Project title: Defining Distinct Subtypes of NECs to Predict Therapeutic Vulnerabilities

Carl Gay, MD, PhD University of Texas MD Anderson Cancer Center

Carl Gay, MD, PhD
  • Status: Active
  • Year(s): 2021
  • Grant Type: Martha O’Donnell Pagel Pilot Research Award


Gay is conducting a small clinical trial on extrapulmonary neuroendocrine small cell cancer and will initiate cell model and genetic analysis studies on these extremely rare tumors.

What question will the researchers try to answer?

Our current understanding, or lack thereof, of extrapulmonary small cell carcinomas is limited by the lack of available resources on which to perform profiling and to test novel therapeutics. Dr. Gay and his team hope to develop new platforms derived from these rare tumor samples to characterize and use to test hypotheses for more effective treatment strategies. 

Why is this important?

This work represents a critical initial step toward establish extrapulmonary small cell carcinomas – not as an umbrella term describing a host of rare diseases, but as a unified disease type worthy of translational and clinical focus. 

What will researchers do?

They have ongoing clinical trials focused specifically on this rare patient population and, with the gracious participation of these patients, they will collect blood and tissue samples from each of them that will be banked or used to generate cell or mouse models. These banked samples will be subjected to analysis of their DNA, RNA, and/or protein landscapes. All of the results of these analyses, as well as the models generated, will be shared with the NET research community. 

How might this improve the treatment of NETs?

Extrapulmonary small cell carcinomas are extremely aggressive and lack any standard of care. This project will form the groundwork for future trials aimed specifically at this underserved population. 

What is the next step?

Once models are established and data is generated representing the molecular landscape of these tumors, rational therapeutic strategies can be proposed and, for the first time, be tested directly in optimized models of this disease. 

Additional Details

  • City: Houston
  • State: Texas
  • Country: USA
  • Grant Duration: 1 year
  • Sponsor: Martha O’Donnell Pagel Fund for Research of Rare Cancers


NETRF funds laboratory research to understand the development of neuroendocrine tumors and translational research to explore new concepts in treatment. Research grant descriptions and research updates from NETRF are not intended to serve as medical advice. It can take years for research discoveries to be fully validated and approved for patient care. Always consult your health care providers about your treatment options.

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