Comparable Outcomes of Pre- Versus Post-Tyrosine Kinase Inhibitor Era Treatment in Chronic Myeloid Leukemia: A Retrospective Cohort Study With Long-term Follow-up.

Imatinib, a selective BCR-ABL tyrosine kinase inhibitor (TKI), was introduced after allogeneic hematopoietic stem cell transplantation (allo-HSCT) for patients with chronic myeloid leukemia (CML). However, the long-term effects of allo-HSCT in chronic phase CML patients are mostly unknown. We retrospectively analyzed the outcomes of 204 patients with sibling donors who received peripheral stem cells and underwent allo-HSCT of chronic phase I (CP1) in the pre- and post-TKI era at Shariati Hospital in Tehran, Iran, from 1998 to 2017 and followed up till the end of 2021. The median follow-up time for all patients was 8.7 (SD = 0.54) years. Fifteen-year overall survival (OS), disease-free survival (DFS), graft-versus-host disease-free relapse-free survival (GRFS), relapse, and non-relapse mortality (NRM) incidence were 65.70%, 57.83%, 17.56%, 13.17%, and 28.98%, respectively. Using multivariable analyses, the only risk factor increasing the hazard of death was the time between diagnosis to allo-HSCT greater than 1 year compared to this time less than 1 year by 74% [hazard ratio (HR) = 1.74, P = 0.039]. Also, age is a significant risk factor for DFS (HR = 1.03, P = 0.031). Our findings suggested that allo-HSCT is still an important treatment option for CP1 patients, especially those resistant to TKI treatment. TKI consumption can have a desirable effect on NRM after allo-HSCT for CP1 CML.

Gene therapy clinical trials, where do we go? An overview.

There have been many ups and downs since the introduction of gene therapy as a therapeutic modality for diseases. However, the journey of gene therapy has reached a fundamental milestone, as evidenced by the increasing number of gene therapy products on the market. Looking at the currently approved and under-approval products, as well as the numerous clinical trials in this field, gene therapy has a promising future. Trend of changes in gene therapy strategies, vectors, and targets could be insightful for pharmaceutical companies, policymakers, and researchers. In this paper, following a brief history of gene therapy, we reviewed current gene therapy products as well as gene therapies that may be approved in the near future. We also looked at ten-year changes in gene therapy clinical trials strategies, such as the use of vectors, target cells, transferred genes, and ex-vivo/in-vivo methods, as well as the major fields that gene therapy has entered. Although gene therapy was initially used to treat genetic diseases, cancer now has the greatest number of gene therapy clinical trials. Changes in gene therapy strategies, particularly in pioneering countries in this field, may point to the direction of future clinical products.

Antigenic targets of CAR T Cell Therapy. A retrospective view on clinical trials.

Chimeric antigen receptor (CAR) T cell therapy is anticipated to be increasingly implemented in the context of cancer treatment after two current FDA approval of anti-CD19 CAR-T cells (Kymriah™ & Yescarta™). The success of CD19 is mainly attributable to the proper selection of the antigen, CD19, as the target of the disease, highlighting the importance of target selection for other CAR therapies. Therefore, here we performed a global analysis of targets that are the prime focus for various CAR T cell therapies in human clinical trials.