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

“…We suggest that frameworks developed for Huntington’s disease will help to accelerate progress for a wide range of other neurodegenerative conditions. 17 …”

Section: Conclusion: Bringing Preclinical Knowledge Into a Clinical S...mentioning

confidence: 99%

“…In order to achieve this in the safest and most efficient way, we have established ourselves as an international consortium of experts, which we call ‘Stem cells for Huntington’s disease’ (SC4HD; www.sc4hd.org ). 17 SC4HD aims to provide a platform for discussion and to share experience in order to provide guidance and to generate a robust clinical development plan across a range of stem cell-based therapies for Huntington’s disease. The consortium works closely with the European Huntington’s Disease Network (EHDN) Advanced Therapies Working Group (ATWG: http://www.ehdn.org/advanced-therapies-wg/ ), which aims to address similar issues for both cells and molecules, and with the California Institute for Regenerative Medicine ( https://www.cirm.ca.gov/ ), that seeks to provide stem cell-based therapies for a range of human diseases.…”

Section: Introductionmentioning

confidence: 99%

Translating cell therapies for neurodegenerative diseases: Huntington’s disease as a model disorder

Rosser

Busse

Gray

et al. 2022

Brain

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There has been substantial progress in the development of regenerative medicine strategies for central nervous system disorders over the last decade, with progression to early clinical studies for some conditions. However, there are multiple challenges along the translational pipeline, many of which are common across diseases and pertinent to multiple donor cell types. These include defining the point at which the preclinical data are sufficiently compelling to permit progression to the first clinical studies; scaling-up, characterization, quality control and validation of the cell product; design, validation and approval of the surgical device; and operative procedures for safe and effective delivery of cell product to the brain. Furthermore, clinical trials that incorporate principles of efficient design and disease specific outcomes are urgently needed (particularly for those undertaken in rare diseases, where relatively small cohorts are an additional limiting factor), and all processes must be adaptable in a dynamic regulatory environment. Here we set out the challenges associated with the clinical translation of cell therapy, using Huntington’s disease as a specific example, and suggest potential strategies to address these challenges. Huntington’s disease presents a clear unmet need, but, importantly, it is an autosomal dominant condition with a readily available gene test, full genetic penetrance and a wide range of associated animal models, which together mean that it is a powerful condition in which to develop principles and test experimental therapeutics. We propose that solving these challenges in Huntington’s disease would provide a road map for many other neurological conditions. This white paper represents a consensus opinion emerging from a series of meetings of the international translational platforms Stem Cells For Huntington’s Disease and the European Huntington’s Disease Network Advanced Therapies Working Group, established to identify the challenges of cell therapy, share experience, develop guidance, and highlight future directions, with the aim to expedite progress towards therapies for clinical benefit in Huntington’s disease.

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Stem Cell–Based Therapies: What Interventional Radiologists Need to Know

Commander

Stavas

2021

Semin intervent Radiol

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As the basic units of biological organization, stem cells and their progenitors are essential for developing and regenerating organs and tissue systems using their unique self-renewal capability and differentiation potential into multiple cell lineages. Stem cells are consistently present throughout the entire human development, from the zygote to adulthood. Over the past decades, significant efforts have been made in biology, genetics, and biotechnology to develop stem cell–based therapies using embryonic and adult autologous or allogeneic stem cells for diseases without therapies or difficult to treat. Stem cell–based therapies require optimum administration of stem cells into damaged organs to promote structural regeneration and improve function. Maximum clinical efficacy is highly dependent on the successful delivery of stem cells to the target tissue. Direct image-guided locoregional injections into target tissues offer an option to increase therapeutic outcomes. Interventional radiologists have the opportunity to perform a key role in delivering stem cells more efficiently using minimally invasive techniques. This review discusses the types and sources of stem cells and the current clinical applications of stem cell–based therapies. In addition, the regulatory considerations, logistics, and potential roles of interventional Radiology are also discussed with the review of the literature.

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

Cited by 3 publications

References 1 publication

In vitro-derived medium spiny neurons recapitulate human striatal development and complexity at single-cell resolution

In vitro-derived medium spiny neurons recapitulate human striatal development and complexity at single-cell resolution

Translating cell therapies for neurodegenerative diseases: Huntington’s disease as a model disorder

Stem Cell–Based Therapies: What Interventional Radiologists Need to Know

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