# eRapa for bladder cancer prevention

> **NIH NIH R01** · UNIVERSITY OF TEXAS HLTH SCIENCE CENTER · 2020 · $486,532

## Abstract

Urinary bladder tumors arise from the transitional epithelium and rarely penetrate the bladder’s detrusor muscle.
Thus, complete tumor resection is frequently possible using a transurethral instrument to remove the tumor at
its root without total bladder removal. Unfortunately, genomic instability is rampant throughout the transitional
epithelium in most patients and tumor relapse is common despite complete tumor removal. As a result, patients
require lifelong endoscopic monitoring, multiple biopsies, and repeated intravesical therapies. mTOR inhibition
treats and prevents bladder cancer (BC) in preclinical models and is thought to work by directly targeting bladder
tumors. While this dogma is true, it provides an incomplete picture of the potential activity of this therapy in BC.
Our preliminary data shows favorable pharmacokinetic and pharmacodynamic activity of low dose rapamycin in
bladder tissues. We also show that a novel encapsulated formulation of rapamycin (eRapa) modulates immune
responses and these effects favor improved anti-BC immunity and improved responses towards BCG, which is
standard-of-care immune therapy for high-grade non-muscle invasive bladder cancer (BC). We propose
investigating eRapa for secondary cancer prevention in patients with newly diagnosed non-muscle invasive
bladder cancer. Aim 1 conducts a phase II double-blind randomized controlled trial of long-term (one year)
prevention with eRapa versus placebo. Outcome measures include efficacy, tolerability, and effects on cognition
and physical function. Efficacy is assessed through standard-of-care surveillance monitoring to capture relapses
and to estimate recurrence-free survival. Tolerability is measured with validated BC-specific symptom
assessments and global quality of life questionnaires. Cognition and physical function are assessed with
validated executive/memory function testing and by physical performance testing. Aim 2 test the hypothesis that
eRapa improves immune function in patients with BC by examining the effects of eRapa on circulating immune
cells, including memory T cell differentiation, and tumor-specific immunity. For patients concurrently receiving
intravesical BCG, the ability of eRapa to boost BCG-specific immunity is tested. If successful, this project will
facilitate a phase III registration trial for a new secondary prevention agent for BC patients. In addition, this work
provides foundational insights into immune events in elderly BC patients that are useful to developing other
cancer immunotherapies.

## Key facts

- **NIH application ID:** 10034559
- **Project number:** 1R01CA252057-01
- **Recipient organization:** UNIVERSITY OF TEXAS HLTH SCIENCE CENTER
- **Principal Investigator:** Robert Scott Svatek
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $486,532
- **Award type:** 1
- **Project period:** 2020-07-01 → 2025-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10034559

## Citation

> US National Institutes of Health, RePORTER application 10034559, eRapa for bladder cancer prevention (1R01CA252057-01). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10034559. Licensed CC0.

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