A team from Wayne State University, led by Miriam Greenberg, Ph.D., professor of biological sciences in the College of Liberal Arts and Sciences, recently received a grant from the National Heart, Lung, and Blood Institute of the National Institutes of Health to work on potential new targets for treating Barth syndrome. The four-year, nearly $1.5 million award, “The role of cardiolipin in the TCA (tricarboxylic acid) cycle: Implications for Barth syndrome,” aims to identify specific metabolites as candidates for new treatments for Barth syndrome and other cardiomyopathies.
According to Greenberg, BTHS causes numerous pathologies, including cardiomyopathy, a disorder of the heart muscle; neutropenia, a reduction in the number of white blood cells; hypotonia, reduced muscle tone; undeveloped skeletal muscles and muscle weakness; delayed growth; decreased stamina; physical disability; and methylglutaconic aciduria, an increase in an organic acid that is characteristic of abnormal mitochondrial function.
Greenberg and her team aim to create a new model of Barth syndrome pathogenesis by elucidating mechanisms where cardiolipin regulates the TCA cycle and intermediary metabolism.
“We aim to reveal a new direction for BTHS treatment based on activation of PDH and/or supplementation of deficient metabolites,” said Greenberg. “The outcome of our study may reveal a new direction for Barth syndrome treatment based on supplementation of deficient metabolites.”
The grant number for this National Institutes of Health award is R01 HL 117880.
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