Micallef Benjamin, Nisticò Robert, Sarac Sinan B, Bjerrum Ole W, Butler Dianne, Sammut Bartolo Nicolette, Serracino-Inglott Anthony, Borg John Joseph
Malta Medicines Authority, Sir Temi Żammit Buildings, Malta Life Sciences Park, San Ġwann SĠN 3000, Malta.
School of Pharmacy, Department of Biology, University of Tor Vergata, Rome, Italy.
Drug Discov Today. 2022 May;27(5):1483-1494. doi: 10.1016/j.drudis.2022.01.002. Epub 2022 Jan 7.
New paediatric acute lymphoblastic leukaemia (ALL) treatments have been developed and innovative products are in the pipeline. However, despite many active clinical trials, bridging bench science to clinical development to authorised medicines remains challenging. Research in first-line treatment continues to focus on multidrug chemotherapy with the potential addition of new targeted molecules being studied. Research in second- and third-line treatment represents a shift from cytotoxic intensification to an area of precision medicine through emergent innovative and immuno-oncology products. The collaborative research model in ALL involving different stakeholders should intensify to facilitate bench-to-bedside clinical translation for the benefit of patients.
新的儿童急性淋巴细胞白血病（ALL）治疗方法已经研发出来，创新产品也在筹备中。然而，尽管有许多正在进行的临床试验，但将基础科学与临床开发转化为获批药物仍然具有挑战性。一线治疗的研究仍集中在多药化疗上，同时也在研究添加新的靶向分子的可能性。二线和三线治疗的研究正从强化细胞毒性治疗转向通过新兴的创新和免疫肿瘤学产品进入精准医学领域。ALL领域涉及不同利益相关者的合作研究模式应得到加强，以促进从实验室到临床的转化，造福患者。
