4hd.org/), a network of researchers and clinicians working to develop guidance and greater standardization for the HD field for stem cell based transplantation therapy for HD with a mission to work to develop criteria and guidance for development of a neural intra-cerebral stem cell-based therapy for HD.

scholarly journals Recent Overview of the Use of iPSCs Huntington’s Disease Modeling and Therapy

2020 ◽  
Vol 21 (6) ◽  
pp. 2239 ◽  
Author(s):  
Maria Csobonyeiova ◽  
Stefan Polak ◽  
Lubos Danisovic
Keyword(s):  
Stem Cells ◽  
Stem Cell ◽  
Huntington's Disease ◽  
Huntington’S Disease ◽  
Disease Modeling ◽  
Cell Types ◽  
Cag Repeats ◽  
Neural Cells ◽  
Behavioral Impairment ◽  
Cell Based Therapy

Huntington’s disease (HD) is an inherited, autosomal dominant, degenerative disease characterized by involuntary movements, cognitive decline, and behavioral impairment ending in death. HD is caused by an expansion in the number of CAG repeats in the huntingtin gene on chromosome 4. To date, no effective therapy for preventing the onset or progression of the disease has been found, and many symptoms do not respond to pharmacologic treatment. However, recent results of pre-clinical trials suggest a beneficial effect of stem-cell-based therapy. Induced pluripotent stem cells (iPSCs) represent an unlimited cell source and are the most suitable among the various types of autologous stem cells due to their patient specificity and ability to differentiate into a variety of cell types both in vitro and in vivo. Furthermore, the cultivation of iPSC-derived neural cells offers the possibility of studying the etiopathology of neurodegenerative diseases, such as HD. Moreover, differentiated neural cells can organize into three-dimensional (3D) organoids, mimicking the complex architecture of the brain. In this article, we present a comprehensive review of recent HD models, the methods for differentiating HD–iPSCs into the desired neural cell types, and the progress in gene editing techniques leading toward stem-cell-based therapy.

J03 Clinical translation of stem cell therapies for huntington’s disease (HD)

2021 ◽  
Author(s):  
Anne Rosser ◽  
Monica Busse ◽  
William Gray ◽  
Romina Aron-Badin ◽  
Anselm Perrier ◽  
...  
Keyword(s):  
Stem Cell ◽  
Huntington's Disease ◽  
Huntington’S Disease ◽  
Clinical Translation ◽  
Stem Cell Therapies ◽  
Cell Therapies

scholarly journals Developing stem cell therapies for juvenile and adult-onset Huntington's disease

2015 ◽  
Vol 10 (5) ◽  
pp. 623-646 ◽  
Author(s):  
Kyle D Fink ◽  
Peter Deng ◽  
Audrey Torrest ◽  
Heather Stewart ◽  
Kari Pollock ◽  
...  
Keyword(s):  
Stem Cell ◽  
Huntington's Disease ◽  
Huntington’S Disease ◽  
Adult Onset ◽  
Stem Cell Therapies ◽  
Cell Therapies

The Effect of Stem Cell Therapy in Autism Spectrum Disorder

2020 ◽  
Author(s):  
Vincent S. Gallicchio
Keyword(s):  
Autism Spectrum Disorder ◽  
Stem Cells ◽  
Immune Response ◽  
Stem Cell ◽  
Cell Therapy ◽  
Autism Spectrum ◽  
Cell Therapies ◽  
Neuro Inflammation ◽  
Inflammatory Molecules ◽  
The Brain

Autism Spectrum Disorder [ASD] is a neuro developmental disorder that is characterized by abnormal social interaction/communication and restricted, repetitive patterns of behavior, interests, or activities. Although there is a lack of knowledge surrounding the ASD’s etiology, one common hypothesis posits that the causative pathology is immune system deregulation [ISD]. Patients with ASD experience ISD in the form of overactive microglia and astroglia in the brain, overactive cytokines in the brain and blood plasma, and underactive T lymphocytes in the blood plasma. Mesenchymal stem cells [MSCs] and mononuclear cells, which contain a mixture of MSCs and hematopoietic stem cells [HSCs], are promising candidates for treatment of ASD. MSCs secrete several molecules that may restore injured tissue and anti-inflammatory molecules that may mediate neuro inflammation. MSCs also exhibit immuno modulatory effects which may regulate the immune response observed in ASD.HSCs secrete various cytokines, chemokines, and growth factors that may further regulate the abnormal immune response observed in ASD. In addition, HSC CD34+ down regulates pro-inflammatory molecules and up regulates anti-inflammatory molecules which may mediate neuro inflammation in ASD. Based on the results of several clinical trials, MSC and mononuclear cell therapies are safe and effective. To date, they have not been shown to cause adverse side effects. In addition, there have been several instances of reduced ASD symptoms due to the therapies. Nevertheless, much research is still needed into further investigating the etiology of ASD and the mechanism of stem cell therapies to truly understand the benefits of stem cell therapy for ASD.

scholarly journals Traumatic Brain Injury and Stem Cells: An Overview of Clinical Trials, the Current Treatments and Future Therapeutic Approaches

Medicina ◽  
2020 ◽  
Vol 56 (3) ◽  
pp. 137 ◽  
Author(s):  
Giovanni Schepici ◽  
Serena Silvestro ◽  
Placido Bramanti ◽  
Emanuela Mazzon
Keyword(s):  
Traumatic Brain Injury ◽  
Stem Cells ◽  
Clinical Trials ◽  
Stem Cell ◽  
Brain Injury ◽  
Brain Damage ◽  
Physical Damage ◽  
Safety And Efficacy ◽  
Cell Based Therapy ◽  
The Brain

Traumatic brain injury represents physical damage to the brain tissue that induces transitory or permanent neurological disabilities. The traumatic injury activates an important inflammatory response, followed by a cascade of events that lead to neuronal loss and further brain damage. Maintaining proper ventilation, a normal level of oxygenation, and adequate blood pressure are the main therapeutic strategies performed after injury. Surgery is often necessary for patients with more serious injuries. However, to date, there are no therapies that completely resolve the brain damage suffered following the trauma. Stem cells, due to their capacity to differentiate into neuronal cells and through releasing neurotrophic factors, seem to be a valid strategy to use in the treatment of traumatic brain injury. The purpose of this review is to provide an overview of clinical trials, aimed to evaluate the use of stem cell-based therapy in traumatic brain injury. These studies aim to assess the safety and efficacy of stem cells in this disease. The results available so far are few; therefore, future studies need in order to evaluate the safety and efficacy of stem cell transplantation in traumatic brain injury.

Stem cell-based therapy for Huntington's disease

2013 ◽  
Vol 114 (4) ◽  
pp. 754-763 ◽  
Author(s):  
Christof Maucksch ◽  
Elena M. Vazey ◽  
Renee J. Gordon ◽  
Bronwen Connor
Keyword(s):  
Stem Cell ◽  
Huntington's Disease ◽  
Huntington’S Disease ◽  
Cell Based Therapy

Differentiation of Rat Neural Stem Cells Following Transplantation in the Brain of Huntington's Disease Rat Model

2009 ◽  
Vol 18 (1) ◽  
pp. 37
Author(s):  
Hwa Lee Ryu ◽  
So Yeon Lee ◽  
Keunwoo Park ◽  
Changhoon Kim ◽  
Byung Kwan Jin ◽  
...  
Keyword(s):  
Stem Cells ◽  
Neural Stem Cells ◽  
Huntington's Disease ◽  
Huntington’S Disease ◽  
Rat Model ◽  
The Brain

Stem cell therapies for malignant glioma

2005 ◽  
Vol 19 (3) ◽  
pp. 1-11 ◽  
Author(s):  
Moneeb Ehtesham ◽  
Charles B. Stevenson ◽  
Reid C. Thompson
Keyword(s):  
Stem Cells ◽  
Stem Cell ◽  
Malignant Glioma ◽  
Treatment Strategies ◽  
Therapeutic Benefit ◽  
Therapeutic Modality ◽  
Clinical Implementation ◽  
Stem Cell Therapies ◽  
Cell Therapies ◽  
The Brain

The prognosis for patients with malignant glioma, which is the most common primary intracranial neoplasm, remains dismal despite significant progress in neurooncological therapies and technology. This is largely due to the inability of current treatment strategies to address the highly invasive nature of this disease. Malignant glial cells often disseminate throughout the brain, making it exceedingly difficult to target and treat all intracranial neoplastic foci, with the result that tumor recurrence is inevitable despite aggressive surgery and adjuvant radiotherapy and/or chemotherapy. The use of neural stem cells (NSCs) as delivery vehicles for tumor-toxic molecules represents the first experimental strategy aimed specifically at targeting disseminated tumor pockets. Investigators have demonstrated that NSCs possess robust tropism for infiltrating tumor cells, and that they can be used to deliver therapeutic agents directly to tumor satellites, with significant therapeutic benefit. With the aim of developing these findings into a clinically viable technology that would not be hindered by ethical and tissue rejection–related concerns, the use of adult tissue–derived stem cells has recently been explored. These technologies represent important progress in the development of a treatment strategy that can specifically target disseminated neoplastic pockets within the brain. Despite encouraging results in preclinical models, however, there are significant impediments that must be overcome prior to clinical implementation of this strategy. Key among these are an inadequate understanding of the specific tropic mechanisms that govern NSC migration toward invasive tumor, and the need to refine the processes used to generate tumor-tropic stem cells from adult tissues so that this can be accomplished in a clinically practicable fashion. Despite these limitations, the use of stem cell therapies for brain tumors holds significant promise and may emerge as an important therapeutic modality for patients with malignant glioma.

scholarly journals Minimally Invasive Delivery of Microbeads with Encapsulated, Viable and Quiescent Neural Stem Cells to the Adult Subventricular Zone

2019 ◽  
Vol 9 (1) ◽  
Author(s):  
Rita Matta ◽  
Seyoung Lee ◽  
Nafiisha Genet ◽  
Karen K. Hirschi ◽  
Jean-Leon Thomas ◽  
...  
Keyword(s):  
Stem Cells ◽  
Stem Cell ◽  
Subventricular Zone ◽  
Critical Role ◽  
Neurological Injury ◽  
Cell Therapies ◽  
Neuronal Stem Cells ◽  
The Brain

AbstractStem cell therapies demonstrate promising results as treatment for neurological disease and injury, owing to their innate ability to enhance endogenous neural tissue repair and promote functional recovery. However, delivery of undifferentiated and viable neuronal stem cells requires an engineered delivery system that promotes integration of transplanted cells into the inflamed and cytotoxic region of damaged tissue. Within the brain, endothelial cells (EC) of the subventricular zone play a critical role in neural stem cell (NSC) maintenance, quiescence and survival. Therefore, here, we describe the use of polyethylene glycol microbeads for the coincident delivery of EC and NSC as a means of enhancing appropriate NSC quiescence and survival during transplantation into the mouse brain. We demonstrate that EC and NSC co-encapsulation maintained NSC quiescence, enhanced NSC viability, and facilitated NSC extravasation in vitro, as compared to NSC encapsulated alone. In addition, co-encapsulated cells delivered to an in vivo non-injury model reduced inflammatory response compared to freely injected NSC. These results suggest the strong potential of a biomimetic engineered niche for NSC delivery into the brain following neurological injury.

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