Approaches for Effective Clinical Application of Stem Cell Transplantation
AbstractPurpose of ReviewThis review highlights problems related to translation of advanced therapy medicinal products (ATMPs) from bench to bedsite. Regenerative medicine within the current regulatory frame reveals common hitches in the course of development, translation, and clinical application. This paper suggests outlining a path from the few examples of successfully approved vs unsuccessful advanced therapies.Recent FindingsIn the multitude of ongoing studies, few of them achieved positive results with a final treatment available to patients; this result was possible due to multidisciplinary teams working together from the beginning of the development and during the hard route to standardization and clinical application.SummaryThe root of success of an advanced therapy requires not only the inescapable scientific and biological knowledge but also requires several contributions as regulatory, ethical, medical, and bio-engineering expertise, from the real beginning. A strong scientific rationale and an integrated network of expertises would contribute to a successful investment of available resources in advanced therapy medicinal products and to a greater confidence in future medicine.
Abstract The transplantation of neural stem cells (NSCs) has become an emerging treatment for neural degeneration. A key factor in such treatments is to manipulate NSC behaviors such as proliferation and differentiation, resulting in the eventual regulation of NSC fate. Novel bionanomaterials have shown usefulness in guiding the proliferation and differentiation of NSCs due to the materials' unique morphological and topological properties. Among the nanomaterials, graphene has drawn increasing attention for neural regeneration applications based on the material's excellent physicochemical properties, surface modif...
Conditions: Hematologic Diseases; Chronic Myeloid Leukemia; Acute Myeloid Leukemia; Acute Lymphoid Leukemia; Myelodysplastic Syndromes; Non Hodgkin Lymphoma; Stem Cell Transplant Complications; Chronic Lymphocytic Leukemia Intervention: Other: No intervention Sponsor: Rigshospitalet, Denmark Not yet recruiting
Condition: Graft Versus Host Disease (GVHD) Interventions: Biological: Alpha-1 antitrypsin (AAT); Drug: Placebo Sponsors: CSL Behring; Blood and Marrow Transplant Clinical Trials Network; National Institutes of Health (NIH); National Heart, Lung, and Blood Institute (NHLBI); National Cancer Institute (NCI) Not yet recruiting
Publication date: Available online 18 November 2019Source: The Journal of Molecular DiagnosticsAuthor(s): Ya-Zhen Qin, Yu Wang, Lan-Ping Xu, Xiao-Hui Zhang, Xiao-Su Zhao, Kai-Yan Liu, Xiao-Jun HuangHigh WT1 expression after allogeneic hematologic stem cell transplantation (allo-HSCT) can strongly predict relapse in acute myeloid leukemia (AML). However, the cutoff values obtained were inconsistent. The precise cutoff values may be optimized through subtype analysis, and the RUNX1-RUNX1T1 fusion transcript provides an ideal reference. RUNX1-RUNX1T1 and WT1 transcript levels were simultaneously measured in 1,299 bone marrow ...
ConclusionsMSCs transplantation proved to be a safe and tolerable therapy. Their potential therapeutic benefits were also validated. However, larger placebo controlled blinded clinical trials will be required to establish the long term safety and efficacy profile of these therapies for MS. Their translation into the clinical practice can provide a new hope for the patients of this highly debilitating disease.
In this study, around 20% of all patients with HBV reactivation developed HBV reactivation after 2 years from the initiation of therapy, unlike malignant lymphoma. This might be due to the fact that almost all of the patients received chemotherapy for a long duration. Therefore, a new strategy for the prevention of HBV reactivation in MM patients is required.
Bone Marrow Transplantation, Published online: 19 November 2019; doi:10.1038/s41409-019-0746-3Predictors of the trajectory of cognitive functioning in the first 6 months after allogeneic hematopoietic stem cell transplantation
Allogeneic hematopoietic stem cell transplantation (allo-SCT) is the most established and commonly used cellular immunotherapy in cancer care. It is the most potent anti-leukemic therapy in patients with acute myeloid leukemia (AML) and is routinely used with curative intent in patients with intermediate and poor risk disease. Donor T cells, and possibly other immune cells, eliminate residual leukemia cells after prior (radio)chemotherapy. This immune-mediated response is known as graft-versus-leukemia (GvL). Donor alloimmune responses can also be directed against healthy tissues, which is known as graft-versus-host diseas...
Conclusion: In a representative Brazilian population of T1DM patients, those with IDAA1c ≤ 9 presented a lower frequency of MC, as well as fewer episodes of hypoglycemia, in the month prior to the analysis.
ConclusionsAn improvement in efficacy could not be observed after modified DHAP regimen; however, manageable toxicity and reduced renal complications suggest further investigation. The study, however, also underlines the need for new concepts in the management of advanced and high-risk lymphomas.