Abstract:In July 2014, the California Institute for Regenerative Medicine held a workshop to consider strategies for overcoming immune rejection in patients receiving stem cell treatments. A Roadmap to Tolerance was developed, identifying four fundamental steps for advancing strategies to prevent graft rejection and induce tolerance to stem cell‐based tissues.
“…Recent clinical experiments using allo‐HSCT in conjunction with renal transplantation have demonstrated feasibility in some patients to achieve transplantation tolerance between donors and recipients with the removal of all post‐HSCT immunosuppressive drugs . Other strategies to induce tolerance were the subject of a CIRM Workshop and Perspective in 2015, and are discussed further there . A third general approach to address complications of tissue mismatch involves development of targeted therapies for the treatment of chronic GVHD including the in vivo expansion of endogenous regulatory T lymphocytes (Treg) with low‐dose interleukin‐2 or ex vivo expansion of Treg subsets followed by their in vivo administration …”
Section: Medical and Procedural Risks Of Hsctmentioning
Hematopoietic stem cell transplantation (HSCT) is broadly used for treating and curing hematological cancers and various disorders of the blood and immune system. However, its true therapeutic potential remains vastly constrained by significant scientific and technical hurdles that preclude expansion to new indications and limit the number of patients who could benefit from, gain access to, or financially afford the procedure. To define and overcome these challenges, the California Institute for Regenerative Medicine (CIRM) held multiple workshops related to HSCT and has subsequently invested in a new generation of approaches to address the most compelling needs of the field, including new sources of healthy and immunologically compatible hematopoietic stem cells for transplant; safe and efficient genome modification technologies for correction of inherited genetic defects and other forms of gene therapy; safer and more tractable transplantation procedures such as nongenotoxic conditioning regimens, methods to accelerate immune reconstitution and recovery of immune function, and innovations to minimize the risk of immune rejection; and other life‐threatening complications from transplant. This Perspective serves to highlight these needs through examples from the recent CIRM‐funded and other notable investigations, presents rationale for comprehensive, systematic, and focused strategies to unleash the full potential of HSCT, thereby enabling cures for a greatly expanded number of disorders and making HSCT feasible, accessible, and affordable to all who could benefit.
“…Recent clinical experiments using allo‐HSCT in conjunction with renal transplantation have demonstrated feasibility in some patients to achieve transplantation tolerance between donors and recipients with the removal of all post‐HSCT immunosuppressive drugs . Other strategies to induce tolerance were the subject of a CIRM Workshop and Perspective in 2015, and are discussed further there . A third general approach to address complications of tissue mismatch involves development of targeted therapies for the treatment of chronic GVHD including the in vivo expansion of endogenous regulatory T lymphocytes (Treg) with low‐dose interleukin‐2 or ex vivo expansion of Treg subsets followed by their in vivo administration …”
Section: Medical and Procedural Risks Of Hsctmentioning
Hematopoietic stem cell transplantation (HSCT) is broadly used for treating and curing hematological cancers and various disorders of the blood and immune system. However, its true therapeutic potential remains vastly constrained by significant scientific and technical hurdles that preclude expansion to new indications and limit the number of patients who could benefit from, gain access to, or financially afford the procedure. To define and overcome these challenges, the California Institute for Regenerative Medicine (CIRM) held multiple workshops related to HSCT and has subsequently invested in a new generation of approaches to address the most compelling needs of the field, including new sources of healthy and immunologically compatible hematopoietic stem cells for transplant; safe and efficient genome modification technologies for correction of inherited genetic defects and other forms of gene therapy; safer and more tractable transplantation procedures such as nongenotoxic conditioning regimens, methods to accelerate immune reconstitution and recovery of immune function, and innovations to minimize the risk of immune rejection; and other life‐threatening complications from transplant. This Perspective serves to highlight these needs through examples from the recent CIRM‐funded and other notable investigations, presents rationale for comprehensive, systematic, and focused strategies to unleash the full potential of HSCT, thereby enabling cures for a greatly expanded number of disorders and making HSCT feasible, accessible, and affordable to all who could benefit.
Ferumoxytol, approved by the U.S. Food and Drug Administration in 2009, is one of the intravenous iron oxide nanoparticles authorized for the treatment of iron deficiency in chronic kidney disease and end‐stage renal disease. With its exceptional magnetic properties, catalytic activity, and immune activity, as well as good biocompatibility and safety, ferumoxytol has gained significant recognition in various biomedical diagnoses and treatments. Unlike most existing reviews on this topic, this review primarily focuses on the recent clinical and preclinical advances of ferumoxytol in disease treatment, spanning anemia, cancer, infectious inflammatory diseases, regenerative medicine application, magnetic stimulation for neural modulation, etc. Additionally, the newly discovered mechanisms associated with the biological effects of ferumoxytol are discussed, including its magnetic, catalytic, and immunomodulatory properties. Finally, the summary and future prospects concerning the treatment and application of ferumoxytol‐based nanotherapeutics are presented.
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