The 2026 Childhood Cancer Report
From diagnosis to research: measuring momentum in the search for cures
Retinoblastoma
Retinoblastoma is a pediatric cancer that occurs in the developing retina and often results in removal of the impacted eye. More than 98% of retinoblastoma tumors form due to a genetic mutation in the tumor in a gene called RB1. Early groundbreaking studies found that 45% of retinoblastoma was inherited, meaning that families carried a gene that causes eye cancer. For kids with the inherited type of retinoblastoma, the disease was most frequently present in both eyes. These early studies were the first time cancer was ever linked to an inherited gene, opening the door to understanding inherited cancer risks for all human cancers.146,147
Retinoblastoma tumors begin in the retina of the eye, with the potential to spread outside the eye to the optic nerve, brain, and throughout the lymph nodes, liver, bones, and lungs if not treated.148
Pediatricians, parents, and other caregivers most frequently notice a glow in the eye, which is medically referred to as leukocoria. Recognizing leukocoria facilitates early detection, and treatment can save both sight and lives.
| Age at Diagnosis | Five-year survival rates149 |
|---|---|
| Ages > 1 | 98.4% |
| Ages 1-4 | 96.2% |
| Ages 5-9 | 100% |
Retinoblastoma cases among children ages 10-19 are extremely rare and the sample size is too small to produce a reliable survival estimate.
The Promise of Liquid Biopsies
Pediatric oncologists often wrestle with a core dilemma: how to treat cancer aggressively without causing lasting harm. In retinoblastoma, removing the eye can cure the disease, but the child will lose their eye and vision.
Traditional biopsies aren’t possible in the retina, but researchers have pioneered a new approach: liquid biopsy. By analyzing a tiny sample of aqueous humor (the fluid in the front of the eye) doctors can gauge tumor aggressiveness, track disease, and tailor treatment. The result: a better chance of saving not just the child’s life but also their vision.
The use of liquid biopsy can also help with diagnosis in unusual cases and can even help detect recurrences before they can be seen visually by the doctor.
Breakthroughs and Research Milestones:
- Laser Therapy (1960s):
A laser beam is directed in the pupil of the patient. The high temperature of the laser is able to kill the tumor cells within the eye.152 - Cryotherapy (1960s):
A metal probe is cooled to very low temperatures and placed on the surface of the eyeball near the tumor. The very low temperature of the probe is able to kill the surrounding tumor cells on the surface of the eye. This is still used today, but in a very limited capacity.153 - External beam radiotherapy (1980s):
Radiotherapy to help kill cancer cells.154 - Chemotherapy (1990-present):
The most common chemotherapy agents used for treating retinoblastoma are vincristine, etoposide, and carboplatin.155 One method of delivering these chemotherapy agents is through a technique called intra-arterial chemotherapy, which delivers chemotherapy agents directly to the ophthalmic artery, which is the primary blood supply source to the eye.156 - Plaque brachytherapy (1993-present):
A radioactive plaque that is made of metal and radioactive material, such as Iodine-125 or Ruthenium-106, is surgically stitched to the surface of the eye, allowing for a continuous low dose of radiation to be administered directly to the eye.157
Retinoblastoma, by the Numbers in the U.S.
199 new cases in 2023150
Standard Treatments
- Surgery
- Chemotherapy
- Specialized radiation
Types of Retinoblastoma
Retinoblastoma can occur in one eye (unilaterally) or both (bilaterally). There are two types:
- Hereditary: 45% (often bilateral)
- Nonhereditary: 55% (typically unilateral)151