PCRF-funded researchers strive to understand and treat conditions such as Ewing sarcoma, the second most frequent bone disease among children. Led by Dr. Bryan Crompton of Dana-Farber Cancer Institute, a team of investigators strives to develop new drugs and predict which patients will need them.
The growth of Ewing sarcoma’s malignant cells is regulated by a protein known as focal adhesion kinase (FAK). Inhibiting FAK cells kills sarcoma cells, without creating tumors. Although no single drug can block FAK, Dr. Crompton and his team have found a group of chemicals that accomplish this vital task. PCRF supports laboratory testing of these drugs.
That is only part of the battle. Science has yet to discover which patients would respond to this treatment and which wouldn’t. Blood tests made possible by Dr. Crompton’s work promise to identify patients who fare poorly in early treatment and could benefit from another approach. Additionally, these tests could diagnose pediatric cancers, detect relapse, and discover resistance to treatments.