VOA健康报道2023--US Approves Two Treatments for Sickle Cell Disease

US Approves Two Treatments for Sickle¹ Cell Disease

United States health officials last week approved two gene² treatments for sickle cell disease. Doctors hope that the treatments will provide a cure for the painful genetic³ blood disorder⁴.

The U.S. Food and Drug Administration (FDA) said the one-time treatments can be used for patients 12 and older with severe forms of the disease.

One treatment is made by Vertex Pharmaceuticals⁵ and CRISPR Therapeutics. The other is made by Bluebird Bio.

"Sickle cell disease is a... life-threatening blood disorder with significant unmet need," the FDA's Dr. Nicole Verdun said in a statement announcing the approvals. "We are excited to advance the field especially for individuals whose lives have been severely⁶ disrupted by the disease."

Sickle cell disease (or SCD) affects millions of people worldwide. The disease is common in places with high cases of malaria⁷, like Africa and India. Scientists believe being a carrier of SCD helps protect against severe malaria.

It is more common among Black Americans, Africans, and Middle Easterners than other population groups. 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.

The new treatments

A genetic change, or mutation⁸, causes blood cells to become defective⁹ in people with sickle cell disease. The unhealthy blood cells result in blockages¹⁰, called blood clots¹¹. The clots cause pain and damage to bones and organs in the human body.

The Vertex treatment is called Casgevy. Its goal is to help the body go back to producing healthy blood cells that are present at birth. It uses CRISPR, the gene editing tool, to knock out a gene in stem cells collected from the patient.

Bluebird's treatment, called Lyfgenia, aims to add copies of a modified gene. The modified gene helps red blood cells to produce a protein that prevents or reverses unhealthy cells.

The two gene treatments are the first approved in the U.S. for sickle cell disease. Vertex is already approved in Britain and Bahrain.

The new treatments are costly¹². The cost for Bluebird Bio is $3.1 million and for Vertex, $2.2 million. To help cover the cost, the U.S. Centers for Medicare and Medicaid says it plans to create partnerships¹³ with state Medicaid agencies and drug companies.

Even with the high cost, many patients say they would consider the gene treatment because of the seriousness of the disease.

Jalen Matthews of Louisville, Kentucky, was diagnosed with sickle cell at birth. She had her first pain episode at age 9. Three years later, the disease led to a spinal¹⁴ cord stroke that left her with some paralysis¹⁵ in her left arm and leg.

"I had to learn how to walk again, feed myself, clothe myself, basically learn how to do everything all over again," said Matthews, who is now 26.

Today, she deals with the disease by having blood transfusions¹⁷. About every two months, the treatment replaces five units of her blood with healthy cells. She said she plans to ask her doctor about the new treatments.

"This one-time kind of cure is very much needed," Matthews said.

Studies testing the treatments suggest they work well. Of 31 people treated in the Vertex study with enough follow-up, 29 were free of pain episodes for at least a year. In the Bluebird study, 28 of 32 patients had no severe pain or organ damage between six and 18 months after the treatment.

But doctors note there are possible side effects. They also say the long-term results are unknown. For both treatments, the required chemotherapy comes with risks such as infertility¹⁸, hair loss and serious infections.

With the Bluebird treatment, blood cancer has happened. The FDA said it will include a "black box warning" about that risk.

With the Vertex therapy, some scientists worry that CRISPR brings the possibility of "off-target effects." These are unexpected changes to a person's genome.

Words in This Story

advance - v. to move forward

knock out - phrasal verb, to cause something to stop working

reverse - v. to return to an earlier state

episode - n. a short time of an illness

paralysis - n. a condition in which you are unable to move

blood transfusion¹⁶ - n. a medical treatment in which someone's blood is put into the body of another person

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 pharmaceuticals   

n.医药品；药物( pharmaceutical的名词复数 )

参考例句：

• the development of new pharmaceuticals 新药的开发
• The companies are pouring trillions of yen into biotechnology research,especially for pharmaceuticals and new seeds. 这些公司将大量资金投入生物工艺学研究，尤其是药品和新种子方面。 来自《简明英汉词典》

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 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。

8 mutation   

n.变化，变异，转变

参考例句：

• People who have this mutation need less sleep than others.有这种突变的人需要的睡眠比其他人少。
• So far the discussion has centered entirely around mutation in the strict sense.到目前为止，严格来讲，讨论完全集中于围绕突变问题上。

9 defective   

adj.有毛病的，有问题的，有瑕疵的

参考例句：

• The firm had received bad publicity over a defective product. 该公司因为一件次品而受到媒体攻击。
• If the goods prove defective, the customer has the right to compensation. 如果货品证明有缺陷， 顾客有权索赔。

10 blockages   

n.堵塞物( blockage的名词复数 )；堵塞，阻塞

参考例句：

• The storms could increase the risks posed by river blockages. 暴风雨会增加因河道堵塞所造成的危险。 来自互联网
• An angiogram shows the location and severity of blockages in blood vessels. 冠状动脉造影能够显示血管内的阻塞位置以及阻塞的严重程度。 来自互联网

11 clots   

n.凝块( clot的名词复数 )；血块；蠢人；傻瓜v.凝固( clot的第三人称单数 )

参考例句：

• When you cut yourself, blood clots and forms a scab. 你割破了，血会凝固、结痂。 来自《简明英汉词典》
• Milk clots when it turns sour. 奶变酸就凝块。 来自《现代英汉综合大词典》

12 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。

13 partnerships   

n.伙伴关系( partnership的名词复数 )；合伙人身份；合作关系

参考例句：

• Partnerships suffer another major disadvantage: decision-making is shared. 合伙企业的另一主要缺点是决定要由大家来作。 来自英汉非文学 - 政府文件
• It involved selling off limited partnerships. 它涉及到售出有限的合伙权。 来自辞典例句

14 spinal   

adj.针的，尖刺的，尖刺状突起的；adj.脊骨的，脊髓的

参考例句：

• After three days in Japan,the spinal column becomes extraordinarily flexible.在日本三天，就已经使脊椎骨变得富有弹性了。
• Your spinal column is made up of 24 movable vertebrae.你的脊柱由24个活动的脊椎骨构成。

15 paralysis   

n.麻痹(症)；瘫痪(症)

参考例句：

• The paralysis affects his right leg and he can only walk with difficulty.他右腿瘫痪步履维艰。
• The paralysis affects his right leg and he can only walk with difficulty.他右腿瘫痪步履维艰。

16 transfusion   

n.输血，输液

参考例句：

• She soon came to her senses after a blood transfusion.输血后不久她就苏醒了。
• The doctor kept him alive by a blood transfusion.医生靠输血使他仍然活着。

17 transfusions   

n.输血( transfusion的名词复数 )；输液；倾注；渗透

参考例句：

• Still, transfusions have apparently never spread the disease, even among hemophiliacs. 还有，输血很明显从未传播过这种病，即使在血友病人之间也是如此。 来自英汉非文学 - 生命科学 - 口蹄疫疯牛病
• Blood transfusions are a special, limited example of tissue transplantation. 输血是一个特殊的、有限制的组织移植的例子。 来自辞典例句

18 infertility   

n.不肥沃，不毛；不育

参考例句：

• It is the Geneva, Switzerland-based Biotech Company's second recombinant infertility drug. 它是瑞士生物技术公司在日内瓦的公司生产的第二种重组治疗不孕症的药。 来自英汉非文学 - 生命科学 - 生物技术制药疫苗
• Endometritis is a cause of infertility. 子宫内膜炎是不育的原子。 来自辞典例句