Our previous studies have demonstrated that a novel natural product derivative, MP1 (Marinopyrrole/Pyrrolomycin-1), has desirable physicochemical and drug-like properties, potent anti-tumor activity in MYCN-amplified neuroblastoma cell lines (IC50 = 96 nM), an acceptable pharmacokinetic profile in mice, and significant antitumor efficacy in vivo against MYCN-driven neuroblastoma when used in combination with temsirolimus, an FDA approved mTOR inhibitor [19, 20]. The gene discussed is MYCN; the disease is neuroblastoma.