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Evaluation of antihypertensive drugs in combination with enzyme replacement therapy in mice with Pompe disease.

Abstract：

:Pompe disease is caused by the deficiency of lysosomal acid α-glucosidase (GAA) leading to progressive myopathy. Enzyme replacement therapy (ERT) with recombinant human (rh) GAA has limitations, including inefficient uptake of rhGAA in skeletal muscle linked to low cation-independent mannose-6-phosphate receptor (CI-MPR) expression. PURPOSE:To test the hypothesis that antihypertensive agents causing muscle hypertrophy by increasing insulin-like growth factor 1 expression can increase CI-MPR-mediated uptake of recombinant enzyme with therapeutic effects in skeletal muscle. METHODS:Three such agents were evaluated in mice with Pompe disease (carvedilol, losartan, and propranolol), either with or without concurrent ERT. RESULTS:Carvedilol, a selective β-blocker, increased muscle strength but reduced biochemical correction from ERT. Administration of drugs alone had minimal effect, with the exception of losartan that increased glycogen storage and mortality either by itself or in combination with ERT. CONCLUSION:The β-blocker carvedilol had beneficial effects during ERT in mice with Pompe disease, in comparison with propranolol or losartan. Caution is warranted when prescribing antihypertensive drugs in Pompe disease.

journal_name

Mol Genet Metab

journal_title

Molecular genetics and metabolism

authors

Han SO,Haynes AC,Li S,Abraham DM,Kishnani PS,Steet R,Koeberl DD

doi

10.1016/j.ymgme.2019.10.005

subject

Has Abstract

pub_date

2020-02-01 00:00:00

pages

73-79

issue

2

eissn

1096-7192

issn

1096-7206

pii

S1096-7192(19)30679-1

journal_volume

129

pub_type

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