Department of Integrative Biology and Pharmacology, The University of Texas Health Science Center at Houston, Houston, Texas.
The Vivian L. Smith Department of Neurosurgery, The University of Texas Health Science Center at Houston, Houston, Texas.
Cancer Res. 2021 Feb 15;81(4):885-897. doi: 10.1158/0008-5472.CAN-19-3219. Epub 2020 Dec 22.
Cancer-associated cachexia, characterized by muscle wasting, is a lethal metabolic syndrome without defined etiology or established treatment. We previously found that p300 mediates cancer-induced muscle wasting by activating C/EBPβ, which then upregulates key catabolic genes. However, the signaling mechanism that activates p300 in response to cancer is unknown. Here, we show that upon cancer-induced activation of Toll-like receptor 4 in skeletal muscle, p38β MAPK phosphorylates Ser-12 on p300 to stimulate C/EBPβ acetylation, which is necessary and sufficient to cause muscle wasting. Thus, p38β MAPK is a central mediator and therapeutic target of cancer-induced muscle wasting. In addition, nilotinib, an FDA-approved kinase inhibitor that preferentially binds p38β MAPK, inhibited p300 activation 20-fold more potently than the p38α/β MAPK inhibitor, SB202190, and abrogated cancer cell-induced muscle protein loss in C2C12 myotubes without suppressing p38α MAPK-dependent myogenesis. Systemic administration of nilotinib at a low dose (0.5 mg/kg/day, i.p.) in tumor-bearing mice not only alleviated muscle wasting, but also prolonged survival. Therefore, nilotinib appears to be a promising treatment for human cancer cachexia due to its selective inhibition of p38β MAPK. SIGNIFICANCE: These findings demonstrate that prevention of p38β MAPK-mediated activation of p300 by the FDA-approved kinase inhibitor, nilotinib, ameliorates cancer cachexia, representing a potential therapeutic strategy against this syndrome.
癌症相关性恶病质以肌肉消耗为特征,是一种致命的代谢综合征,其病因不明,也没有既定的治疗方法。我们之前发现,p300 通过激活 C/EBPβ 介导癌症引起的肌肉消耗,C/EBPβ 继而上调关键的分解代谢基因。然而,激活 p300 以响应癌症的信号机制尚不清楚。在这里,我们表明,在骨骼肌肉中 Toll 样受体 4 被癌症激活后,p38β MAPK 将 p300 上的丝氨酸 12 磷酸化,以刺激 C/EBPβ 乙酰化,这对于引起肌肉消耗是必要且充分的。因此,p38β MAPK 是癌症引起的肌肉消耗的中央介质和治疗靶点。此外,nilotinib 是一种经美国食品和药物管理局批准的激酶抑制剂,优先与 p38β MAPK 结合,比 p38α/β MAPK 抑制剂 SB202190 更有效地抑制 20 倍的 p300 激活,并在不抑制 p38α MAPK 依赖性肌发生的情况下消除 C2C12 肌管中的癌细胞诱导的肌肉蛋白损失。在荷瘤小鼠中以低剂量(0.5 mg/kg/天,腹腔内)全身性给予 nilotinib 不仅缓解了肌肉消耗,而且延长了生存期。因此,由于其对 p38β MAPK 的选择性抑制,nilotinib 似乎是治疗人类癌症恶病质的一种很有前途的方法。
这些发现表明,通过美国食品和药物管理局批准的激酶抑制剂 nilotinib 预防 p38β MAPK 介导的 p300 激活可改善癌症恶病质,代表了针对该综合征的潜在治疗策略。