Enhancing Treatment Response in Recurrent/Metastatic Osteosarcoma with Hydroxychloroquine

Principal Investigator: Dr. Nancy Gordon
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Disease: Osteosarcoma

Research Description: Osteosarcoma (OS) is an orphan disease affecting approximately 400 children and adolescents in the US each year. It almost always metastasizes to the lungs, which often results in a fatal outcome. Patients with relapsed/recurrent disease have limited therapeutic options, and long-term survival rates are less than 20%. Autophagy is a survival mechanism that protects OS cells from dying upon stressful conditions such as chemotherapy, often leading to tumor cell survival. Hydroxychloroquine (HCQ) has been used to treat malaria for years, and it is known to block autophagy. This clinical trial will explore the feasibility, safety and potential efficacy of HCQ in combination with the currently recommended treatment combination of gemcitabine and docetaxel for patients with relapsed/recurrent OS. The team also plans to identify potential biomarkers to understand why certain patients may or may not benefit from adding HCQ. If successful, this research has the potential to improve survival for pediatric and adolescent patients with OS.

Funding Partners: Cures Within Reach

CWR funding role: Participating funder