Blocking glutamine uptake in MYCN‑amplified neuroblastoma

Targeting Glutamine Uptake via ASCT2 Inhibition in MYCN-amplified Neuroblastomas

NIH-funded research University of Kentucky · NIH-11251303

This project aims to stop growth of high-risk neuroblastoma with MYCN amplification by blocking a key glutamine transporter.

Quick facts

Grant typeNIH-funded research
Study typeNIH-funded research
Funding institutionUniversity of Kentucky NIH-funded
Lab location1 site (Lexington, United States)
Project IDNIH-11251303 on NIH RePORTER

What this research studies

Researchers will use patient-derived tumor tissue grown as 3-D organoids and patient-derived orthotopic xenografts in mice to study tumors with MYCN amplification. They will turn off the ASCT2 glutamine transporter using CRISPR-Cas9 and test a drug called V-9302 that blocks ASCT2 to see how tumors respond. Stable-isotope metabolomics will trace how cancer cells rewire their metabolism when ASCT2 is blocked. An unbiased CRISPR dropout screen will look for other genes that become essential when ASCT2 is inhibited, revealing possible combination targets.

Who could benefit from this research

Good fit: The work is most relevant to children with neuroblastoma whose tumors show MYCN amplification or to patients who can donate tumor tissue for research models.

Not a fit: Patients whose tumors do not have MYCN amplification or whose cancer does not rely on ASCT2-mediated glutamine uptake are unlikely to benefit from this approach.

Why it matters

Potential benefit: If successful, this work could point to new treatments that slow or stop growth and spread of MYCN-amplified neuroblastoma by targeting glutamine uptake.

How similar studies have performed: Preclinical studies have shown ASCT2 supports MYC-driven cancers and early lab tests of ASCT2 inhibitors are promising, but clinical benefit in patients has not yet been demonstrated.

Where this research is happening

Lexington, 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.