This study uses a new investigational (not yet approved by the FDA for widespread use) drug
called ZIO-101, an organic arsenical. You must be diagnosed to have relapsed/refractory
leukemia or lymphoma (blood cancer) and have tried other standard therapies.
This study is designed to determine whether ZIO-101 may be given safely. The study will also
test whether ZIO-101 helps to treat blood cancer.
We anticipate that approximately 22 to 35 patients will take part in this study.
Arsenic has been used as a medicinal agent for centuries in many different cultures. Most
recently in the United States, an inorganic arsenic compound was approved by the FDA for the
treatment of patients with relapsed acute promyelocytic leukemia (APL). However, use of
inorganic arsenic is limited by a narrow range of activity and systemic toxicity, most
notably of the cardiac system.
ZIO-101 is an organic arsenic derivative. In vitro testing in both the National Cancer
Institute (NCI) cancer cell panel and in vivo testing in a leukemia animal model demonstrated
substantial activity of SGLU against hematologic cancers. In vitro testing of SGLU using the
NCI human cancer cell panel also detected activity against lung, colon and brain cancers,
melanoma, and ovary and kidney cancers. Moderate activity was seen against breast and
prostate cancers cells. Data suggest that organic arsenic generates reactive oxygen species
in the cells to induce apoptosis and cell cycle arrest.