syndrome is an inherited cancer disorder caused by mutations in the
tumor suppressing protein p53. Individuals with Li-Fraumeni syndrome are at an increased risk for a
number of cancers, including breast and bone cancer.
Mutations in the protein p53 have also been linked to increased mitochondrial metabolism. It is not clear whether targeting these
metabolic changes can effectively reduce the risk of cancer associated
with p53 mutations.
‘Metformin can ameliorate abnormal mitochondrial metabolism and may be a potential strategy for tumor prevention in Li-Fraumeni syndrome.’
In the JCI
, research led by
Paul Hwang at the National Heart, Lung, and Blood Institute discovered
that treatment with the commonly-prescribed diabetes medication
metformin treatment blocked increases in mitochondrial metabolism in a
mouse model of Li-Fraumeni syndrome, leading to a lower rate of tumor
formation and increased survival time.
Furthermore, in a pilot study
involving Li-Fraumeni patients, metformin treatment produced a similar
inhibition of mitochondrial metabolism and activated of
The finding that this FDA-approved drug
can ameliorate abnormal mitochondrial metabolism suggests that it may be
a potential strategy for tumor prevention in Li-Fraumeni syndrome.