# A novel natural history study of medullary thyroid carcinoma: Incorporating the patient perspective to inform advanced disease management

> **NIH FDA R01** · UNIVERSITY OF TX MD ANDERSON CAN CTR · 2021 · $398,554

## Abstract

Abstract
Medullary thyroid carcinoma (MTC) is a rare tumor with a current prevalence in the United States of 13,500
Americans. Advanced disease is mortal; patients with extra-cervical metastases have 5- and 10-year survival
rates of 26% and 14%, respectively. After decades of limited effective available therapies, the Food and Drug
Administration recently approved two new drugs, vandetanib (2011) and cabozantinib (2012), for the treatment
of progressive, metastatic MTC; unfortunately, these drugs are not curative, response to therapy is not durable,
and individual institutional and investigator experience remains limited due to the rarity of the disease. As a
result, expert clinicians are left with unanswered questions regarding optimal drug use, and there continues to
be a need for innovative drug research. Quality of life was not definitively studied as a goal of therapy in the
trials for either drug, or little is known about how patients with advanced MTC make decisions regarding taking
these newly-approved drugs and/or participating in clinical trials. The goal of this research is to describe the
natural history of MTC via the utilization of a multi-institutional registry in order to understand the pathway to
progressive disease requiring drug therapy, and integrating for the first time the patient perspective in order to
identify patient reported outcome measures necessary to consider in current treatment and to include in clinical
trial design. This data will inform emerging drug therapies for advanced MTC.
This study will gather a comprehensive natural history to fully characterize the disease longitudinally from both
the clinical and patient perspectives to identify demographic, clinical, pathologic, and genotypic predictors of
progression to advanced MTC requiring approved drugs or enrollment in clinical trial, and patients’ response to
such therapy. Additionally, the study will characterize patients’ experience with the progressive stages of MTC
and treatment initiation through a systematic, longitudinal evaluation of patient-reported outcomes (PROs) that
are relevant to the disease, including quality of life, symptoms and other outcomes.

## Key facts

- **NIH application ID:** 10237146
- **Project number:** 5R01FD006650-03
- **Recipient organization:** UNIVERSITY OF TX MD ANDERSON CAN CTR
- **Principal Investigator:** Elizabeth Gardner Grubbs
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** FDA
- **Fiscal year:** 2021
- **Award amount:** $398,554
- **Award type:** 5
- **Project period:** 2019-09-01 → 2023-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10237146

## Citation

> US National Institutes of Health, RePORTER application 10237146, A novel natural history study of medullary thyroid carcinoma: Incorporating the patient perspective to inform advanced disease management (5R01FD006650-03). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10237146. Licensed CC0.

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