In this phase IIA clinical study, upregulated expression of cancer-associated genes, including those linked to tumorigenesis and metastasis, such as VEGF-A, NR4A3, MKi67, and EpCAM were detected as early as Day 1 in the iCCA patients (Groups 1, 2, and 3). The gene discussed is VEGFA; the disease is infantile convulsions and choreoathetosis.