Brain tumors are hard to treat with the currently available therapy. The major obstacle in the treatment of brain tumors is the lack of therapeutic strategies capable to penetrate the blood–brain barrier (BBB). The BBB is an endothelial interface that separates the brain from the circulatory blood system and prevents the exposure of the central nervous system (CNS) to circulating toxins and potentially harmful compounds. Unfortunately, the BBB prevents also the penetration of therapeutic compounds into the brain. We present here a drug-delivery liposomal carrier, conjugated to a peptide inserted in the liposomal membrane, which is putatively recognized by BBB transporters. The peptide is a short sequence of 5 amino acids (RERMS) present in the amyloid precursor protein (APP). This APP-targeted liposomal system was designed specifically for transporting compounds with anti-cancer activity via the BBB into the brain in an effective manner. This drug-delivery liposomal carrier loaded with the anti-cancer compounds temozolomide (TMZ), curcumin, and doxorubicin crossed the BBB in an in vitro model as well as in vivo (mice model). In the in vitro model, the targeted liposomes crossed the BBB model fourfold higher than the non-targeted liposomes. Labeled targeted liposomes penetrated the brain in vivo 35% more than non-targeted liposomes. Treatment of mice that underwent intracranial injection of human U87 glioblastoma, with the targeted liposomes loaded with the three tested anti-cancer agents, delayed the tumor growth and prolonged the mice survival in a range of 45% -70%. It appears that the targeted liposomal drug-delivery system enables better therapeutic efficacy in a SCID mouse model of glioblastoma compared to the corresponding non-targeted liposomes and the free compounds.

目前可用的疗法很难治疗脑肿瘤。脑肿瘤治疗的主要障碍是缺乏能够穿透血脑屏障（BBB）的治疗策略。 BBB 是一个内皮界面，它将大脑与循环血液系统分开，并防止中枢神经系统 (CNS) 暴露于循环毒素和潜在有害化合物。不幸的是，血脑屏障也阻止治疗化合物渗透到大脑中。我们在这里提出了一种药物递送脂质体载体，与插入脂质体膜中的肽缀合，该肽被推定被 BBB 转运蛋白识别。该肽是淀粉样前体蛋白 (APP) 中存在的 5 个氨基酸的短序列 (RERMS)。这种以 APP 为靶点的脂质体系统是专门为通过 BBB 有效地将具有抗癌活性的化合物转运到大脑而设计的。这种载有抗癌化合物替莫唑胺 (TMZ)、姜黄素和阿霉素的药物递送脂质体载体在体外模型和体内（小鼠模型）中穿过血脑屏障。在体外模型中，靶向脂质体通过 BBB 模型的能力比非靶向脂质体高四倍。标记的靶向脂质体在体内对大脑的渗透率比非靶向脂质体多 35%。用载有三种测试抗癌药物的靶向脂质体对颅内注射人U87胶质母细胞瘤的小鼠进行治疗，延缓了肿瘤的生长，并将小鼠的存活时间延长了45%-70%。 与相应的非靶向脂质体和游离化合物相比，靶向脂质体药物递送系统似乎能够在胶质母细胞瘤 SCID 小鼠模型中获得更好的治疗效果。