Pilot Study Assessing Tolerability and Metabolic Effects of Metformin in Patients With Li-Fraumeni Syndrome.
Farzana L WalcottPing-Yuan WangChristine M BrylaRebecca D HuffstutlerNeha SinghMichael N PollakPayal P KhinchaSharon A SavagePhuong L MaiKevin W DoddPaul M HwangAntonio T FojoChristina M AnnunziataPublished in: JNCI cancer spectrum (2020)
Metformin was safe and tolerable in patients with LFS. It suppressed hepatic mitochondrial function as expected in these individuals. This study adds to the rationale for development of a pharmacologic risk-reduction clinical trial of metformin in LFS.