Department of Neurology and Human Oncology and Pathogenesis Program, Memorial Sloan Kettering Cancer Center, New York, NY.
Department of Neurology, Ronald Reagan UCLA Medical Center, University of California, Los Angeles, CA.
J Clin Oncol. 2022 Oct 20;40(30):3510-3519. doi: 10.1200/JCO.21.02166. Epub 2022 Feb 24.
The process of developing cancer therapies is well established and has enabled the incorporation of many new drugs and classes of agents into the standard of care for common cancers. Clinical drug development is fundamentally different for rare and difficult-to-treat solid tumors, such as glioma or pancreatic cancer. The failure to develop effective new agents for the latter diseases has discouraged the development of therapeutics for these cancers. Using glioma as an example, we describe a process toward obtaining more reliable early-stage signals of drug activity and a process toward translating those signals into clinical benefits with more efficient late-stage development. If linked together, these processes should increase the likelihood of benefit in late-stage settings at a lower cost and encourage more drug development for patients with rare and difficult-to-treat cancers.
癌症治疗方法的开发过程已经成熟，并已将许多新药和药物类别纳入常见癌症的标准治疗方法中。对于罕见且难以治疗的实体肿瘤，如神经胶质瘤或胰腺癌，临床药物开发则完全不同。由于未能为后一类疾病开发出有效的新药，这些癌症的治疗方法的开发也受到了阻碍。我们以神经胶质瘤为例，描述了一种获得更可靠的药物活性早期信号的方法，以及一种将这些信号转化为临床获益的方法，从而更有效地进行晚期开发。如果将这些过程联系起来，应该可以降低成本，提高晚期治疗获益的可能性，并鼓励为患有罕见且难以治疗的癌症的患者开发更多的药物。
