FUS1 was discovered by a research team led by Roth at M. D. Anderson and by John Minna, M.D., of the Department of Internal Medicine and Pharmacology, Hamon Center for Therapeutic Oncology Research, at The University of Texas Southwestern Medical Center at Dallas. Roth and Minna are the co-principal investigators of a National Cancer Institute Specialized Program of Research Excellence in Lung Cancer.
"As a clinician, I am very cautious about saying that we have shown clinical activity at this point. We have some encouraging data. The number of patients is too small to draw any definite conclusions, however," Lu said.
Three patients of eight who received two or more doses experienced stable disease for three to seven months. Median survival time for all patients is 14.6 months, which Lu notes compares favorably to a seven-month median survival time for patients receiving second line therapy.
All patients on the trial had been treated with front line cisplatin combination chemotherapy, which failed to halt their disease. The clinical trial continues. No maximum tolerated dose has been established, Lu says.
The nanoparticle delivery system consists of a plasmid gene expression cassette loaded with DNA that encodes the FUS1 protein. This is wrapped tightly in a form of cholesterol to protect it from the body's defense mechanisms. The nanoparticles accumulate mainly in the lungs, particularly in the tumors, where the genes repeatedly express FUS1 tumor-suppressing proteins.
Lung cancer is the leading cause of cancer death in the United States, causing 160,000 deaths annually. About 80 percent of lung cancer is of the non-small cell type.