A phase I/II multi-center study of intravesical nanoparticle albumin-bound rapamycin (ABI-009) in the treatment of BCG refractory non-muscle invasive bladder cancer.

TPS4576 Background: Inactivation of p53 and PTEN mutations promotes progression in bladder cancer via deregulation of the mammalian target of rapamycin signaling pathway (mTOR). mTOR expression increases as a function of the disease stage as it progresses from superficial to invasive bladder cancer. Our group has proven the efficacy of intravesical rapamycin to treat bladder cancer in a mouse model. With the advent of the nanoparticle albumin bound (nab) version of rapamycin (ABI-009), this hydrophobic entity can now be tested intravesically. We sought to investigate the safety and efficacy in the management of Bacillus Calmette-Guerin (BCG) refractory non-muscle invasive urothelial carcinoma of the bladder (NMIBC). Methods: Funded via the NIH small business innovation research program (grants 1R42CA171552 and 3R42CA171552), this combined phase I/II study is currently enrolling patients with BCG refractory NMIBC at Columbia University and Vanderbilt University. BCG refractory is defined as disease recurre...