Richon Victoria M
1Sanofi Oncology, Cambridge, MA.
Hematology Am Soc Hematol Educ Program. 2013;2013:19-23. doi: 10.1182/asheducation-2013.1.19.
Over the past decade, the number of new therapies developed for the treatment of rare diseases continues to increase. The most rapid growth has been in the development of new drugs for oncology indications. One focus in drug discovery for oncology indications is the development of targeted therapies for select patient subgroups characterized by genetic alterations. The identification of these patient subgroups has increased in the past decade and has resulted in a corresponding increase in the development of new drugs for genetically defined patient subgroups. As an example of the development of new therapeutics for rare indications, I describe here the drug discovery efforts leading to the development of DOT1L inhibitors for the treatment of MLL-rearranged leukemia.
在过去十年中，用于治疗罕见病的新疗法数量持续增加。增长最为迅速的是针对肿瘤学适应症的新药研发。肿瘤学适应症药物研发的一个重点是为以基因改变为特征的特定患者亚组开发靶向疗法。在过去十年中，这些患者亚组的识别有所增加，相应地，针对基因定义的患者亚组的新药研发也有所增加。作为罕见适应症新疗法研发的一个例子，我在此描述了导致开发用于治疗MLL重排白血病的DOT1L抑制剂的药物研发工作。
