Valsasina Barbara, Orsini Paolo, Terenghi Chiara, Ocana Alberto
Nerviano Medical Sciences, Viale Pasteur 10, 20014 Nerviano, Italy.
Department of Pharmaceutical Sciences, University of Milan, Via Mangiagalli 25, 20133 Milan, Italy.
Pharmaceuticals (Basel). 2024 Oct 7;17(10):1338. doi: 10.3390/ph17101338.
ADCs have emerged as a promising class of therapeutics, combining the targeting specificity of monoclonal antibodies with the cytotoxic potency of small-molecule drugs. Although the majority of approved ADCs are still based on microtubule binder payloads, the recent success of topoisomerase I inhibitors has revitalized interest in the identification of novel agents overcoming present limitations in the field including narrow therapeutic window and chemoresistance. The success of DNA binders as payload for ADCs has been very limited, up to now, due, among other factors, to high hydrophobicity and planar chemical structures resulting in most cases in ADCs with a strong tendency to aggregate, poor plasma stability, and limited therapeutic index. Some of these molecules, however, continue to be of interest due to their favorable properties in terms of cytotoxic potency even in chemoresistant settings, bystander and immunogenic cell death effects, and known combinability with approved drugs. We critically evaluated several clinically tested ADCs containing DNA binders, focusing on payload physicochemical properties, cytotoxic potency, and obtained clinical results. Our analysis suggests that further exploration of certain chemical classes, specifically anthracyclines and duocarmycins, based on the optimization of physicochemical parameters, reduction of cytotoxic potency, and careful design of targeting molecules is warranted. This approach will possibly result in a novel generation of payloads overcoming the limitations of clinically validated ADCs.
抗体偶联药物(ADCs)已成为一类很有前景的治疗药物,它将单克隆抗体的靶向特异性与小分子药物的细胞毒效力结合在一起。尽管大多数已获批的ADCs仍基于微管结合剂载荷,但拓扑异构酶I抑制剂最近的成功重新激发了人们对寻找新型药物的兴趣,以克服该领域目前存在的局限性,包括治疗窗狭窄和化疗耐药性。到目前为止,DNA结合剂作为ADCs的载荷,其成功非常有限,这主要是由于高疏水性和平坦的化学结构等因素,在大多数情况下导致ADCs有强烈的聚集倾向、血浆稳定性差以及治疗指数有限。然而,其中一些分子仍备受关注,因为即使在化疗耐药的情况下,它们在细胞毒效力、旁观者效应和免疫原性细胞死亡效应方面具有良好的特性,并且已知可与已获批药物联合使用。我们严格评估了几种含有DNA结合剂的经临床测试的ADCs,重点关注载荷的物理化学性质、细胞毒效力以及获得的临床结果。我们的分析表明,基于物理化学参数的优化、细胞毒效力的降低以及靶向分子的精心设计,对某些化学类别,特别是蒽环类药物和双吖丙啶类药物进行进一步探索是有必要的。这种方法可能会产生新一代克服临床验证的ADCs局限性的载荷。