Purpose of Review

Acute lymphoblastic leukemia (ALL) is a widely heterogeneous disease in terms of genomic alterations, treatment options, and prognosis. While ALL is considered largely curable in children, adults tend to have higher risk disease subtypes and do not respond as favorably to conventional chemotherapy. Identifying genomic drivers of leukemogenesis and applying targeted therapies in an effort to improve disease outcomes is an exciting focus of current ALL research. Here, we review recent updates in ALL targeted therapy and present promising opportunities for future research.

Recent Findings

With the utilization of next-generation sequencing techniques, the genomic landscape of ALL has greatly expanded to encompass novel subtypes characterized by recurrent chromosomal rearrangements, gene fusions, sequence mutations, and distinct gene expression profiles. The evolution of small molecule inhibitors and immunotherapies, and the exploration of unique therapy combinations are some examples of recent advancements in the field.

Summary

Targeted therapies are becoming increasingly important in the treatment landscape of ALL to improve outcomes and minimize toxicity. Significant recent advancements have been made in the detection of susceptible genomic drivers and the use of novel therapies to target them.

中文翻译：

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ALL 中的新型生物标志物和分子靶标

审查目的

急性淋巴细胞白血病 (ALL) 是一种在基因组改变、治疗选择和预后方面存在广泛异质性的疾病。虽然儿童 ALL 被认为在很大程度上是可以治愈的，但成人往往具有较高风险的疾病亚型，并且对传统化疗的反应不佳。识别白血病发生的基因组驱动因素并应用靶向治疗以改善疾病结果是当前 ALL 研究的一个令人兴奋的焦点。在这里，我们回顾了所有靶向治疗的最新进展，并为未来的研究提供了有希望的机会。

最近的发现

随着新一代测序技术的应用，ALL 的基因组图谱已大大扩展，涵盖了以反复染色体重排、基因融合、序列突变和独特基因表达谱为特征的新亚型。小分子抑制剂和免疫疗法的演变以及独特治疗组合的探索是该领域最新进展的一些例子。

概括

靶向治疗在 ALL 的治疗领域中变得越来越重要，以改善结果并最大限度地减少毒性。最近在易感基因组驱动因素的检测和针对它们的新疗法的使用方面取得了重大进展。