How early retinal changes lead to retinoblastoma
Official title: Production and progression of premalignant retinoblastoma lesions.
Funded at Children's Hospital of Los Angeles · NIH-11170656 · Data updated 2026-11-12
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 type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hospital of Los Angeles NIH-funded |
| Lab location | 1 site (Los Angeles, United States) |
| Project start | March 1, 2011 |
| Project end | July 31, 2029 |
| Funding year | 2025 |
| Award (this year) | $560,197 |
| NIH project number | 5R01CA137124-13 |
| Project ID | NIH-11170656 on NIH RePORTER |
Who could benefit from this research
✓Could help
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.
✕Less likely to help
People without RB1-related risk or adults with unrelated eye diseases are unlikely to receive direct benefit from this project.
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.
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 gone: 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
-
Children's Hospital of Los Angeles
Los Angeles, United States Active
Researchers
- Principal investigator: Cobrinik, David — Children's Hospital of Los Angeles
- Study coordinator: Cobrinik, David
Related research
Other NIH-funded projects on the same topics.
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Understanding how premalignant retinoblastoma lesions form and progress in children
Researchers are studying how early retinal lesions in kids with RB1 loss become malignant, with the goal of finding ways to prevent retinoblastoma progression.
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Aqueous humor liquid biopsy to guide prognosis and monitoring in children with retinoblastoma
A liquid test using a small amount of eye fluid could reveal important tumor details to help predict how retinoblastoma in young children will behave and how well treatment is working.
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Parent-of-Origin Effects in Retinoblastoma: Epigenetic Drivers of Tumor Risk and Progression
A study using long-read DNA sequencing to understand how the parent of origin of RB1 gene variants affects retinoblastoma risk and progression, with potential to guide personalized monitoring and treatment.
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Retinoblastoma eye-sparing treatment for young children
A new, targeted eye treatment for young children with retinoblastoma aims to save eyes and reduce radiation exposure by delivering medicine directly into the eye.
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A tiny eye-fluid test to predict and track retinoblastoma in young children
This project uses a very small sample of eye fluid to look for tumor DNA that could help predict how retinoblastoma will behave and to follow treatment in children.
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Handheld wide-view 3D eye scanner to find tiny retinoblastoma tumors
This project is building a new handheld 3D eye scanner intended to help find very small retinoblastoma tumors early in young children who need screening.
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.