# Preclinical evaluation of small molecules with proven benefit in FA animal models

> **NIH NIH P01** · OREGON HEALTH & SCIENCE UNIVERSITY · 2020 · $503,402

## Abstract

Project summary
Fanconi Anemia (FA) is a devastating genetic disease characterized by progressive
bone marrow failure, birth defects and cancer susceptibility. The clinical outcomes in
terms of morbidity and mortality remain poor and the pharmacological therapy of FA has
not changed in over 30 years.
In the past funding period we identified multiple novel small molecules and therapeutic
targets that have beneficial effects in FA models. In this project we will follow up on the
following candidates: 1) metformin (Aim1), P38-MAPK inhibitors (Aim 2) and
combinations of small molecules (Aim 3), including those from Aims 1 and 2 as well as
Project 2.
We will use murine models of FA and xenotransplants (Core B) to assess the effects of
these regimens on human blood progenitors in vivo. Core C will perform DNA damage
assays for our project and Project 3 will test our candidates on human FA cells in vitro.
In aggregate these experiments will define the therapeutic potential of small molecule
monotherapy or drug combinations for the treatment of bone marrow failure in FA.

## Key facts

- **NIH application ID:** 9983800
- **Project number:** 5P01HL048546-25
- **Recipient organization:** OREGON HEALTH & SCIENCE UNIVERSITY
- **Principal Investigator:** Markus Grompe
- **Activity code:** P01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $503,402
- **Award type:** 5
- **Project period:** — → 2022-02-28

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9983800

## Citation

> US National Institutes of Health, RePORTER application 9983800, Preclinical evaluation of small molecules with proven benefit in FA animal models (5P01HL048546-25). Retrieved via AI Analytics 2026-07-13 from https://api.ai-analytics.org/grant/nih/9983800. Licensed CC0.

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