This has been another exciting and productive year for research in the sarcomas of children and young adults. Let me highlight four major advances and approaches:
- We have successfully begun a clinical trial of a radically new approach for the treatment of Ewing sarcoma. Dr. Jeffrey Toretsky has developed a compound which completely stops the growth of Ewing sarcoma in the laboratory. In collaboration with colleagues at MD Anderson and UCLA, we have now enrolled 20 patients in a clinical trial to test this compound in children. We are required by the FDA to proceed slowly to prove that it is safe, but we are now reaching the doses that should have a beneficial effect. We are extremely excited about the possibilities for this line of investigation.
- We have designed a trial for newly diagnosed patients with Ewing sarcoma that incorporates into our initial therapy two new drugs that achieved a high rate of response in patients with recurrent Ewing sarcoma. Our preliminary data are showing that we have increased the probability for cure for children who present without metastatic disease to 95%, which is a dramatic increase over our historical results of 70%.
- Working with MSKCC’s Dr. Nai-Kong Cheung and his colleagues, we have developed an antibiotic that can help to control metastases of osteosarcoma, the most common form of primary bone cancer in children and young adults. We have treated over 40 children and young adults with this antibody. The results are sufficiently exciting and a company has agreed to sponsor a Phase III front line trial for patients with osteosarcoma which will be led by MSKCC but will include the participation of partners at other major cancer centers around the US.
- Dr. Neerav Shukla, another MSKCC pediatric oncologist, has focused research on the genomics of pediatric tumors including Ewing sarcoma. He has developed an extremely sensitive assay to test for the presence of a DNA sequence unique to the tumor in 40 patients with Ewing sarcoma and it has shown exciting promise as an early warning detector for tumor recurrence before it becomes detectible by scans or X-rays. His exciting work has been published in the Journal of Precision Oncology and led to a leadership role in the Children’s Oncology Group to extend this work nationwide.
So to all Margaux’s Miracle Foundation supporters, I urge you to help us continue these vital efforts to eradicate these deadly and rare cancers. I look forward to seeing you on March 5th.