Nanoliposome Delivery of Ceramide to Cancer Cells

1532GCC: Phase I Study of C6 Ceramide NanoLiposome in Patients with Advanced Solid Tumors

PI:  Edward Sausville, MD, PhD
Professor of Medicine, University of Maryland School of Medicine
Co-Investigator:  Yixing Jiang, MD
Associate Professor of Medicine, University of Maryland School of Medicine
Contact:  Cheryl Young, Study Coordinator

For about two decades, ceramide has been known to activate apoptosis in cancer cells while showing no toxicity to normal cells, but it has not been applied therapeutically because it is hydrophobic, insoluble and cannot be given to patients intravenously without inducing hemolysis. However, a C6 nanoparticle called NanoLiposome has the ability to encapsulate ceramide and act as a delivery mechanism of this agent to tumor cells.

The University of Maryland Marlene and Stewart Greenebaum Comprehensive Cancer Center (UMGCCC) is the lead institution in a trial funded by the National Cancer Institute through a Small Business Innovation Grant to Keystone Nano, a small pharmaceutical company seeking to create new therapies that are more selective and less toxic. This trial will investigate the safety and efficacy of using Ceramide NanoLiposome. Because NanoLiposome acts as a protective membrane until the molecule can be taken up by the cancer cell, it is thought that Ceramide NanoLiposome will be well-tolerated by patients.

Cohorts of one new patient will be enrolled at each dose level, and if there is no grade 2 or higher toxicity after one cycle of treatment, a second patient will be enrolled at the next escalated dose level. This will continue until a participant experiences a grade 2 toxicity (excepting alopecia), and then the study will follow a 3+3 design at the acceptable dose level for further cohorts. Treatment will be given on an outpatient basis, and participants will be monitored for safety assessment and pharmacokinetics sampling for at least three hours following treatment.

This trial is currently open to patients with any advanced solid tumor who have no curative treatment options or standard chemotherapy for their cancers. Future phases of the trial will test Ceramide NanoLiposome for hepatocellular cancer, of which UMGCCC treats a high volume of cases and for which there is no known effective chemotherapy. The trial will also be open at the Medical University of South Carolina and the University of Virginia.

If this trial shows that NanoLiposome is safe and useful, this nanoparticle could be the basis for the delivery of other therapeutic agents as well.

For more information about this trial at UMGCCC, contact the Study Coordinator: Cheryl Young, 410-328-8611.