JAMA Neurol

Can an iron chelator slow cognitive decline in early Alzheimer’s?

November 7, 2024

Deferiprone decreased brain iron accumulation but accelerated cognitive decline in patients with early Alzheimer disease (AD) in a phase 2 randomized trial, suggesting that lowering brain iron with deferiprone may be detrimental for AD patients.

Conducted over 12 months at nine sites in Australia, the study included 81 participants with amyloid-confirmed mild cognitive impairment or early AD. Participants were randomized in a 2:1 ratio to receive either deferiprone 15 mg/kg bid (n = 53) or placebo administered orally for one year (n = 28).

Key findings: In an intention-to-treat analysis, those in the deferiprone group experienced a greater decline in cognitive function compared with the placebo group (neuropsychological test battery composite z score change: -0.80 vs. -0.30). While deferiprone reduced brain iron levels in the hippocampus, it didn't affect overall brain volume and was associated with a higher incidence of neutropenia.

Source:

Ayton S, et al. (2024, November 4). JAMA Neurol. Deferiprone in Alzheimer Disease: A Randomized Clinical Trial. https://pubmed.ncbi.nlm.nih.gov/39495531/