# IND: 113343 Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients with Fanconi Anemia

> **NIH FDA R01** · CINCINNATI CHILDRENS HOSP MED CTR · 2022 · $433,807

## Abstract

Project Summary/Abstract
Current therapies for children with Fanconi anemia (FA) and squamous cell carcinoma (SCC) (commonly seen
in older and/or post-transplant (HCT) patients) include radiation and chemotherapy, which are associated with
significant morbidity and mortality. Thus, there is clearly a need for a novel approach that has fewer and less
severe side effects. Animal and humans studies indicate that reactive-oxygen species (ROS) play a key role in
the pathogenesis of SCC in these children. Our long-term goal is to interdict the development of SCC in post-
HCT patients with FA. Our overall objective, is to develop, at a phase 2 level, a novel approach to treatment of
SCC in FA that is safer and more efficacious compared to existing approaches. It is our central hypothesis that
treatment with the ROS scavenger quercetin will modulate systemic and salivary/mucosal ROS levels in patients
with FA, which in turn will ameliorate development of SCC. In non-FA setting, Quercetin is shown to prevent or
modulate SCC formation in mice. Our own preliminary data show that quercetin reverses the ill effects of ROS
on FA SCC tumor cells and kills them in a dose dependent fashion. Buccal brushing samples from children with
FA showed decreased number of micronuclei (marker of DNA damage/tumor formation) after treatment with oral
Quercetin. These data strongly suggest that quercetin will be beneficial in decreasing ongoing DNA damage and
preventing SCC in children with FA. Our multidisciplinary team is well prepared and will have access to sufficient
number of patients. Quercetin is a naturally occurring anti-oxidant, and was well tolerated and able to achieve
biologically relevant in patients with FA in our recent Phase 1 pilot study. In this Phase 2 study, we will test the
above hypothesis with the following specific aims: 1.Determine the efficacy of Quercetin in reducing buccal
micronuclei (a surrogate marker of DNA damage and susceptibility to SCC due to genomic instability) in
post-HCT patients with FA. Thirty eight post-HCT FA patients will receive oral quercetin for a total of 24 months
and be followed with assessment of serial buccal micronuclei. 2. Measure the impact of Quercetin therapy on
additional potential surrogate markers Peripheral blood ROS and salivary ROS, Salivary total antioxidant
capacity, Biomarkers measured via Exhaled Breath Condensate (EBC) - anti-oxidants, aldehydes etc., Oral
microbiome, and Skin elasticity. The impact of quercetin on reduction of buccal micronuclei and blood as well as
salivary ROS will serve as surrogate markers for prevention of SCC. Additionally, effect on improving salivary
total antioxidant capacity, oral microbiome and skin elasticity will be quantified. Expected outcomes include the
demonstration that long-term quercetin therapy decreases buccal micronuclei (a surrogate marker of DNA
damage and susceptibility to squamous cell carcinoma due to genomic in-stability) in post-HCT patients with FA.
Expected...

## Key facts

- **NIH application ID:** 10425219
- **Project number:** 5R01FD006353-03
- **Recipient organization:** CINCINNATI CHILDRENS HOSP MED CTR
- **Principal Investigator:** PARINDA A. MEHTA
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** FDA
- **Fiscal year:** 2022
- **Award amount:** $433,807
- **Award type:** 5
- **Project period:** 2019-09-01 → 2024-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10425219

## Citation

> US National Institutes of Health, RePORTER application 10425219, IND: 113343 Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients with Fanconi Anemia (5R01FD006353-03). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10425219. Licensed CC0.

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