New Treatments for Sickle Cell Remain Out of Reach for Many

United States health officials recently approved two new gene treatments for sickle cell disease (SCD). The treatments provide hope that the disease might be curable. But experts say high costs and limited availability will prevent many people from receiving them.

美国卫生官员最近批准了两种针对镰状细胞病（SCD）的新基因疗法。 这些治疗为这种疾病的治愈带来了希望。 但专家表示，高昂的成本和有限的供应将阻止许多人接受它们。

SCD is a genetic disorder that causes oxygen-carrying red blood cells in the body to become misshapen. The condition can block blood flow and cause severe pain and organ damage.

SCD 是一种遗传性疾病，会导致体内携氧红细胞畸形。 这种情况会阻碍血液流动并导致严重疼痛和器官损伤。

SCD is more common among Black Americans, Africans and Middle Easterners than other population groups. Many children with the most severe form of the disease die before age five, usually from an infection or severe blood loss.

与其他人群相比，SCD 在美国黑人、非洲人和中东人中更为常见。 许多患有最严重疾病的儿童在五岁之前死亡，通常是由于感染或严重失血。

Worldwide, the disease is believed to affect millions. It is known to be common in places with high cases of malaria, like Africa and India. Scientific research has shown that people who have SCD seem to react less severely when they are infected with malaria.

在世界范围内，这种疾病据信影响了数百万人。 众所周知，这种疾病在非洲和印度等疟疾病例高发的地区很常见。 科学研究表明，患有 SCD 的人在感染疟疾时反应似乎不那么严重。

While an estimated 1 million people suffer with the condition in India, many of them will not get the chance to receive the new gene treatments. The situation is not new to developing countries. Many have long faced limitations on medicines and other treatment methods for disease.

虽然印度估计有 100 万人患有这种疾病，但其中许多人将没有机会接受新的基因治疗。 对于发展中国家来说，这种情况并不新鲜。 许多人长期以来一直面临药物和其他疾病治疗方法的限制。

But the problem is even more severe with the new SCD treatments and other gene-based methods. This is mainly because of the high cost of treatment. The two methods approved in the U.S. – both of which are given as a single treatment – cost between $2.2 million and $3.1 million.

但新的 SCD 治疗方法和其他基于基因的方法使问题变得更加严重。 这主要是因为治疗费用较高。 美国批准的两种方法（均作为单一治疗）的费用在 220 万至 310 万美元之间。

In addition, the treatment itself can be difficult to carry out in developing nations. This is because the process generally involves several steps and requires longer stays in well-equipped hospitals.

此外，治疗本身在发展中国家可能很难进行。 这是因为该过程通常涉及多个步骤，并且需要在设备齐全的医院停留更长的时间。

Vertex's treatment, called Casgevy, is designed to repair the genetic condition that causes misshapen cells in sickle cell sufferers. It uses a gene editing technology called CRISPR to do so. The other treatment, offered by a company called Bluebird Bio, does not use CRISPR technology. But it requires a similar treatment process as Vertex’s version.

Vertex 的治疗方法称为 Casgevy，旨在修复导致镰状细胞患者细胞畸形的遗传状况。 它使用一种称为 CRISPR 的基因编辑技术来做到这一点。 另一种治疗方法由一家名为 Bluebird Bio 的公司提供，不使用 CRISPR 技术。 但它需要与 Vertex 版本类似的处理过程。

Dr. David Altshuler is the chief scientific officer at Vertex. He told The Associated Press that while there is clearly a “great unmet need” for the new SCD treatments, the process also presents “challenges.”

David Altshuler 博士是 Vertex 的首席科学官。 他告诉美联社，虽然新的 SCD 治疗显然存在“巨大的未满足需求”，但该过程也提出了“挑战”。

Altshuler said Vertex is working to simplify the treatment process to make it be available to more people. For example, his team is seeking to develop a medication in pill form that could perform similarly to the gene editing method.

Altshuler 表示，福泰 (Vertex) 正在努力简化治疗过程，以便更多的人能够使用它。 例如，他的团队正在寻求开发一种药丸形式的药物，其效果与基因编辑方法类似。

But experts have warned that simpler treatments will likely still be too costly for many people. This has led to calls for governments and non-profit organizations to get involved to help provide financial assistance to patients.

但专家警告说，对于许多人来说，更简单的治疗方法可能仍然过于昂贵。 这导致人们呼吁政府和非营利组织参与进来，帮助为患者提供经济援助。

Gautam Dongre lives in the city of Nagpur in central India. His two children suffer from SCD. He told the AP he has been “praying the treatment should come to us.”

高塔姆·东格尔 (Gautam Dongre) 住在印度中部那格浦尔市。 他的两个孩子患有 SCD。 他告诉美联社，他一直在“祈祷我们能得到治疗”。

Dongre’s son is now 19 years old and his daughter is 13. They both currently take a drug called hydroxyurea. That drug has long been used to help prevent the formation of sickle-shaped red blood cells in an effort to control pain and other damaging effects.

东热的儿子现年 19 岁，女儿 13 岁。他们目前都服用一种名为羟基脲的药物。 这种药物长期以来一直被用来帮助防止镰状红细胞的形成，以控制疼痛和其他破坏性影响。

Dongre leads India’s National Alliance of Sickle Cell Organizations. He said many patients in rural parts of India are dying at very young ages without getting effective treatment.

东格尔领导着印度全国镰状细胞组织联盟。 他说，印度农村地区的许多患者在没有得到有效治疗的情况下就在很小的时候就去世了。

He added that if the gene treatments for SCD one day make it to India, he would like his children to be among the first to get them.

他补充说，如果有一天 SCD 的基因治疗方法能够到达印度，他希望他的孩子能够成为第一批接受治疗的人。

But Dongre noted that people in all countries – rich or poor – should be able to get the latest treatments available. “We are all part of one single planet,” he said.

但东格尔指出，所有国家的人们，无论贫富，都应该能够获得最新的治疗方法。 “我们都是同一个星球的一部分，”他说。