VOA健康报道2023--FDA Health Advisors Vote Against ALS Treatment

FDA Health Advisors¹ Vote Against ALS Treatment

United States health advisers² voted against an experimental treatment for ALS disease last week. The vote came after years of efforts by ALS patients and their families in support of the unproven medical treatment.

ALS, or amyotrophic lateral³ sclerosis, is also known as Lou Gehrig's disease. Patients with ALS slowly lose the ability to control muscle movements, the ability to speak, eat, move and breathe. The U.S. National Institutes of Health says people can get the disease at any age. It most commonly develops between the ages of 55 and 75.

Last Wednesday, a group of health advisors for the U.S. Food and Drug Administration (FDA) voted 17 to one against the treatment from BrainStorm⁴ Cell Therapeutics. The Israel-based company's treatment, NurOwn, uses stem cells. These are cells that can develop into many kinds of body cells. The group found the treatment was not effective for patients with ALS. A member of the group representing ALS patients was the only vote in favor. One expert did not register a vote.

Lisa Lee, a health expert from Virginia Tech University voted against the treatment. She said, "Creating false hope can be considered a moral injury and the use of statistical⁵ magic or manipulation to provide false hope is problematic."

The FDA is not required to accept the group's vote. However, the agency's own scientists said earlier in the week that BrainStorm's treatment was "scientifically incomplete."

Brainstorm and the ALS community wanted to use the meeting to change the FDA's thinking about the NurOwn treatment.

Brainstorm's 200-patient study failed to show that NurOwn extended life, slowed the disease, or improved patients' ability to move. But the FDA agreed to bring outside advisers together after ALS patients and supporters submitted 30,000 signatures seeking a public meeting.

In the last year, the FDA has approved two new experimental drugs for ALS, Relyvrio and Qalsody. The approvals followed intense campaigns from the ALS community although there have been a limited number of scientific studies of the treatments.

The FDA has used the term "regulatory flexibility⁶" when considering experimental treatments for some conditions, including ALS and Alzheimer's. But the agency appears unwilling⁷ to overlook the results of BrainStorm's approval effort. Important information about manufacturing and quality control was missing.

Dr. Kenneth Fischbeck of the National Institutes of Health said, "It really is a disease that needs a safe and effective treatment and there are a lot of other prospects⁹ out there that we need to encourage. Approving one like this would get in the way of that."

At last week's meeting, ALS patients, their family members and doctors spoke¹⁰ in support of BrainStorm's treatment. Several presented before-and-after videos that showed patients who had taken part in BrainStorm's study. The videos showed the patients walking, climbing stairs and doing other activities.

Mitze Klingenberg spoke for her son Matt Klingenberg who doctors found to have ALS in 2018. She said, "When Matt is on NurOwn it helps him, when he's off of it he gets worse."

The FDA is expected to issue its decision on the treatment by December 8.

Words in This Story

statistical –adj. related to numbers used to provide information about people or things

manipulation –n. the process of using or changing information in a way to gets a desired result

prospect⁸ –n. something or someone who is a candidate for a use or a position

 

encourage –v. to make something more likely to happen

1 advisors   

n.顾问，劝告者( advisor的名词复数 )；（指导大学新生学科问题等的）指导教授

参考例句：

• The governors felt that they were being strung along by their advisors. 地方长官感到他们一直在受顾问们的愚弄。 来自《现代汉英综合大词典》
• We will consult together with advisors about her education. 我们将一起和专家商议她的教育事宜。 来自互联网

2 advisers   

顾问，劝告者( adviser的名词复数 )； （指导大学新生学科问题等的）指导教授

参考例句：

• a member of the President's favoured circle of advisers 总统宠爱的顾问班子中的一员
• She withdrew to confer with her advisers before announcing a decision. 她先去请教顾问然后再宣布决定。

3 lateral   

adj.侧面的，旁边的

参考例句：

• An airfoil that controls lateral motion.能够控制横向飞行的机翼。
• Mr.Dawson walked into the court from a lateral door.道森先生从一个侧面的门走进法庭。

4 brainstorm   

vi.动脑筋，出主意，想办法，献计，献策

参考例句：

• The women meet twice a month to brainstorm and set business goals for each other.她们每个月聚会两次，在一起出谋献策，为各自制定生意目标。
• We can brainstorm a list of the most influential individuals in the company.我们可以集体讨论，列出该公司中最有影响的人员的名单。

5 statistical   

adj.统计的，统计学的

参考例句：

• He showed the price fluctuations in a statistical table.他用统计表显示价格的波动。
• They're making detailed statistical analysis.他们正在做具体的统计分析。

6 flexibility   

n.柔韧性，弹性，（光的）折射性，灵活性

参考例句：

• Her great strength lies in her flexibility.她的优势在于她灵活变通。
• The flexibility of a man's muscles will lessen as he becomes old.人老了肌肉的柔韧性将降低。

7 unwilling   

adj.不情愿的

参考例句：

• The natives were unwilling to be bent by colonial power.土著居民不愿受殖民势力的摆布。
• His tightfisted employer was unwilling to give him a raise.他那吝啬的雇主不肯给他加薪。

8 prospect   

n.前景，前途；景色，视野

参考例句：

• This state of things holds out a cheerful prospect.事态呈现出可喜的前景。
• The prospect became more evident.前景变得更加明朗了。

9 prospects   

n.希望，前途(恒为复数)

参考例句：

• There is a mood of pessimism in the company about future job prospects. 公司中有一种对工作前景悲观的情绪。
• They are less sanguine about the company's long-term prospects. 他们对公司的远景不那么乐观。

10 spoke   

n.（车轮的）辐条；轮辐；破坏某人的计划；阻挠某人的行动 v.讲，谈（speak的过去式）；说；演说；从某种观点来说

参考例句：

• They sourced the spoke nuts from our company.他们的轮辐螺帽是从我们公司获得的。
• The spokes of a wheel are the bars that connect the outer ring to the centre.辐条是轮子上连接外圈与中心的条棒。