New treatment for retinoblastoma in young children
Official title: Retinoblastoma Phase II Expanded Access Clinical Trial
Funded at Targeted Therapy Technologies, LLC · NIH-11105727 · Data updated 2026-11-12
This study is testing a new way to treat retinoblastoma, a rare eye cancer in babies and young kids, by using a special device to deliver a chemotherapy drug right to the eye, which could help keep the cancer away without needing more intense treatments like surgery or radiation.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Targeted Therapy Technologies, LLC NIH-funded |
| Lab location | 1 site (Somerset, United States) |
| Project start | September 15, 2024 |
| Project end | June 30, 2028 |
| Funding year | 2024 |
| Award (this year) | $650,000 |
| NIH project number | 1R01FD008443-01 |
| Project ID | NIH-11105727 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are infants and young children diagnosed with retinoblastoma who have not responded to standard treatments.
✕Less likely to help
Patients with retinoblastoma who are not eligible for the trial due to age or those who have already undergone extensive treatment may not benefit from this research.
What this research studies
This research focuses on a novel treatment for retinoblastoma, a rare eye cancer affecting infants and young children. The approach involves using a specially designed Chemoplaque to deliver the chemotherapy drug Topotecan directly to the eye, minimizing systemic exposure and potential side effects. The trial aims to evaluate the effectiveness of this treatment in achieving sustained cancer remission without the need for more invasive standard care options, such as eye removal or radiation therapy.
Why it matters
Potential benefit: If successful, this research could provide a safer and more effective treatment option for children with retinoblastoma, potentially preserving their vision and reducing the risk of long-term complications.
How similar studies have gone: Previous research has shown promising results with similar localized chemotherapy delivery methods, indicating potential for success in this novel approach.
Where this research is happening
-
Targeted Therapy Technologies, LLC
Somerset, United States Active
Researchers
- Principal investigator: Carvalho, Ricardo a — Targeted Therapy Technologies, LLC
- Study coordinator: Carvalho, Ricardo a
Related research
Other NIH-funded projects on the same topics.
-
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.
-
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.
-
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.
-
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.
-
How early retinal changes lead to retinoblastoma
This project looks at how early abnormal cells in the retina form and later turn into retinoblastoma in children, using lab-grown retinal tissue.
-
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.
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.