Jonathan Kim Laboratory
Targeted eye therapies
Retinoblastoma is a very rare cancer that develops in the eyes of young children, affecting one in 18,000 babies in the United States. Although rare, retinoblastoma is devastating when it occurs, since in the majority of cases there is no family history and therefore no warning. Although there are many effective therapies available to treat retinoblastoma, we face many challenges in trying to save the vision and the lives of affected children. Retinoblastoma develops in the retina of the developing eye due to a specific mutation in a gene on chromosome 13. With proper diagnosis and treatment, spread outside the eye is fortunately rare, but many children lose their vision and sometimes the eye itself.
From an epidemiologic standpoint, there are many challenges to preventing retinoblastoma. In most children, the cancer develops from a random, rare mutation that cannot be predicted based on any historical or clinical factors. Typically, the diagnosis cannot be made until the tumor becomes quite large and causes signs such as leukocoria (white pupil reflex) or strabismus. Therefore, even well-trained pediatricians are not able to diagnose retinoblastoma at an early stage since there are no external signs of abnormalities and vision in pre-verbal children cannot be accurately measured.
As the Director of the Retinoblastoma service, my role is to provide the full spectrum of clinical services when a child is diagnosed with retinoblastoma. I follow the child from the time of diagnosis to the end of their treatment course as adults. At Children's Hospital Los Angeles, we offer all of the treatments available in the world, including laser photocoagulation, systemic, regional and ocular chemotherapy as well as brachytherapy and external beam radiation treatment.
Our main research mission is to develop targeted therapies for retinoblastoma that will kill cancer cells while sparing other normal cells. Unlike traditional therapies such as chemotherapy and radiation, targeted therapies have the potential to achieve cancer cures while minimizing side effects. To accomplish this goal, we need partnerships between experts in different fields, from cell biologists to radiation oncologists to hematology oncologists. We also need collaboration between clinicians and basic science researchers at The Saban Research Institute. The basic paradigm for developing a new therapy starts with an idea based on a clinical need, followed by development of a research strategy based on basic research principles. We may test the therapy on retinoblastoma cells in a test tube (in vitro), followed by an animal model (in vivo). If promising results are achieved, then a limited clinical trial on carefully selected patients may follow. Translational research is the key to achieving progress in this field, and we are committed to developing new therapies to improve clinical outcomes in our children with retinoblastoma.