我想向中华慈善总会求援，需要大家的帮助

唯心 · 发表于 2011-5-8 23:45:57

希望能成功！

唯心 · 发表于 2011-5-8 23:46:50

希望能成功！

webslave · 发表于 2011-5-11 10:12:07

我在肝胆相照的论坛里，相同的帖子，bjjww回复了一些非常有用的帖子，教大家怎么个人申请国外用药，有兴趣的可以先读一下，慈善总会一直没有消息，怕是希望不大，大家看看自己能不能申请用药，我现在主要是有些担心此药的疗效，它的一个靶点VEGF和索拉菲尼是一样的，区别是一个不让VEGF生成，一个是生成此细胞因子后消灭，另一个靶点是控制有丝分裂的，这个是我最担心的，因为如果抑制了骨髓制造白细胞，血小板等经常有丝分裂的细胞，那就和化疗药物从结果上没有太多区别，他们的解释是通过低密度脂让肝脏吸收，也就是他们认为找到了高效介导的办法，但是结果如何还要看他们提交的报告。而且参加实验，也有双盲的问题，所以我现在也是不知道如何是好呢。

Michaelfung · 发表于 2011-5-11 16:31:04

6月3日--7日，ASCO开会，到时候会公布ALNYLAM公司的一期临床试验的结果。我们拭目以待

lymxx1981 · 发表于 2011-5-13 12:46:49

回复 webslave 的帖子

你的邮箱我没法发

为母亲祈祷 · 发表于 2011-5-17 21:08:49

我也申请，我妈肝癌晚期，双肺转移，肾上腺占位我的电话 134-5529-6868

Michaelfung · 发表于 2011-6-8 10:00:19

阿尔尼拉姆公司在美国临床肿瘤协会年会上，公布了肝癌的一期临床实验结果，一半的病人12/24,表现稳定SD或以上。

webslave · 发表于 2011-6-12 15:52:50

本帖最后由 webslave 于 2011-6-13 21:00 编辑

好像说是剂量大于0.7毫克/公斤的对药物有响应的病人，大概50%有效，还是很失望，很像另一种多吉美，而不是能根治性的药物。
阿尔尼拉姆提交的给asco的报告，31个病人中，27个对药物有响应，其中12个剂量低于0.4的1人至少2个月稳定期SD,15个大于0.7的，6个人SD，一个人部分缓解。临床没显示药物毒性。
下面是报告摘要。
Phase I dose-escalation study of ALN-VSP02, a novel RNAi therapeutic for solid tumors with liver involvement.

Abstract:

Background: Malignancies involving the liver represent a significant unmet medical need. ALN-VSP02 is a novel RNA interference (RNAi) therapeutic comprised of lipid nanoparticle-formulated small interfering RNAs (siRNAs) targeting the expression of vascular endothelial growth factor (VEGF)-A and kinesin spindle protein (KSP).
Methods: A multicenter, open label, phase 1 dose escalation trial of ALN-VSP02 administered as a 15-minute iv infusion every two weeks was initiated in March 2009. Patients with advanced solid tumors are eligible if they have at least one measurable liver lesion and adequate liver function. The study uses a 3+3 design with 8 potential dose levels: 0.1, 0.2, 0.4, 0.7, 1.0, 1.25, 1.5, and 1.7 mg/kg. The primary objective is evaluation of safety and tolerability. Secondary objectives include assessment of PK and pharmacodynamic (PD) activity through DCE-MRI, biomarkers of angiogenesis, tumor biopsies, and response rate.
Results: As of December 2009, 12 patients (most with colorectal cancer) have been treated on the first 4 dose levels. Forty-one doses have been administered; 0.1-0.4 mg/kg ALN-VSP02 was well- tolerated, with no hepatotoxicity. The only significant adverse event (AE) was a grade 2 infusion reaction in one patient at 0.4 mg/kg that responded to slowing of the infusion. At 0.7 mg/kg, a patient with pancreatic neuroendocrine tumor extensively involving both lobes of the liver died of hepatic failure following the second dose; this was deemed possibly related to study drug. Two additional patients treated at 0.7 mg/kg did not exhibit hepatotoxicity or any other significant AEs. PK showed Cmax and AUC that were dose proportional with no accumulation. Serial DCE-MRI scans performed 2-9 days after the first dose on 8 of the 12 patients showed a ≥40% decline in Ktrans in 12 of 15 liver tumors (80%) evaluated. This decline in blood flow was associated with extensive tumor necrosis in the patient with the neuroendocrine tumor.
Conclusions: ALN-VSP02 was well-tolerated by the majority of patients across the first 4 dose levels. DCE-MRI results show preliminary evidence of an anti-VEGF effect. Accrual is continuing, and additional safety and PD data will be forthcoming.