Lee Hyun-Ju, Kang Sora, Jeong Yoo Joo, Park Jin-Hee, Hwang Jeong-Woo, Gu Chan-Hu, Jung Tae-Mi, Oh Seokjun, Jang Ji-Yeong, Hoe Hyang-Sook
Department of Neural Development and Disease, Korea Brain Research Institute (KBRI), 61, Cheomdan-ro, Daegu, 41062, Republic of Korea.
AI-based neurodevelopmental diseases digital therapeutics group, Korea Brain Research Institute (KBRI), 61, Cheomdan-ro, Daegu, 41062, Republic of Korea.
Mol Brain. 2025 Jul 21;18(1):65. doi: 10.1186/s13041-025-01225-7.
We previously demonstrated that ibrutinib has therapeutic efficacy against AD pathologies when injected intraperitoneally at a lower dosage (10 mg/kg, daily for 2 weeks) or orally at a higher dosage (30 mg/kg, daily for 1 month) in AD mice models. However, the effect of chronic lower dose of ibrutinib by oral administration on AD pathologies has not been investigated yet. Therefore, we investigated whether long-term oral administration of ibrutinib at a lower dose (1 or 10 mg/kg, daily for 5 months) on AD pathology and in vivo toxicity in 5xFAD mice. We found ibrutinib enhanced cognitive function and alleviated Aβ pathology in 5xFAD mice without hepatotoxicity. Furthermore, ibrutinib-treated 5xFAD mice decrease tau hyperphosphorylation, p-GSK3α/β levels, and markers of neuroinflammation such as Iba-1, GFAP, and NLRP3. Collectively, these translational studies indicate chronic oral administration of ibrutinib at low doses improves cognitive function and suppresses AD pathology/neuroinflammation in an AD mice model thereby having potential as an effective multitarget AD therapeutic in clinical application.
我们之前证明,在AD小鼠模型中,当以较低剂量(10毫克/千克,每日一次,共2周)腹腔注射或较高剂量(30毫克/千克,每日一次,共1个月)口服时,依鲁替尼对AD病理具有治疗效果。然而,口服慢性低剂量依鲁替尼对AD病理的影响尚未得到研究。因此,我们研究了在5xFAD小鼠中,长期口服低剂量(1或10毫克/千克,每日一次,共5个月)依鲁替尼对AD病理和体内毒性的影响。我们发现依鲁替尼增强了5xFAD小鼠的认知功能,减轻了Aβ病理,且无肝毒性。此外,经依鲁替尼治疗的5xFAD小鼠降低了tau蛋白过度磷酸化、p-GSK3α/β水平以及神经炎症标志物如Iba-1、GFAP和NLRP3。总的来说,这些转化研究表明,在AD小鼠模型中,长期口服低剂量依鲁替尼可改善认知功能并抑制AD病理/神经炎症,因此在临床应用中具有作为有效的多靶点AD治疗药物的潜力。