Project 1: Phase I/II clinical trial studying the safety and efficacy of HDAC inhibition in combination with doxorubicin for patients with advanced sarcomas resistant to chemotherapy. This is a clinical trial that directly developed out of observations in our laboratory showing that an inhibitor of HDAC, PCI-24781, can reverse doxorubicin resistance in osteosarcoma cell lines. If successful, HDAC can become a useful target for future treatment of osteosarcomas and other sarcomas.
Project 2: High-throughput oncogene genotyping of osteosarcoma tumor samples.
We have one of the world's largest collection of tumor samples from patients with osteosarcoma. In collaboration with laboratories at DFCI and MIT, we have been able to interrogate 100 osteosarcoma tumors and cell lines for mutational status at over 1000 points across the human genome. We hope that our discoveries can lead to better understanding of the cause and origin of osteosarcomas, thereby leading to better ways to treat and cure this disease.
Project 3: I am working with Amgen in a proposal to use a drug that inhibits RANKL to treat patients with advanced osteosarcomas. This drug has already proven to be substantially beneficial to patients with another bone tumor called Giant Cell Tumor. If approved, this drug can potentially be the first major advance in the treatment of metastatic osteosarcoma in twenty years.