VOA健康报道2024--New Treatments for Sickle Cell Remain Out of Reach for Many

New Treatments for Sickle¹ Cell Remain Out of Reach for Many

United States health officials recently approved two new gene² 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.

SCD is a genetic³ disorder⁴ 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 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.

Worldwide, the disease is believed to affect millions. It is known to be common in places with high cases of malaria⁵, like Africa and India. Scientific research has shown that people who have SCD seem to react less severely⁶ when they are infected with malaria.

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.

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.

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.

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.

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."

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.

But experts have warned that simpler treatments will likely still be too costly⁷ for many people. This has led to calls for governments and non-profit organizations to get involved to help provide financial assistance to patients.

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."

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.

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.

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.

But Dongre noted⁸ 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.

Words in This Story

gene editing – n. the process of changing genes⁹ using scientific methods in an effort to prevent or cure disease

challenge – n. something difficult that tests one's ability or determination

1 sickle   

n.镰刀

参考例句：

• The gardener was swishing off the tops of weeds with a sickle.园丁正在用镰刀嗖嗖地割掉杂草的顶端。
• There is a picture of the sickle on the flag. 旗帜上有镰刀的图案。

2 gene   

n.遗传因子，基因

参考例句：

• A single gene may have many effects.单一基因可能具有很多种效应。
• The targeting of gene therapy has been paid close attention.其中基因治疗的靶向性是值得密切关注的问题之一。

3 genetic   

adj.遗传的，遗传学的

参考例句：

• It's very difficult to treat genetic diseases.遗传性疾病治疗起来很困难。
• Each daughter cell can receive a full complement of the genetic information.每个子细胞可以收到遗传信息的一个完全补偿物。

4 disorder   

n.紊乱，混乱；骚动，骚乱；疾病，失调

参考例句：

• When returning back,he discovered the room to be in disorder.回家后，他发现屋子里乱七八糟。
• It contained a vast number of letters in great disorder.里面七零八落地装着许多信件。

5 malaria   

n.疟疾

参考例句：

• He had frequent attacks of malaria.他常患疟疾。
• Malaria is a kind of serious malady.疟疾是一种严重的疾病。

6 severely   

adv.严格地；严厉地；非常恶劣地

参考例句：

• He was severely criticized and removed from his post.他受到了严厉的批评并且被撤了职。
• He is severely put down for his careless work.他因工作上的粗心大意而受到了严厉的批评。

7 costly   

adj.昂贵的，价值高的，豪华的

参考例句：

• It must be very costly to keep up a house like this.维修这么一幢房子一定很昂贵。
• This dictionary is very useful,only it is a bit costly.这本词典很有用，左不过贵了些。

8 noted   

adj.著名的，知名的

参考例句：

• The local hotel is noted for its good table.当地的那家酒店以餐食精美而著称。
• Jim is noted for arriving late for work.吉姆上班迟到出了名。

9 genes   

n.基因( gene的名词复数 )

参考例句：

• You have good genes from your parents, so you should live a long time. 你从父母那儿获得优良的基因，所以能够活得很长。 来自《简明英汉词典》
• Differences will help to reveal the functions of the genes. 它们间的差异将会帮助我们揭开基因多种功能。 来自英汉非文学 - 生命科学 - 生物技术的世纪