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A clinical trial involves the study of the safety, efficacy and/or dosage regimen of a therapeutic intervention (such as a drug) in humans selected according to predetermined criteria of eligibility (such as a defined severity of a specific disease), who are observed for predefined evidence of favourable and unfavourable effects.
In this phase 1 trial, Hegde et al. treat 13 individuals with advanced sarcoma with lymphodepletion followed by HER2-specific CAR T cells, which were found to be safe and showed antitumor activity.
CAR-T cell therapies targeting BCMA have shown promising responses in patients with multiple myeloma (MM), however primary resistance and relapse are frequently observed. Here the authors report the results of a phase I//II study of bispecific CAR T-cells targeting BCMA and CD19 in relapsed/refractory MM.
An argument framework, grounded in the sciences of reasoning, provides an alternative to medicine’s measurement framework for evaluating and synthesizing evidence in healthcare.
An mRNA-based drug aims to replace a faulty enzyme and restore metabolic function in children with propionic acidemia — with encouraging early clinical results.
In a multicenter clinical trial, patients with early-stage Parkinson’s disease treated with lixisenatide, a drug currently used for the treatment of diabetes, showed improvement in their motor scores compared with those on placebo.
