The US Food and Drug Administration has approved this drug (aducanumab or Aduhelm) under their accelerated approval program ‘which provides patients suffering from a serious disease earlier access to drugs when there is an expectation of the clinical benefit despite some uncertainty about the clinical benefit of the drug’. The drug has been shown by brain imaging to reduce brain Amyloid Beta Plaque, which appears to be an important component of the Alzheimer process. One study also showed that the treatment slowed deterioration on measures of cognitive function. It did not improve cognitive function. The US Alzheimer’s Association has interpreted these results as providing a chance for ‘more time’. That is, more time for a person with early Alzheimer’s disease, and their families, to enjoy their lives while only affected to a mild degree. The Alzheimer’s Association mounted a campaign to have Aduhelm approved by FDA.
There are critics of this decision. Neurologist David Knopman, who resigned from an advisory committee to the FDA because of their decision, had previously written: ‘contrary to the hope that aducanumab will help Alzheimer patients, the evidence shows it will offer improvement to none, it will harm some of those exposed, and it will consume enormous resources’.
The treatment involves an intravenous infusion every four weeks. Adverse effects including accumulation of fluid (oedema) in the brain (ARIA-E), or microscopic haemorrhages in the brain (ARIA-H), neither of which have been shown to have major or life-threatening effects and both of which can be managed by stopping the treatment, either temporarily or completely.
The treatment will cost USD 56,000 (AUD 72,000) per year.