Childhood Retinoblastomas
Retinoblastoma is a malignant tumor that develops in the retina, the layer of light-sensitive tissue at the back of the eye. This tumor arises from immature retinal cells called “developing retinal cells.” Retinoblastoma is a cancer that mainly affects young children, typically before the age of 5 (95% of cases). Estimates suggest that about 50 to 60 new cases are diagnosed each year in France (approximately 2% of pediatric cancers).
The treatment of retinoblastoma depends on the size, location, and spread of the tumor. Treatment options may include surgery, radiotherapy, chemotherapy, or a combination of these methods. The prognosis for children with retinoblastoma also depends on the extent of the disease and how quickly it is diagnosed and treated.
About the Project
Thanks to research, the survival rate for retinoblastoma is now excellent in the most developed countries (survival of over 98%), but this disease can severely compromise a child’s vision. Current treatments for retinoblastoma involve chemotherapy administration and ophthalmologic treatments (laser, cryotherapy, etc.). In some advanced cases, surgical removal of the eye may still be necessary. Tumor invasion of the vitreous (the central part of the eye) remains problematic and requires multiple injections of chemotherapy directly into the eye (intravitreal injections).
The most commonly used molecule currently is melphalan. While this treatment is effective, it remains toxic to the eye and can severely compromise a child’s vision after treatment, or even the preservation of the eye. Recent work conducted in laboratories at the Institut Curie has shown that intravitreal injection of carboplatin (a chemotherapy molecule already used intravenously for retinoblastoma treatment) is equally effective as melphalan and significantly less toxic.
Dr. Malaise’s team aims to conduct an early-phase clinical study (Phase I) to evaluate the toxicity and safety of intravitreal carboplatin injections in children with retinoblastoma. Current treatments with intravitreal melphalan injections can be toxic to the structures inside the eye. This toxicity can compromise a child’s vision but may also require premature cessation of anti-tumor treatments and justify surgical removal of the eye. Since retinoblastoma affects both eyes in nearly half of cases, limiting ocular toxicity of treatments and maintaining the best possible visual acuity remain crucial for young children (visual development, schooling, autonomy, etc.).
This clinical research program is unique: Institut Curie is the only center in France authorized to manage retinoblastoma and therefore the only one capable of conducting clinical studies on this disease. Additionally, there is no reliable data regarding the use of intravitreal carboplatin injections for this tumor.
Initially, the team will determine the maximum dose of carboplatin that can be safely and non-toxically administered via intravitreal injection in children with retinoblastoma. Subsequently, this dose will be used in a Phase II clinical study to also validate the efficacy of carboplatin compared to the current standard treatment.
Project Summary
- Promoter: Institut Curie
- Principal Investigator: Dr. Denis Malaise
- Program Duration: September 2022 – September 2025
- Number of patients: 22
- Countries involved: France
- Funding from Imagine for Margo: €50,000
This trial was co-funded by the Rallye du Cœur de Paris 2022 and was selected by the SFCE as part of its 2021 call for projects.
