The US Food and Drug Administration's (FDA) Advisory Committee for Cellular, Tissue and Gene Therapies has voted that there is insufficient evidence that experimental stem-cell therapy for ALS is effective enough to warrant approval by the agency.
Amyotrophic Lateral Sclerosis (ALS) is an incurable, fatal motor neurone disease, where progressive degeneration of nerve cells leads to muscle weakness affecting movement, speech swallowing and breathing. The committee recognised the 'urgent unmet need for additional effective treatments' for the condition, but concluded that the 'NurOwn' therapy did not demonstrably extend life, slow the disease or improve patient mobility.
'False hope is provided when the probability of a positive outcome is overestimated. And I think that seems to be the case here,' said panel member Dr Lisa Lee, who specialises in bioethics and public health at Virginia Tech.
'As a person living with ALS ... I am very sensitive to approving a drug ... that could ultimately result in more harm than good in the long run,' added the panel's patient representative, Andrew Buckley.
The treatment was developed by New York-based BrainStorm Cell Therapeutics, who have had two prior applications rejected by the FDA. It is a personalised treatment, built from stem cells collected from each patient, which are then injected into the cerebrospinal fluid (CSF), where they are intended to secrete proteins, helping neurons survive.
The Committee found that NurOwn did not demonstrate effectiveness in a Phase 3 clinical trial of nearly 200 people with ALS. A total of 13 deaths occurred during the post-treatment follow-up: ten had received the drug, compared to three in the placebo group.
They also noted that the FDA had described BrainStorm's application as 'scientifically incomplete' and that the information about manufacturing processes was 'grossly deficient'.
The Committee voted 17 to 1 – with one abstention – that 'substantial evidence of effectiveness meeting the approval standard' had not been demonstrated. The FDA is not required to follow the advice of its advisory committees, but usually does.
The FDA has previously committed to a flexible approach when reviewing experimental therapies for fatal, hard-to-treat conditions. Last year, they approved a new ALS medicine for the first time in 20 years, and approved gene therapies earlier this year for Duchenne muscular dystrophy and dystrophic epidermolysis bullosa (see BioNews 1196 and 1191).
Commenting on the committee's review, Dr Kenneth Fischbeck from the US National Institutes of Health said: '[ALS] 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.'
The FDA is expected to issue a final decision about whether the therapy will receive approval by 8 December.
Sources and References
-
FDA Briefing Document BLA 125782 Drug name: debamestrocel (MSC-NTF, NurOwn) Applicant: Brainstorm Cell Therapeutics
-
BrainStorm's ALS cell therapy resoundingly rejected by FDA advisers
-
FDA advisers vote against experimental ALS treatment pushed by patients
-
US FDA panel votes against BrainStorm's ALS therapy over effectiveness concern
-
FDA staff hold 'major concerns' with Brainstorm ALS therapy, documents show
-
BrainStorm's ALS therapy not effective, FDA panel finds
Leave a Reply
You must be logged in to post a comment.