Expert opinion on biological therapy
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Expert Opin Biol Ther · Feb 2020
ReviewRole of tagraxofusp in treating blastic plasmacytoid dendritic cell neoplasm (BPDCN).
Introduction: Advances and drug development in rare diseases, such as blastic plasmacytoid dendritic cell neoplasm (BPDCN), has historically been limited by small numbers of patients in the target population. In recent years, the development of tagraxofusp (SL-401) (ELZONRIS, Stemline Therapeutics) for the treatment of adult and pediatric BPDCN has been a successful story that led to US FDA approval in December 2018. ⋯ In phase I/II clinical trial, major responses were observed in 90% of treatment-naïve patients, with 72% of the responses observed as complete remissions. Limitations on the usage of tagraxofusp and strategies to handle those limitations were further explored in this review.
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Expert Opin Biol Ther · Feb 2020
Multicenter Study Observational StudyReal-life efficacy of vedolizumab on endoscopic healing in inflammatory bowel disease - A nationwide Hungarian cohort study.
Background: GEMINI trials demonstrated the therapeutic efficacy of vedolizumab (VDZ) in Crohn's disease (CD) and ulcerative colitis (UC). Research design and methods: Aim of this study was to determine the real-life effectiveness of VDZ on endoscopic healing in the Hungarian nationwide cohort of inflammatory bowel disease (IBD) patients based on the changes on clinical and endoscopic scores. Every adult IBD patient in the country (121 UC and 83 CD) who completed the short-term VDZ therapy was enrolled, of which 72 UC and 52 CD patients could complete the long-term therapy. ⋯ The average disease duration was significantly lower in CD patients achieving endoscopic healing at week 52 (11.75 vs. 5.27 years, p = 0.007). Conclusions: VDZ therapy is an effective therapeutic option in anti-TNF-α refractory IBD. However, the endoscopic healing rate was substantially lower and showed a significant delay in CD compared with UC.
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Expert Opin Biol Ther · Feb 2020
ReviewTherapeutic potential of mesenchymal stem/stromal cell-derived secretome and vesicles for lung injury and disease.
Introduction: The acute respiratory distress syndrome (ARDS) is a devastating clinical condition common in patients with respiratory failure. Based largely on numerous preclinical studies and recent Phase I/II clinical trials, administration of stem cells, specifically mesenchymal stem or stromal cells (MSC), as a therapeutic for acute lung injury (ALI) holds great promise. However, concern for the use of stem cells, specifically the risk of iatrogenic tumor formation, remains unresolved. ⋯ Expert opinion: Clinical application of MSC or their secreted CM may be limited by the cost of growing enough cells, the logistic of MSC storage, and the lack of standardization of what constitutes MSC CM. However, the clinical application of MSC EV remains promising, primarily due to the ability of EV to maintain the functional phenotype of the parent cell as a therapeutic. However, utilization of MSC EV will also require large-scale production, the cost of which may be prohibitive unless the potency of the EV can be increased.