Targeting specific proteins to treat rare appendix cancers

CDK4/6 inhibition: a novel therapeutic strategy for GNAS-mutant gastrointestinal malignancies

['FUNDING_R21'] · UNIVERSITY OF CALIFORNIA, SAN DIEGO · NIH-10675743

This study is looking at a new way to treat a type of appendix tumor that can become aggressive, by using a special treatment that targets certain proteins, and it aims to help patients with these tough cancers feel better and have better outcomes.

Quick facts

Phase['FUNDING_R21']
Study typeNih_funding
SexAll
SponsorUNIVERSITY OF CALIFORNIA, SAN DIEGO (nih funded)
Locations1 site (LA JOLLA, UNITED STATES)
Trial IDNIH-10675743 on ClinicalTrials.gov

What this research studies

This research investigates a new treatment approach for mucinous neoplasms of the appendix, which can progress to aggressive cancer. The study focuses on inhibiting cyclin-dependent kinases (CDK) 4 and 6, which may be particularly effective against these tumors. By understanding the genetic mutations involved, particularly in the GNAS oncogene, the research aims to develop targeted therapies that could improve outcomes for patients with these challenging malignancies. Patients may be treated with this novel approach to potentially enhance the effectiveness of their cancer care.

Who could benefit from this research

Good fit: Ideal candidates for this research are patients diagnosed with mucinous neoplasms of the appendix or related gastrointestinal malignancies, particularly those with inoperable disease.

Not a fit: Patients with benign appendiceal conditions or those with other types of cancers not related to GNAS mutations may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to more effective treatments for patients with aggressive appendix cancers, improving survival rates and quality of life.

How similar studies have performed: Previous research has shown promising results in targeting CDK4/6 in other cancer types, suggesting potential for success in this novel application.

Where this research is happening

LA JOLLA, UNITED STATES

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.

View on NIH RePORTER →

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.