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AACR: Nanoparticle Gene Therapy Studied in Lung Cancer

Researchers show for first time that gene therapy can be administered intravenously

WEDNESDAY, April 18 (HealthDay News) -- A novel intravenous gene therapy administered via a lipid nanoparticle has been tested in patients with stage 4 lung cancer, according to research presented this week at the American Association for Cancer Research annual meeting in Los Angeles.

In a phase 1 clinical trial, Charles Lu, M.D., of the University of Texas M.D. Anderson Cancer Center in Houston, and colleagues studied 13 stage 4 lung-cancer patients who had not responded to front line cisplatin combination chemotherapy. All patients received injections of the apoptosis-inducing gene FUS1, which is often lost in cancer cells.

After treatment, the researchers found that high levels of FUS1 were expressed in the patients' cancer cells. Fever was the only clinically significant side effect, but the researchers found they could control it by premedicating patients with a steroid and diphenhydramine. Of the eight patients who received two or more doses of FUS1, three experienced stabled disease for three to seven months, which is similar to the median seven-month survival time for patients receiving second line therapy.

"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," Jack Roth, M.D., of the M.D. Anderson Department of Thoracic and Cardiovascular Surgery, said in a statement.


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