VOA健康报道2023 FDA或批准首个可能治愈镰状细胞病的疗法（在线收听）

FDA或批准首个可能治愈镰状细胞病的疗法

A group of health experts recently announced that a new gene editing treatment for sickle cell disease was safe for patients.

一个健康专家小组最近宣布，一种新的针对镰状细胞病的基因编辑疗法对患者来说是安全的。

The treatment could provide a new cure that attacks the disease at its genetic source.

这种疗法可能会提供一种新的从基因源头攻克这种疾病的治愈方法。

Sickle cell disease (SCD) is a genetic blood disorder that affects millions of people worldwide.

镰状细胞病是一种遗传性血液疾病，影响全球数百万人。

The disease is common in places with high cases of malaria, like Africa and India.

这种疾病在疟疾高发区很常见，比如非洲和印度。

It strikes among Black Americans, Africans, and Middle Easterners more than other groups.

相较于其他群体，美国黑人、非洲人和中东人更容易患镰状细胞病。

Scientists believe being a carrier of SCD helps protect against severe malaria.

科学家认为，成为镰状细胞病的携带者有助于预防严重的疟疾。

People with SCD carry unhealthy blood cells that result in blockages, called blood clots.

患有镰状细胞病的人携带着不健康的血细胞，它们会导致阻塞，即血栓。

This can cause pain and damage bones and organs in the human body.

这会引起疼痛，损害人体的骨骼和器官。

The World Health Organization said many children with the most severe form of the disease die before the age of five, usually from an infection or severe blood loss.

世界卫生组织表示，许多患有最严重镰状细胞病的儿童在五岁之前死亡，通常死于感染或严重失血。

Vertex Pharmaceuticals and CRISPR Therapeutics make the new treatment, called "exa-cel."

福泰制药和CRISPR Therapeutics公司创造了这种名为“exa-cel”的新疗法。

The treatment involves permanently changing the genetic material, DNA, in a patient's blood cells.

这种疗法包括永久改变患者血细胞中的遗传物质DNA。

The goal is to help the body return to healthy blood cell production.

其目标是帮助身体恢复健康的血细胞生产功能。

Healthy blood cells are present at birth but abnormal cells begin to develop in people with sickle cell disease.

健康的血细胞在出生时就存在，但患有镰状细胞病的人开始出现异常细胞。

When patients receive the treatment, stem cells are removed from their blood and the gene-editing tool CRISPR is used to remove the switching gene.

当患者接受治疗时，医生会从他们的血液中移除干细胞，并使用基因编辑工具CRISPR移除开关基因。

Patients get medicines to kill off other damaged blood-producing cells and then are given back their changed stem cells.

患者服用药物来杀死其他受损的造血细胞，然后医生再把编辑过的干细胞放回原位。

Vertex reported 46 people got the treatment in its study.

福泰制药报告称，有46人在其研究中接受了这种治疗。

Among 30 of them who had at least 18 months of observation, 29 were free of pain crises for at least a year.

在30名至少被观察了18个月的患者中，有29名患者至少一年没有出现疼痛危机。

All 30, Vertex reported, avoided being hospitalized for pain crises for that long.

据福泰制药报告，所有30名患者在很长一段时间内都没有因疼痛危机而住院。

Victoria Gray, of Mississippi, was the first patient to test the "exa-cel" treatment.

密西西比州的维多利亚·格雷是第一个测试这种“exa-cel”疗法的患者。

Gray told researchers at a scientific gathering earlier this year that she had suffered from pain since childhood.

格雷在今年早些时候的一次科学会议上告诉研究人员，她从小就饱受疼痛之苦。

She described feeling "reborn" the day she got the treatment.

她称，在接受治疗的那一天，她感觉自己“重获新生”。

"My children no longer have a fear of losing their mom to sickle cell disease," she said.

她说：“我的孩子们再也不用害怕因为镰状细胞病而失去他们的母亲”。

The U.S. Food and Drug Administration (FDA) had already found the treatment effective in a study of SCD patients.

美国食品药品监督管理局已经在一项针对镰状细胞病患者的研究中发现了这种治疗方法的有效性。

The health agency, however, was not sure if the small genetic sample size used in the study captured the entire U.S. population for sickle cell disease.

然而，该卫生机构不确定研究中使用的少量基因样本是否涵盖了整个美国镰状细胞病患者群。

With the experts' findings, the FDA is likely to approve the treatment in early December for patients age 12 and older.

根据专家的研究结果，美国食品药品监督管理局可能会在12月初批准对12岁及以上患者使用这种疗法。

If the treatment is approved, Vertex has also proposed to follow up with patients to study its safety for another 15 years.

如果该疗法获得批准，福泰制药还提议对患者进行随访，再进行15年的安全性研究。

Vertex has not said how much the treatment will cost.

福泰制药尚未透露治疗费用。

But the Institute for Clinical and Economic Review estimates the cost could be around $2 million for the treatment.

但临床和经济审查研究所估计，这种疗法的费用可能在200万美元左右。

By comparison, research earlier this year showed medical costs for sickle cell treatments, from birth to age 65, could add up to $1.7 million.

相比之下，今年早些时候的研究显示，镰状细胞病患者从出生到65岁的医疗费用总计可能高达170万美元。

Dr. Allison King of Washington University School of Medicine in St. Louis, Missouri cares for children and young adults with sickle cell disease.

密苏里州圣路易斯市华盛顿大学医学院的艾利森·金博士负责治疗患有镰状细胞病的儿童和年轻人。

She told the Associated Press that the new treatments would be costly.

她告诉美联社，新的治疗方法费用高昂。

"But if you think about it," she said, "how much is it worth for someone to feel better and not be in pain and not be in the hospital all the time?"

她说：“但如果你仔细想想，让人感觉好一些，不再痛苦，不再一直待在医院里，这值多少钱？”