MYC as a target for cancer treatment

The MYC gene which consists of 3 paralogs, C-MYC, N-MYC and L-MYC, is one the most frequently deregulated driver genes in human cancer. Because its high prevalence deregulation causal role cancer formation, maintenance progression, targeting theoretically an attractive strategy for treating As a potential anticancer target, was traditionally regarded as undruggable due to absence suitable pocket high-affinity binding by low molecular weight inhibitors. In recent years however, several compounds that directly or indirectly inhibit have been shown activity preclinical tumor models. Amongst detailed investigated strategies are inhibition obligate interaction partner MAX, prevention expression blocking exhibiting synthetic lethality with overexpression MYC. One extensively inhibitors peptide/mini-protein known OmoMYC. OmoMYC, acts all forms their target promoters, has exhibit diverse range models, minimal side effects. Based on broad efficacy limited toxicity, OmoMYC currently being developed evaluation clinical trials. Although no compound yet progressed testing, APTO-253, partly decreasing MYC, undergoing phase I trial patients relapsed/refractory acute myeloid leukemia myelodysplastic syndrome.