Oral Presentation Royal Australian Chemical Institute National Congress 2026

Allosteric modulation of paclitaxel efficacy by human tubulin variants (136566)

Jingyi Luo 1 , Chen Jing KHOO 1 , Shih Chieh Ti 1
  1. The University of Hong Kong, Hong Kong, CHINA, China

Therapeutic agents that target tubulin and disrupt microtubule dynamics have shown established clinical efficacy against diverse hematological malignancies and solid tumors for decades. However, the structure-activity relationship between therapeutic drugs and prognosis-associated variations in tubulin primary sequences remains unknown. Here, we reveal that tubulin protein families (i.e., isotypes) determine the intrinsic affinities for paclitaxel, a cornerstone chemotherapeutic. Biophysical characterizations and site-directed mutagenesis identify a point mutation, which is distal from the drug-binding pocket, can invert the hypo-sensitivity of human β3-tubulin to paclitaxel. High-resolution (~2.3 Å) cryo-electron microscopy reveals this mutation allosterically remodels the paclitaxel-binding pocket and concurrently enhances the microtubule intrinsic lattice stability. The allosteric network initiated by this paclitaxel-sensitizing mutation extends to α-tubulin E254, reducing its catalytic efficiency for GTP hydrolysis during microtubule assembly. This results in an expanded GTP-cap at growing ends, consistent with the observed preference of EB proteins for dynamic mutant microtubules, and culminates in a significantly lower catastrophe frequency. Furthermore, site-specific genomic integration of this point mutation within the endogenous β3-tubulin locus renders cancer cells profoundly susceptible to paclitaxel. Our findings illuminate how tubulin primary sequences confer divers conformational landscapes that determines the efficacy of paclitaxel, provide a structural and mechanistic blueprint for exploiting tubulin allosteric effect as an approach to design novel microtubule target agent based therapies to circumvent clinical resistance.