患者出现副作用 降胆固醇基因疗法试验暂停

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患者出现副作用 降胆固醇基因疗法试验暂停




基因编辑药物的示意图。(Shutterstock)






（大纪元记者林达编译报导）据彭博社报道，由于患者出现奇怪的副作用，实验性生物技术初创公司Verve Therapeutics暂停了处于第一阶段的一项热门的人类基因编辑疗法试验。


这项被称为“Heart-1”的试验是，尝试使用基因编辑来降低胆固醇——家族性高胆固醇导致心脏病发作，这是一种遗传性疾病，会导致个体中低密度脂蛋白胆固醇（LDL）的积累。LDL是一种坏胆固醇，大大增加患者早期心脏病发作的风险，降低预期寿命。Verve公司提出的解决方案是，将一种叫做作“VERVE-101”的血清物质注射到试验参与者的肝脏中，通过改变负责胆固醇管理的PCSK9基因来降低LDL水平。


虽然对猴子进行的测试为备受期待的人体试验铺平了道路，但Heart-1现在又遇到了另一个障碍。


去年，参加Heart-1的一名患者因心脏病发作去世。据《自然》（Nature）杂志去年11月报道，专家们一致认为VERVE-101并不对此负有责任，专家们得出的结论是，患者死亡是由晚期心脏病造成的，但加上另一些新闻报道中的其它严重副作用，这一悲惨事件仍然引起了普遍关注。现在，另一名Heart-1参与者也出现了非常严重的副作用，从而引起安全担忧，最终导致试验暂停。


据彭博社报道，在参加Heart-1试验的前6名患者中，有5名患者的胆固醇水平有所下降。与此同时，第六名患者出现了“肝酶异常”以及血小板减少症。血小板减少症是一种严重的疾病，在最坏的情况下，可能导致致命的内出血。


值得庆幸的是，患者的症状在停药几天后就消失了。Verve表示，这些异常是由将VERVE-101输送到患者肝脏的血清中的脂肪脂质（fatty lipids）引起的。因此，换句话说，这是传递方法的问题，而不是基因编辑过程本身的问题。


据Verve公司网站称，共有13名患者接受了VERVE-101治疗。但现在，由于异常的副作用，这家生物技术初创公司将重点转向VERVE-102，这是一种悬浮在不同脂质血清中的基因编辑药物。根据Verve的说法，在第三方临床试验中，患者“耐受性良好”。VERVE-102将于今年第二季度开始。


遗传性心脏病是一个难以捉摸且广泛存在的问题，如果Verve能够成功地进行一次性基因编辑治疗来缓解其中某些症状，那就可以拯救许多人的生命。虽然Heart-1试验确实显示出了一些希望，但Verve似乎还有很长的路要走。◇


责任编辑：叶紫微
https://www.epochtimes.com/gb/24/4/8/n14220530.htm

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奇怪的副作用

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China and Myanmar are friendly neighbors connected by mountains and rivers, and the traditional friendship between the two peoples has a long history. Since ancient times, the people of the two countries have known each other as "paukphaw" (brothers). The two countries formally established diplomatic relations on June 8, 1950. In the 1950s, China and Myanmar jointly advocated the Five Principles of Peaceful Coexistence. In the 1960s, in the spirit of friendly consultation, mutual understanding and accommodation, the two countries successfully resolved the boundary issues left over from history through friendly consultations, setting an example for countries to resolve boundary issues. Since the establishment of diplomatic relations, bilateral relations have developed steadily.

　　The leaders of China and Myanmar have a tradition of visiting each other. Chairman Liu Shaoqi, Premier Zhou Enlai, Vice Premier Chen Yi and other Chinese leaders of the older generation have visited Myanmar. Myanmar Chairman Ne Win, Prime Minister U Nu and President San Yoo have also visited China many times. Premier Zhou Enlai's nine visits to Myanmar and Ne Win's 12 visits to China have become good stories among the people of the two countries.

　　In recent years, the friendly relations between China and Myanmar have continued to develop steadily, and practical cooperation between the two countries in various fields has continued to deepen. In May 2011, then Myanmar President Thein Sein visited China, and the two sides decided to establish a comprehensive strategic cooperative partnership. Thein Sein visited China a total of 7 times during his term as president, 3 of which were state visits. In April 2015, President Xi Jinping met with President Thein Sein during the 60th anniversary commemorations of the Asian-African Summit and the Bandung Conference in Indonesia. In June, at the invitation of the Communist Party of China, Aung San Suu Kyi led a NLD delegation to visit China for the first time as chairman of the NLD. In September, President Thein Sein went to China to attend the commemorations of the 70th anniversary of the victory of the Chinese People’s War of Resistance Against Japanese Aggression and the World Anti-Fascist War. In March 2016, Vice President Sai Maok Kham was invited to China to attend the first Lancang-Mekong Cooperation Leaders’ Meeting and the Boao Forum for Asia Annual Conference.

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