Blocking CDC42 to help immune cells fight cancer

Rational targeting of Cdc42 to benefit immunotherapy

NIH-funded research Cincinnati Childrens Hosp Med Ctr · NIH-11238882

This project explores a drug that blocks CDC42 to weaken immune-suppressing Treg cells so cancer immunotherapies can work better for people whose tumors don't respond now.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionCincinnati Childrens Hosp Med Ctr NIH-funded
Lab location1 site (Cincinnati, United States)
Project IDNIH-11238882 on NIH RePORTER

What this research studies

From a patient's perspective, researchers are working to make immune-based cancer treatments more effective by targeting a protein called CDC42 that helps regulatory T cells (Tregs) keep immune responses in check. In lab models they reduce CDC42 activity genetically and with a small molecule called CASIN to make Tregs less stable, which appears to boost anti-tumor T cells. The team plans to combine CDC42 inhibition with existing immune checkpoint drugs to see if the combination produces stronger tumor control without triggering harmful autoimmunity. Most work is preclinical in controlled lab and animal models at Cincinnati Children’s, with the goal of guiding future clinical trials.

Who could benefit from this research

Good fit: Ideal candidates for future trials would be people with cancers that are not responding adequately to current immunotherapies such as checkpoint inhibitors or CAR-T cell treatments.

Not a fit: Patients whose tumors are not driven by Treg-mediated suppression or who have active autoimmune disease may not benefit and could face higher risk of immune side effects.

Why it matters

Potential benefit: If successful, this approach could help more people respond to immunotherapy by removing a brake on the immune system that allows tumors to hide.

How similar studies have performed: Early preclinical experiments showed that partial loss of CDC42 or the drug CASIN can destabilize Tregs and boost anti-tumor immunity in animal models, but human testing has not yet been reported.

Where this research is happening

Cincinnati, 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.
Last reviewed 2026-06-13 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.