The Effect of Deferiprone on Iron Level in the Brain and Cognitive Decline in Alzheimer’s Disease
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Reviewed by Dat Tien Nguyen, B.A, ScM.
Translated by Nhi Phuong Quynh Le, B.A |
Posted on March 3rd, 2025
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Previous neurological research indicates that elevated iron accumulation in the brain is linked to neurodegeneration in patients with Alzheimer’s disease, potentially due to iron’s role in promoting cell death through ferroptosis. Supported by funding from ApoPharma, a study was conducted to evaluate the use of deferiprone, an iron chelator, for managing neuropsychological symptoms in Alzheimer’s disease.
The study involved 167 participants, averaging 72.5 years of age, who had been diagnosed with Alzheimer’s disease and met either a minimum amyloid concentration of 656 pg/mL in the cerebrospinal fluid or a minimum score of 25 on the Centiloid scale. Patients were randomly assigned to receive either a placebo or deferiprone at a dose of 15 mg per kilogram of body weight, administered twice daily. Deferiprone is an iron chelator capable of crossing the blood-brain barrier and binding excess iron for excretion via urine.
After 12 months of treatment, evaluations using a neuropsychological test battery—measuring attention, language, memory, spatial, and executive abilities—revealed that patients receiving deferiprone experienced significantly greater cognitive decline. MRI analysis showed a substantial decrease in iron accumulation in the brain among those treated with deferiprone, but no significant difference in hippocampal volume changes. These results indicate that reducing iron levels in the brains of Alzheimer’s patients may hasten neurocognitive deterioration. The researchers suggested that deferiprone’s iron-removal action could interfere with tyrosine hydroxylase, the enzyme responsible for dopamine production, and may also lead to metabolic dysfunction in the brain.
The study involved 167 participants, averaging 72.5 years of age, who had been diagnosed with Alzheimer’s disease and met either a minimum amyloid concentration of 656 pg/mL in the cerebrospinal fluid or a minimum score of 25 on the Centiloid scale. Patients were randomly assigned to receive either a placebo or deferiprone at a dose of 15 mg per kilogram of body weight, administered twice daily. Deferiprone is an iron chelator capable of crossing the blood-brain barrier and binding excess iron for excretion via urine.
After 12 months of treatment, evaluations using a neuropsychological test battery—measuring attention, language, memory, spatial, and executive abilities—revealed that patients receiving deferiprone experienced significantly greater cognitive decline. MRI analysis showed a substantial decrease in iron accumulation in the brain among those treated with deferiprone, but no significant difference in hippocampal volume changes. These results indicate that reducing iron levels in the brains of Alzheimer’s patients may hasten neurocognitive deterioration. The researchers suggested that deferiprone’s iron-removal action could interfere with tyrosine hydroxylase, the enzyme responsible for dopamine production, and may also lead to metabolic dysfunction in the brain.