Cell-replacement therapy has emerged during the past decade as a potential solution for many diseases. However, for this promise to be fulfilled, numerous process development challenges specific to these products need to be overcome. This editorial overview highlights some key observations derived from research on an allogeneic somatic cell therapy product for the treatment of Parkinson's disease.
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CD7 CART Therapy Bridging Allo-HSCT Remarkably Improves Long-Term DFS in Refractory/Relapsed T-ALL/LBL.
Transplant Cell Ther
November 2024
Department of Bone Marrow Transplantation, Beijing Gobroad Boren Hospital, Beijing, China. Electronic address:
T-ALL is caused by abnormal proliferation of T cells. It comprises 25%-50% of ALL cases in children and adults. Outlook for R/R T-ALL/LBL and patients over 60 is even dimmer.
View Article and Find Full Text PDFClonal dynamics after allogeneic haematopoietic cell transplantation.
Nature
November 2024
Cancer, Ageing and Somatic Mutation Programme, Wellcome Sanger Institute, Hinxton, UK.
Article Synopsis
- Allogeneic hematopoietic cell transplantation (HCT) involves replacing a recipient's blood stem cells with those from a donor, and this study analyzed the long-term effects on stem cell populations over 9-31 years post-transplant.
- Researchers sequenced genomes from nearly 3,000 single-cell-derived blood colonies from ten donor-recipient pairs and found that younger donors contributed significantly more engrafted stem cells compared to older donors, leading to differences in blood cell types produced.
- The study identified that recipients experienced a decrease in clonal diversity, akin to accelerated aging, due to two types of selection processes: one occurring in the donor before the transplant and another in the recipient's marrow after engraftment, which revealed
A phase 2, multicenter, clinical trial of CPX-351 in older patients with secondary or high-risk acute myeloid leukemia: PETHEMA-LAMVYX.
Cancer
January 2025
Department of Hematology, Hospital Universitari i Politècnic La Fe, Valencia, Spain.
Background: LAMVYX was a multicenter, single-arm, phase 2 trial designed to validate the safety and efficacy of CPX-351 in patients aged 60-75 years with newly diagnosed, secondary acute myeloid leukemia and to generate evidence on key issues not addressed in the preceding regulatory pivotal trial.
Methods: The primary end point of the study was the complete remission (CR)/CR with incomplete hematologic recovery (CRi) rate after induction. Eligible patients were recommended to undergo allogeneic hematopoietic stem cell transplantation after the first consolidation cycle.
VEXAS syndrome: an adult-onset autoinflammatory disorder with underlying somatic mutation.
Curr Opin Rheumatol
January 2025
University Hospital Eppendorf, Department Internal Medicine III, Division of Rheumatology and Inflammatory Systemic Autoimmune Diseases, Hamburg.
Article Synopsis
- VEXAS syndrome is a recently identified condition that affects primarily men over 50, with a focus on its genetic link to mutations in the UBA1 gene and unexplained inflammation.
- Recent treatments include Janus-Kinase inhibitors like Ruxolitinib and Azacitidine, which show promise in managing symptoms, though the disease has a high mortality rate primarily due to infections.
- The review emphasizes the importance of improving knowledge on VEXAS through updated research, diagnostic criteria, and enrolling patients in clinical trials to enhance treatment outcomes.
Influence of the Bone Marrow Microenvironment on Hematopoietic Stem Cell Behavior Post-Allogeneic Transplantation: Development of Clonal Hematopoiesis and Telomere Dynamics.
Int J Mol Sci
September 2024
Catholic Genetic Laboratory Center, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul 06591, Republic of Korea.
Article Synopsis
- Allogeneic hematopoietic stem cell transplantation (allo-HSCT) shows promise as a cure for myelodysplastic neoplasms (MDSs) and other blood cancers.
- A study of 21 MDS patients post-transplantation revealed 38% developed new genetic mutations, indicating clonal hematopoiesis (CH) from donor cells and a higher incidence of CH compared to healthy individuals.
- Additionally, telomere length in these patients shortened significantly, suggesting stress and rapid cell division in the new bone marrow environment may accelerate genetic changes.
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