Use of CFTR Modulators For Treating Cerebrovascular Conditions
AssigneeQanatpharma AG
InventorSteffen-Sebastian Bolz
Life of this patent
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Abstract
Methods of use of CFTR corrector compounds, particularly lumacaftor and tezacaftor, to treat reduced cerebral perfusion associated with heart failure, sudden sensoneurinal hearing loss, vascular dementia, arterial hypertension, ischemic stroke, hemorrhagic stroke, heart disease, diabetes and Alzheimer's disease by treatment with an amount sufficient to provide a proteostatic effect on cystic fibrosis transmembrane conductance regulator (CFTR) protein expression in cerebral artery smooth muscle cells and an increase in cerebrovascular perfusion in the patient.
Claims31
A method for the treatment of a reduced cerebral vascular perfusion in cerebral arteries of a patient, where the reduced cerebral vascular perfusion is associated with a condition selected from the group consisting of heart failure, sudden sensoneurinal hearing loss, vascular dementia, arterial hypertension, ischemic stroke, hemorrhagic stroke, heart disease, diabetes and Alzheimer's disease, comprising administering a CFTR corrector compound to the patient in an amount sufficient to provide a proteostatic effect on cystic fibrosis transmembrane conductance regulator (CFTR) protein expression in the cerebral arteries and an increase in cerebral vascular perfusion in the cerebral arteries.
A method for the treatment of a reduced cerebrovascular perfusion associated with a condition selected from the group consisting of heart failure, sudden sensoneurinal hearing loss, vascular dementia, arterial hypertension, ischemic stroke, hemorrhagic stroke, heart disease, diabetes and Alzheimer's disease in a patient, comprising administering a CFTR corrector compound to the patient in an amount sufficient to provide an increase in cerebrovascular perfusion in the patient.
A method for the treatment of a reduced expression of cystic fibrosis transmembrane conductance regulator (CFTR) proteins in cerebral arteries associated with heart failure in a patient, comprising administering a CFTR corrector compound to the patient in an amount sufficient to provide a proteostatic effect on cystic fibrosis transmembrane conductance regulator (CFTR) protein expression in cerebral artery smooth muscle cells and an increase in cerebrovascular perfusion in the patient.
A method for increasing the abundance of cystic fibrosis transmembrane conductance regulator (CFTR) proteins in cerebral arteries of a patient, in which the expression of CFTR in smooth muscle cells of cerebral arteries is reduced due to a condition selected from the group consisting of heart failure, sudden sensoneurinal hearing loss, vascular dementia, arterial hypertension, ischemic stroke, hemorrhagic stroke, heart disease, diabetes and Alzheimer's disease, comprising administering an effective amount of a CFTR corrector compound sufficient to stabilize CFTR proteins against internalization and subsequent degradation in smooth muscle cells of cerebral arteries of the patient, whereby there is an increase in cerebrovascular perfusion in the patient.
A method for the treatment of reduced cerebral perfusion associated with a condition selected from the group consisting of heart failure, sudden sensoneurinal hearing loss, vascular dementia, arterial hypertension, ischemic stroke, hemorrhagic stroke, heart disease, diabetes and Alzheimer's disease in a human in need of treatment for the reduced cerebral perfusion associated with the condition selected from the group consisting of heart failure, sudden sensoneurinal hearing loss, vascular dementia, arterial hypertension, ischemic stroke, hemorrhagic stroke, heart disease, diabetes and Alzheimer's disease, comprising administering to the human in need of treatment for the reduced cerebral perfusion associated with heart failure an effective amount of at least one cystic fibrosis transmembrane conductance regulator (CFTR) corrector compound.
Dependencies are inferred from the claim text.