A phase I trial examining safety, pharmacokinetics and anti-tumour activity of ADH-1 + LPAM in stage IIIB/C melanoma patients with in-transit limb metastases demonstrated complete response in 50% of patients (N = 16) (Beasley et al., 2009), with a subsequent phase II study revealing the combination therapy resulted in significantly improved response rates when compared with standard-of-care isolated limb infusion (ILI) alone [NCT00421811 (Beasley et al., 2011)]. The gene discussed is ADH1A; the disease is neoplasm.