Blocking cancer exosomes to help immunotherapy work better
Evaluating and Optimizing Novel nSMase2 Inhibitors in the Treatment of Cancers
['FUNDING_R01'] · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · NIH-11115683
New oral drugs that block an enzyme called nSMase2 aim to help adults whose cancers don't respond to immune checkpoint therapy.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF CALIFORNIA, SAN FRANCISCO (nih funded) |
| Locations | 1 site (SAN FRANCISCO, UNITED STATES) |
| Trial ID | NIH-11115683 on ClinicalTrials.gov |
What this research studies
Researchers are improving a first-in-class compound (PDDC) that blocks nSMase2, a protein cancer cells use to produce exosomes that blunt anti-tumor immunity. They will use medicinal chemistry to make compounds more potent, soluble, and selective, then measure drug levels and protein binding in laboratory tests. Promising leads will be tested in multiple animal tumor models together with immune checkpoint inhibitors to see if they restore immune responses and stop tumor growth. The goal is to select a candidate with good safety and exposure to advance toward early human trials.
Who could benefit from this research
Good fit: Adults with solid tumors that have not responded to immune checkpoint inhibitors would be the most likely future candidates for this approach.
Not a fit: Children, patients whose cancers are driven by different resistance mechanisms, or those whose disease is already controlled by other effective therapies are unlikely to benefit from this specific approach.
Why it matters
Potential benefit: If successful, these drugs could help more patients' tumors respond to immunotherapy and shrink cancers that previously did not respond.
How similar studies have performed: Genetic nSMase2 loss and the early compound PDDC showed encouraging results in mouse models, but no nSMase2 inhibitor has yet reached human trials.
Where this research is happening
SAN FRANCISCO, UNITED STATES
- UNIVERSITY OF CALIFORNIA, SAN FRANCISCO — SAN FRANCISCO, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: BLELLOCH, ROBERT — UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
- Study coordinator: BLELLOCH, ROBERT
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.