How early retinal changes lead to retinoblastoma

Production and progression of premalignant retinoblastoma lesions.

NIH-funded research Children's Hospital of Los Angeles · NIH-11170656

This project looks at how early abnormal cells in the retina form and later turn into retinoblastoma in children, using lab-grown retinal tissue.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionChildren's Hospital of Los Angeles NIH-funded
Lab location1 site (Los Angeles, United States)
Project IDNIH-11170656 on NIH RePORTER

What this research studies

Researchers grow miniature human retinas in the lab to mimic early events that happen when the RB1 gene is lost in cone precursor cells. The team watches how these cells divide, stay quiet as premalignant lesions, or later form tumor-like foci, and they measure genetic and epigenetic changes over time. By comparing stages that stay dormant with those that progress, they aim to find molecular switches that let tumors start or stay stopped. The work uses stem cell–derived organoids, genetic tools, and molecular analyses to follow these changes at tissue ages similar to those seen in children.

Who could benefit from this research

Good fit: Ideal candidates would be infants or children with a known germline RB1 mutation or a family history of retinoblastoma who might provide clinical samples or be considered for future trials.

Not a fit: People without RB1-related risk or adults with unrelated eye diseases are unlikely to receive direct benefit from this project.

Why it matters

Potential benefit: If successful, this work could point to ways to prevent or delay retinoblastoma in children at genetic risk and to markers for earlier monitoring.

How similar studies have performed: Lab-grown retinal organoid models have previously reproduced early retinoblastoma-like changes, but turning those findings into patient treatments remains novel.

Where this research is happening

Los Angeles, 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.
Conditions Cancers
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.