"We've treated 13 patients in this first-in-human study and we've seen an exciting proof of concept with no significant drug-related toxicity," says principal investigator Charles Lu, M.D., associate professor in M. D. Anderson's Department of Thoracic, Head and Neck Medical Oncology.
Blinded analysis of pretreatment and post-treatment biopsies of three patients' tumors show that expression of FUS1 was absent from pretreatment samples while a high level of FUS1 was expressed in tumors after treatment. FUS1 can induce apoptosis - programmed cell death - in cancer cells but is frequently lost when normal cells become cancerous.
Lu presented a poster on the study on April 17 at the late-breaking abstract session of the American Association for Cancer Research annual meeting in Los Angeles.
Other attempts at gene therapy have employed an adenovirus to deliver the therapeutic gene. "Here we are using a non-viral, non-infectious delivery system," Lu says.
The only clinically significant side effect so has been fever, but Lu says premedication with a steroid and diphenhydramine has eliminated that so far.
Previous gene therapy clinical trials also involved direct injection into tumors. "This is the first time anyone has shown that a gene can be injected and then be taken up and expressed in cancer cells at distant sites," said Jack Roth, M.D., professor of the M. D. Anderson Department of Thoracic and Cardiovascular Surgery and a pioneer in the field of gene therapy.
FUS1 can induce apoptosis - programmed cell death - in cancer cells but is absent in
Source:University of Texas M. D. Anderson Cancer Center