相关疾病：
值得一读。
Why such success? Is it a pure coincidence? Or has China something to offer that other
countries don’t have?
（在USA，EU都失败的东西）为什么在中国成功呢？纯属巧合吗？还是中国提供了其他国家不曾提供的便利呢？One reason why experimental therapies have reached the market in China, but languished in the West, is cultural.in the United States, treatments, such as gene therapy, have been stigmatized by highly publicized failures like the death of Jesse Gelsinger during a trial at the University of Pennsylvania; this stigma does not exist in China.
原因一：文化。Jesse Gelsinger Case对于美国人接受生物疗法心理上存在阴影。
Jesse Gelsinger Case：一个基因治疗失败的案例，Jesse Gelsinger，18岁，男性，患有轻度鸟氨酸转移酶缺乏症（OTC），该症是由于代谢紊乱影响到氨的降解，但利用药物治疗和低蛋白饮食可使疾病得到控制。1999年9月，Gelsinger自愿参加针对OTC缺乏症的基因治疗的I期临床试验。结果对病毒载体产生严重免疫反应，后死于多器官功能衰竭。美国宾州大学人类基因治疗研究所进行的这项临床试验，是研究对患有致命的OTC缺乏症的婴儿的基因治疗方法。而这一I期临床试验对Gelsinger并没有预期的利益。
Another possible key factor for success is the ability of Chinese firms to easily recruit
patients.
原因二：中国容易招募到病人。
“All three cases are a result of the heavy, even desperate, government investments,” Medgenn receives most of the RMB100 ($12.4) million research funding from various levels of government in the coastal province Shandong, one of the richest in China. Similarly, （深圳）Sibiono receives nearly all of its RMB80 ($9.6) million research funding from government bodies, public foundations and major universities.
（不知道是不是）原因三：政府从纳税人手里凑了一大笔钱，总不能打水漂吧？（是原文的最后一段，我觉得是原因三）
Are the successes of these Chinese companies likely to make the country more attractive to US firms？
既然中国的生物药物公司取得这么多成功（2003年3月深圳的Sibiono，2005年9月烟台的Medgenn Co.，2005年11月上海的Sunway都有新药上市），是否会吸引美国的公司到中国来呢？
US companies, however, would still not necessarily opt for the Chinese approval system. For example, Introgen intends to sell its gene therapy products throughout the world, but the company has no intention of moving its testing to China or anywhere else to speed development. “We’re very focused on creating the global standard for this new class of products, and that’s going to be defined in the US and in Europe”
结论是：（尽管中国对生物药容易上市）旦人家不会选择在中国上市的。人家关注的是符合US，EU框价下的新药上市标准（全球标准）。
补充一点：
Intellectual property (IP) could be a factor limiting export of the technology to the US
market
另外谈到今后出口，可能引起知识产权的纠纷 作者: bluelake 时间: 2014-10-9 11:15
三期临床的结果
Results of phase III trial of rh-endostatin (YH-16) in advanced non-small cell lung cancer (NSCLC) patients
Y. Sun, J. Wang, Y. Liu, X. Song, Y. Zhang, K. Li, Y. Zhu, Q. Zhou, L. You and C. Yao
Cancer Hosp Chinese Acad of Medcl Science, Beijing, China; Liaoning Provincial Cancer Hosp, Shenyang, China; Cancer Hosp, Guangxi Medcl Univ, Nanning, China; Zhejiang Provincial Cancer Hosp, Hangzhou, China; Tianjin Cancer Hosp, Tianjin, China; Thoracic Tumor Hosp, Beijing, China; West China Hosp, Sichun Univ, Chengdu, China; Medgenn Bioengeering Co., Yantai, China; Beijing Univ First Hosp, Beijing, China
7138
Background: Endostar (rh-endostatin, YH-16) is a new recombinant human endostatin developed by Medgenn Co. Ltd. Pre-clinical study indicates that it can inhibit tumor endothelial cell proliferation, angiogenesis, and tumor growth. Phase I and Phase II studies revealed that Endostar was effective as single agent with good tolerance in clinical use. This study is to compare the response rate, median time to progression (TTP), clinical benefit, and safety in patients with advanced NSCLC, treated with Endostar plus vinorelbine and cisplatin (NP) or placebo plus NP. Methods: 493 histology or cytology confirmed stage IIIB and IV NSCLC patients, life expectancy >3 months, and WHO performance status 0–2 patients were entered in a double-blind, placebo-controlled, prospectively randomized trial after stratification, either Arm A: NP plus Endostar (n=326; vinorelbine 25mg/m2 on day 1 and 5, cisplatin 30mg/ m2 on days 2 to 4, Endostar 7.5 mg/ m2 on days 1 to 14) or Arm B: NP plus placebo (n=167; vinorelbine 25mg/m2 on day 1 and day5, cisplatin 30mg/ m2 on days 2 to 4, 0.9% sodium-chloride 3.75 ml on days 1 to 14) every 3 weeks for 2–6 cycles, according to the response after the second cycles, i.e. CR,PR,SD continue to receive treatment; while PD stop treatment. The response and adverse effects were evaluated by an Independent Experts Evaluation Committee (IEEC) before open the code. Results: Of the 486 assessable patients, overall response rates were 35.4% in Arm A and 19.5% in Arm B (P=.0003). The median TTP were 6.3 and 3.6 months for Arm A and B respectively (P<.001). The clinical benefit rates were 73.3% in Arm A and 64.0% in Arm B (P=.035). Grade 3/4 neutropenia, anemia, nausea/vomiting were 28.5%, 3.4%, 8.0% respectively in Arm A, as compared with 28.2%, 3.0%, 6.6% in Arm B(P>.05). There were 2 treatment-related death in Arm A and 1 in Arm B (P>.05). Conclusion: The addition of Endostar to NP regimen resulted in significant and clinically meaningful improvement in response rate, median TTP, and clinical benefit rate compared with NP alone in advanced NSCLC patients. Recombinant rh-Endostatin in combination with chemotherapy showed a synergic activity and a favorable toxic profile in advanced cancer patients.作者: dodoit 时间: 2014-10-9 11:41
Journal of the National Cancer Institute, Vol.98, No.11,June 7,2006
Endostatin: Are We Waiting For Godot? 一文谈到了Endostar比Endostatin好的可能的原因:引用的是Folkman的话:
1.氨基的改变是使得水溶性更好,使得在血中的时间呆的更长了,病人一天一次给药改为了几天一次.
2.中国科学家在三期的临床中合用了传统的中药,这种联合用药可能是这种成功的关键.
个人一些看法:
Endostar和中国以前的生物技术一类新药一样,只是在一些氨基上做了修饰,没有真正改变其骨架.这些所谓的一类新药,其实骨子里还是仿制药.因为,生物药物不像化学药一样,改变几个官能团就可能无效了.当然,可能就像前面讲的提高了水溶性等原因,使得生物利用度提高了很多.
不可否认,能在这个方面做那么多的工作需要很大的勇气和执着的.但是,中国最缺的还是像Folkman这样的科学家,像他那样所做的工作,因为这样才是真正的创新.作者: dodoit 时间: 2014-10-9 14:30
强烈建议大家读一读nature Group旗下的nature biotechnology杂志,
volume 24,number 2,february 2006,篇名"china offers alternative gateway for experimental drugs".看了之后一定让你明白所谓国家一类抗癌新药恩度的真相.作者: 轰轰 时间: 2014-10-9 14:31
强烈建议大家读一读nature Group旗下的nature biotechnology杂志,
volume 24,number 2,february 2006,篇名"china offers alternative gateway for experimental drugs".看了之后一定让你明白所谓国家一类抗癌新药恩度的真相.
