Finally, we found that palbociclib could work synergistically with the PI3K/mTOR inhibitor PF-04691502 to induce tumor regression in HMGA1-driven iCCA preclinical models, suggesting that by combining FDA-approved drugs with minimal AEs, we could improve the treatment outcomes of this deadly malignancy. The gene discussed is HMGA1; the disease is infantile convulsions and choreoathetosis.