# Using clinical data to identify FDA-approved drugs for cancer prevention and therapeutic repurposing

> **NIH NIH K01** · UNIVERSITY OF CHICAGO · 2020 · $1

## Abstract

Project Summary/Abstract
Clinical data collection is accelerating rapidly, and in the future it will include both provider- and patient-
generated data. Hidden within this mass of noisy observational data are clues as to factors influencing disease
onset and outcome. Finding ways to exploit this trove of disease data can unlock a new perspective on disease
processes. We can tackle disease both from the bottom-up, from experimental data generated in the
laboratory, and from the top down, from clinical phenomena observed across human populations. A particularly
impactful and prevalent disease is cancer. Each tumor harbors a unique combination of mutations driving a
distinct set of oncogenic processes. Targeted therapies have been proposed to pinpoint these mutations,
potentially requiring a vast array of therapeutic options. Cancer treatment often fails when drug resistance
arises, another result of the complex combinatorial nature of tumor alterations. Combination therapies have
been proposed as an approach to interfere with multiple disease signals simultaneously. However, identifying
effective drug combinations, and the cancer types in which they are effective, is experimentally infeasible,
leading to a push for computational solutions.
In this proposal, we combine methods from social sciences and biostatistics to find the causal effect of a drug
on cancer onset from observational clinical data. Both increased and decreased cancer rates in drug-takers
are of equal interest, as they can inform us of disease processes and provide clinical impact. We are
particularly interested in finding drug combinations that impact cancer. These combination effects are unlikely
to have been detected, and our clinical data provides a unique resource for observing the effects of tens of
thousands of drug combinations. We will pool the resulting causal drug effect estimates across the many
cancers present in our data. To gain insight into the cellular processes underlying clinical effect, we will
examine the impact of known cancer-causing drugs in vitro, using large public cell line assays.
The accompanying goal is to provide Dr. Rachel Melamed with a career development experience to become
an independent scientist. Her research will use observational health data to understand the genesis of cancer,
prevent the disease, and discover new therapeutic options. This proposal takes advantage of the
interdisciplinary strengths of the University of Chicago in computation, biostatistics, and medicine, as well as
institutional resources in terms of data access and infrastructure. Dr. Melamed has assembled a team
consisting of complementary mentors and collaborators with expertise in computation, statistics, translational
medicine, personalized therapy, and cancer therapy. The career development plan focuses on enhancing her
statistics and machine learning skills with structured coursework and mentorship, and gaining experience in
biomedical applications via applied work and...

## Key facts

- **NIH application ID:** 9963220
- **Project number:** 5K01ES028055-04
- **Recipient organization:** UNIVERSITY OF CHICAGO
- **Principal Investigator:** Rachel Dania Melamed
- **Activity code:** K01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $1
- **Award type:** 5
- **Project period:** 2017-06-01 → 2020-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9963220

## Citation

> US National Institutes of Health, RePORTER application 9963220, Using clinical data to identify FDA-approved drugs for cancer prevention and therapeutic repurposing (5K01ES028055-04). Retrieved via AI Analytics 2026-05-27 from https://api.ai-analytics.org/grant/nih/9963220. Licensed CC0.

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