FDA Health Advisors Vote Against ALS Treatment
2023-10-03
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1United States health advisers voted against an experimental treatment for ALS disease last week.
2The vote came after years of efforts by ALS patients and their families in support of the unproven medical treatment.
3ALS, or amyotrophic lateral sclerosis, is also known as Lou Gehrig's disease.
4Patients with ALS slowly lose the ability to control muscle movements, the ability to speak, eat, move and breathe.
5The U.S. National Institutes of Health says people can get the disease at any age.
6It most commonly develops between the ages of 55 and 75.
7Last Wednesday, a group of health advisors for the U.S. Food and Drug Administration (FDA) voted 17 to one against the treatment from BrainStorm Cell Therapeutics.
8The Israel-based company's treatment, NurOwn, uses stem cells.
9These are cells that can develop into many kinds of body cells.
10The group found the treatment was not effective for patients with ALS.
11A member of the group representing ALS patients was the only vote in favor.
12One expert did not register a vote.
13Lisa Lee, a health expert from Virginia Tech University voted against the treatment.
14She said, "Creating false hope can be considered a moral injury and the use of statistical magic or manipulation to provide false hope is problematic."
15The FDA is not required to accept the group's vote.
16However, the agency's own scientists said earlier in the week that BrainStorm's treatment was "scientifically incomplete."
17Brainstorm and the ALS community wanted to use the meeting to change the FDA's thinking about the NurOwn treatment.
18Brainstorm's 200-patient study failed to show that NurOwn extended life, slowed the disease, or improved patients' ability to move.
19But the FDA agreed to bring outside advisers together after ALS patients and supporters submitted 30,000 signatures seeking a public meeting.
20In the last year, the FDA has approved two new experimental drugs for ALS, Relyvrio and Qalsody.
21The approvals followed intense campaigns from the ALS community although there have been a limited number of scientific studies of the treatments.
22The FDA has used the term "regulatory flexibility" when considering experimental treatments for some conditions, including ALS and Alzheimer's.
23But the agency appears unwilling to overlook the results of BrainStorm's approval effort.
24Important information about manufacturing and quality control was missing.
25Dr. 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 prospects out there that we need to encourage. Approving one like this would get in the way of that."
26At last week's meeting, ALS patients, their family members and doctors spoke in support of BrainStorm's treatment.
27Several presented before-and-after videos that showed patients who had taken part in BrainStorm's study.
28The videos showed the patients walking, climbing stairs and doing other activities.
29Mitze Klingenberg spoke for her son Matt Klingenberg who doctors found to have ALS in 2018.
30She said, "When Matt is on NurOwn it helps him, when he's off of it he gets worse."
31The FDA is expected to issue its decision on the treatment by December 8.
32I'm Gena Bennett.
1United States health advisers 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. 2ALS, or amyotrophic lateral 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. 3Last Wednesday, a group of health advisors for the U.S. Food and Drug Administration (FDA) voted 17 to one against the treatment from BrainStorm 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. 4Lisa 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 statistical magic or manipulation to provide false hope is problematic." 5The 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." 6Brainstorm and the ALS community wanted to use the meeting to change the FDA's thinking about the NurOwn treatment. 7Brainstorm'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. 8In 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. 9The FDA has used the term "regulatory flexibility" when considering experimental treatments for some conditions, including ALS and Alzheimer's. But the agency appears unwilling to overlook the results of BrainStorm's approval effort. Important information about manufacturing and quality control was missing. 10Dr. 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 prospects out there that we need to encourage. Approving one like this would get in the way of that." 11At last week's meeting, ALS patients, their family members and doctors spoke 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. 12Mitze 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." 13The FDA is expected to issue its decision on the treatment by December 8. 14I'm Gena Bennett. 15Hai Do adapted this report for VOA Learning English from the Associated Press and other sources. 16_________________________________________________ 17Words in This Story 18statistical -adj. related to numbers used to provide information about people or things 19manipulation -n. the process of using or changing information in a way to gets a desired result 20prospect -n. something or someone who is a candidate for a use or a position 21encourage -v. to make something more likely to happen