2013

Dr. Anna Pawlowska

City of Hope

Ewing’s Sarcoma

Grant Amount: $37,000

Ewing’s sarcoma is the second most common primary bone tumor in children and adolescents.  While advances in treatment have increased survival rates for children with localized disease to nearly 70 percent, fewer than 30 percent of patients who experience relapse or the spread of their cancer survive.  In addition to these grim statistics, there is no nationally accepted “gold standard” of treatment for patients with advanced Ewing’s sarcoma.  More effective therapies are urgently needed to save the lives of young patients.

 City of Hope will be launching a five-year pilot clinical trial to study the effectiveness of a promising treatment for high-risk patients with advanced Ewing’s sarcoma: whole-body MRI-guided radiation therapy followed by autologous hematopoietic cell transplant, with a novel combination of high-dose chemotherapy.  Led by Anna Pawlowska, M.D., Clinical Professor of Pediatrics, the proposed study will assess whether this therapy is feasible, safe and likely to increase patient survival rates.  A similar approach tested in Germany and Austria increased children’s survival rate to 70 percent after two years and 56 percent after three years with minimal toxicity, but this approach has never been tested in the United States.

 In a novel approach, guests at the 2013 PHASE ONE Gala DIRECTLY donated $37,000 to help fund part of this five-year trial by supporting MRI tests for patients not covered by their insurance and/or support a portion of the salary of a Clinical Research Assistant.  This critical seed funding would help young patients with Ewing’s sarcoma access a promising new therapy now and start collecting the patient data needed to secure additional public and private funding for future years of the trial.