Retinoblastoma Center of Excellence

RetinoblastomaRetinoblastoma (Rb) is a rapidly developing cancer that forms in the cells of the retina. In developed nations, Rb has one of the best cure rates of all childhood cancers (95-98 percent) with more than 90 percent of patients surviving into adulthood. Usually manifesting between the ages of 2 and 6, Rb is a very treatable cancer when it is detected early. However, in developing nations, the death rate can be as high as 70 percent due to late diagnosis.

Accepting the challenge of discovering new and better treatments for retinoblastoma, our multi-disciplinary Retinoblastoma Center of Excellence is a collaborative effort.

Our Research Process

The research process begins when a patient with retinoblastoma comes to Lurie Children's for a diagnosis. Marilyn Mets examines the patient and makes the diagnosis based on an ophthalmological examination and an MRI, B-scan ultrasonography or CT. Since there is no safe method for taking a biopsy of a retinal tumor currently at our institution, the Lurie Children's standard of care is to have two ophthalmologists agree on the diagnosis. Dr. Mets has two goals for each patient: to save the patient’s life and as much of their vision as possible.

Next, Joanna Weinstein of the Center for Cancer and Blood Disorders is called upon to discuss various treatment options with the patient. In advanced cases, enucleation is often the most appropriate option and the tissue samples are sent to Paul Bryar for classification. In less advanced cases, efforts to save the eye and vision are made. Current treatments include chemotherapy, laser or radiotherapy, but these have significant side effects that are either acute or latent. Clinical research has resulted in recently developed therapies, such as administering chemotherapy directly into an ophthalmic artery, which takes a collaborative effort between ophthalmology, interventional neuro-radiology and oncology. 

Paul Bryar, one of only a handful of ophthalmic pathologists in the U.S., then assesses the tumor specimen. After examining the tissue to identify the presence of certain high-risk features, he communicates his findings to the clinical team so they can modify the treatment plan as needed. Acting as a bridge between the clinical and basic scientists, he provides tissue to the Human Molecular Genetics Program for extraction and analysis of RNA and DNA.

Working at the bench is Nikia Laurie, who conducts microRNA profiling of the tumor samples from Dr. Bryar, endeavoring to discover the reasons why some genes and microRNAs are overexpressed and others underexpressed. MicroRNA profiling of the tumors may shed light on why some tumors are more likely to spread or become more invasive. These profiles will help to clarify the mystery of the disease and develop additional therapies. Additionally, since the behavior of microRNAs affects a wide range of cancers, discoveries regarding their effects should be germane to other cancers, both adult and pediatric.


Nikia A. Laurie, PhD

Marilyn B. Mets, MD

Joanna L. Weinstein, MD