Novel gene therapy

Published on Sep 11, 2004 04:44 PM IST

A novel gene therapy holds promise for treating aggressive superficial bladder cancer.

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PTI | ByAsian News International, Washington

Researchers at The University of Texas M. D. Anderson Cancer have claimed that a novel gene therapy strategy tested by them on mice holds much promise for treating aggressive human superficial bladder cancer.

The findings, published in the recent issue of Molecular Therapy, recognised that human bladder tumours growing in experimental mice substantially decreased in size after two treatments with the novel gene-based therapy with little or no evidence of cancer cells remaining in the bladder after treatment.

"Of course these results have been achieved in mice, not humans, but they are very exciting. I have never seen a potential therapy for superficial bladder cancer that could produce such marked regression of tumours within the bladder," said the lead researcher Dr. William Benedict, professor in the Department of Genitourinary Medical Oncology.

The researchers looked at use the of gene therapy to deliver interferon-alpha, an immune system modulator, which can improve a patient's natural response against cancer as well as kill cancer cells directly.

Interferon-alpha is commonly used as treatment in a number of cancers such as leukemias, lymphoma, melanoma and kidney cancer. However, it has been observed that tumour cells can become resistant to the immune protein.

"This is a major finding since many human bladder cancer cell lines are resistant to the interferon-alpha protein, including the ones used in this study. In addition, there was little apparent toxicity," said Benedict.

Until now, the researchers were using standard biological therapies like BCG or chemotherapy to cure bladder cancer patients, but these were not fully effective, as the tumours reappeared in nearly 50 percent of patients after treatment. Up to 30 per cent of patients were reported to have died from the disease.

Now the successful pre-testing of the gene therapy has provided a hope to the scientists to prevent bladder cancer from becoming aggressive.

"The degree of effectiveness of the Ad-IFN_/Syn3 therapy was a surprise to all of us. We know that going from mouse to man is a crucial step, but if the therapy performs half as well in the clinic as in this pre-clinical study, we may well significantly advance the care of patients with bladder cancer," Benedict concluded.

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