Expert opinion on biological therapy
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Expert Opin Biol Ther · Nov 2019
ReviewB cell maturation antigen (BCMA)-based immunotherapy for multiple myeloma.
Introduction: B cell maturation antigen (BCMA) contributes to MM pathophysiology and is a target antigen for novel MM immunotherapy. Complete responses have been observed in heavily pretreated MM patients after treatment with BCMA antibody-drug conjugates (ADC), chimeric antigen receptor T, and bi-specific T cell engagers (BiTE®). These and other innovative BCMA-targeted therapies transform the treatment landscape and patient outcome in MM. ⋯ Long-term follow-up and correlative studies using immuno-phenotyping and -sequencing will delineate mechanisms of overcoming the immunosuppressive MM bone marrow microenvironment to mediate additive or synergistic anti-MM cytotoxicity. Moreover, they will delineate cellular and molecular events underlying the development of resistance underlying relapse of disease. Most importantly, targeted BCMA-based immunotherapies used earlier in the disease course and in combination (adoptive T cell therapy, mAbs/ADCs, checkpoint and cytokine blockade, and vaccines) have great promise to achieve long-term disease control and potential cure.
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Expert Opin Biol Ther · Nov 2019
ReviewCaplacizumab: a change in the paradigm of thrombotic thrombocytopenic purpura treatment.
Introduction: Immune thrombotic thrombocytopenic purpura (iTTP) is an immune-mediated deficiency in von Willebrand factor-cleaving protease ADAMTS13 allowing unrestrained adhesion of von Willebrand factor multimers to platelets and microthrombosis. Caplacizumab, an anti-von Willebrand factor humanized, bivalent single-domain nanobody preventing its binding to the platelet has been investigated and approved for use in the treatment of iTTP. Areas covered: The purpose of this article is to summarize the available clinical data on the efficacy and safety of caplacizumab in iTTP and to provide our opinion on the place of caplacizumab in current treatment regimens. ⋯ It demonstrated efficacy in clinical trials through a faster platelet count normalization and protection of patients from exacerbations and refractoriness. Caplacizumab is well tolerated with minor bleeds as the most important side effect. The efficacy of caplacizumab now needs to be assessed in real-life but definitely, this drug opens hope for a significant improvement in iTTP prognosis at the very early, critical step of the disease.
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Expert Opin Biol Ther · Nov 2019
Review Comparative StudyChimeric antigen receptor T-cell therapy for the treatment of aggressive B-cell non-Hodgkin lymphomas: efficacy, toxicity, and comparative chimeric antigen receptor products.
Introduction: Traditionally, outcomes for patients with relapsed/refractory aggressive B-cell non-Hodgkin lymphoma have been poor. There has been a clear need for effective therapeutic options that could produce durable remissions with a reasonable safety profile. The approval of chimeric antigen receptor (CAR) T-cell therapies has been revolutionary in the field because CAR T-cells meet this need for a substantial number of patients. ⋯ Mechanisms of resistance, current unanswered questions in the field, issues associated with choosing a CAR T-cell product, and future directions for the advancement of CAR T-cell therapy. Expert Opinion: Due to differences in study populations and manufacturing times, it is too early to know if there is a 'best' choice for CAR T-cell therapy. Decisions must be individualized taking into account patient factors and expected toxicity.