MAL3-101 targets an allosteric site in the ATP-binding domain of Hsp70, specifically inhibits the ability of Hsp40 co-chaperones to activate Hsp70 ATPase activity, and has demonstrated in vitro and in vivo efficacy in Merkel Cell carcinoma and multiple myeloma as well as rhabdomyosarcoma cell lines (Adam et al., 2014; Braunstein et al., 2011; Sabnis et al., 2016; Sannino et al., 2018; Wisén et al., 2010). The gene discussed is DNAJB1; the disease is AL amyloidosis.