# Phase II Trial of Anti-PD1 Therapy for HPV-associated Recurrent Respiratory Papillomatosis (IND#128874)

> **NIH FDA R01** · MASSACHUSETTS GENERAL HOSPITAL · 2020 · $55,000

## Abstract

PROJECT ABSTRACT
Recurrent Respiratory Papillomatosis (RPP) is an orphan disease with approximately 20,000 active cases in
the United States and with an incidence of approximately 4.3 per 100,000 children and 1.8 per 100,000 adults.
RRP is caused by infection with human papillomavirus (HPV) types 6 and 11 and is characterized by mucosal
tumors that grow in the respiratory tract, impacting breathing, swallowing, and speech. In 3-5% of patients, the
lesions can undergo malignant transformation and the prognosis for patients who develop cancers derived
from their RRP is poor. There is no effective systemic therapy for RRP, despite the fact that it typically involves
multiple sites. Thus, there is a strong unmet need to develop novel treatment options for RRP patients. We and
others have shown that RPP is a disease characterized by an ineffective host immune response to HPV, in
part, through the upregulation of the PD1:PDL1 axis. Thus, we hypothesize that administration of a blocking
PD-1 monoclonal antibody, pembrolizumab, can re-activate host anti-tumor responses, leading to the
regression of existing RPP lesions, clearance of chronic HPV infection, and prevention of new disease as a
means to improve the quality of life and achieve possible cure. To test this hypothesis, we have opened an
investigator-initiated phase II clinical trial administering pembrolizumab to subjects diagnosed with advanced
RPP to evaluate the effectiveness of immunotherapy as a novel treatment option. In this study, biopsies and
peripheral blood samples are obtained every 12 weeks while receiving study treatment, which provides a
unique longitudinal sampling of tissue to understand how the tumor, tumor immune microenvironment, and
host immune responses may evolve over time. We seek support to perform key correlative analyses of the pre-
and on-treatment human samples to generate essential data, investigating the role of immunotherapy as a
novel and effective treatment option for RRP patients.

## Key facts

- **NIH application ID:** 10246615
- **Project number:** 3R01FD006341-02S1
- **Recipient organization:** MASSACHUSETTS GENERAL HOSPITAL
- **Principal Investigator:** Sara Isabel Pai
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** FDA
- **Fiscal year:** 2020
- **Award amount:** $55,000
- **Award type:** 3
- **Project period:** 2019-09-01 → 2022-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10246615

## Citation

> US National Institutes of Health, RePORTER application 10246615, Phase II Trial of Anti-PD1 Therapy for HPV-associated Recurrent Respiratory Papillomatosis (IND#128874) (3R01FD006341-02S1). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10246615. Licensed CC0.

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