New ways to control the Notch1 protein in T-cell leukemia

New control of oncogene activation in T-cell leukemia

['FUNDING_R01'] · WISTAR INSTITUTE · NIH-11299573

Researchers aim to lower activity of the Notch1 protein to help children and adults with T-cell acute lymphoblastic leukemia.

Quick facts

Phase['FUNDING_R01']
Study typeNih_funding
SexAll
SponsorWISTAR INSTITUTE (nih funded)
Locations1 site (PHILADELPHIA, UNITED STATES)
Trial IDNIH-11299573 on ClinicalTrials.gov

What this research studies

This project builds on lab discoveries in fruit flies and mice showing that a protein called UVRAG can change how the cancer-driving Notch1 protein is broken down. Scientists will use mouse T-ALL models and molecular lab experiments to map how UVRAG and the endo-lysosomal system control Notch1 before it becomes active. The team will look for genes and pathways that could be safer targets than currently toxic Notch1 inhibitors. Findings may point to new drug targets that could make treatment for Notch1-driven T-ALL more effective and less damaging.

Who could benefit from this research

Good fit: Children and adults with T-cell acute lymphoblastic leukemia that is driven by Notch1 alterations would be the most relevant patients for future therapies from this work.

Not a fit: Patients whose leukemia is not driven by Notch1 or those needing immediate clinical treatment are unlikely to gain direct benefit from this laboratory-focused project.

Why it matters

Potential benefit: If successful, this work could identify safer ways to remove or weaken Notch1-driven leukemia cells and lead to better treatments with fewer side effects.

How similar studies have performed: Previous drug attempts to block Notch1 have struggled with toxicity, and targeting UVRAG-mediated degradation is a newer, largely preclinical approach.

Where this research is happening

PHILADELPHIA, 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.