Dr. Brian Crompton is a physician-scientist at Dana-Farber Cancer Institute and his research focuses on finding new treatment approaches for patients with Ewing sarcoma. Ewing sarcoma is an often devastating pediatric cancer of the bone whose only effective treatment employs intensive chemotherapy, surgery, and radiation. Even with these aggressive treatments, less than 75% of children with this disease are cured and that rate has changed very little over the past few decades. There are two major challenges that we need to overcome to improve our treatment approach: we need better drugs and we need to be able to predict which patients need these new treatments.
Through work funded by the Pediatric Cancer Research Foundation, Dr. Crompton is making progress on overcoming both of these challenges. In recent years, he has found that Ewing sarcoma cancer cells are dependent on the activity of a protein called focal adhesion kinase also know as FAK. When FAK is inhibited, Ewing sarcoma cells die and can’t form tumors. Dr. Crompton is now partnering with a major pharmaceutical company that has multiple FAK inhibitors in clinical trials for adult tumors to complete necessary experiments to test the effects of FAK inhibition in a clinical trial for patients with Ewing sarcoma.
However, we know that a single new drug will not be enough to cure patients with this disease. Therefore, Dr. Crompton and his colleagues have now screened a library of nearly 2,000 cancer drugs and found a class of compounds that are highly synergistic in Ewing sarcoma when combined with FAK inhibitors. Through the support of the Pediatric Cancer Research Foundation, he is now testing the effect of this drug combination on tumor progression in laboratory models of this disease.
However, it is still unknown exactly which patients would benefit most from new treatments. For the majority of the newly diagnosed patients with Ewing sarcoma, we are currently unable to predict which patients will be cured with standard therapy and which ones will relapse and ultimately succumb to their disease. With the help of the Pediatric Cancer Research Foundation, Dr. Crompton is developing a blood test that will help to track an individual patient’s response to therapy. This assay should enable identification of patients who are not responding well to therapy very early in their treatment course and eventually allow the development of patient-specific, risk-stratified approaches to treatment.
Some people say that children make up 10% of our cancer population, but they are 100% of our future! ~ Dr. Alex Huang, Case Western