Columbia University – Lead Investigator Dr. Tito Fojo

Our group is examining SDHB loss in pheochromocytoma in two projects. We have begun contacting patients and families to create a registry in which we will ask patients to contribute blood samples. From these samples, we will look for evidence of succinate excess in an effort to develop an earlier biomarker for the disease. We believe that several signature genes will be downregulated in cells exposed to chronic levels of succinate. In the second project, we have begun looking for drug candidates that could turn off the excess succinate. Such an agent could be treatment for patients with overt malignancy. However, a nontoxic, low dose agent without significant side effects could also be considered as a preventive measure. Our laboratory has screened a large number of compounds in collaboration with the National Center for Advancing Translational Sciences (NCATS, https://ncats.nih.gov/). Several hits emerged from this study and are being validated and pursued. Our overarching goal is to find new treatment options for SDHB-loss pheochromocytoma and to find an option that can be used very early in the natural history of the disease.