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Gene therapy hope for cancer patients
LONDON -- A gene therapy that has shrunk and even eradicated tumors in dying patients is being hailed by scientists as a significant breakthrough in the fight against cancer.
The therapy works by using a genetically engineered virus to home in on and then destroy cancer cells.
Researchers reporting in the journal Nature Medicine said the treatment had shown strong and lasting effects when combined with standard chemotherapy.
They said 25 out of 30 patients with head and neck cancer saw their tumors shrink. In eight cases the tumors disappeared completely and had not returned in the five months before the trial reported.
The researchers said most of the patients, who volunteered when critically ill, eventually died of their cancer because other untreated tumors in their body spread.
But doctors hope that if the virus passes the next trial stage, Phase III, and is approved for general use, it could cure some cancers.
David Kirn, the former head of the clinical research team that developed the virus, said: "This therapy, when moved to treat patients where the cancer is less advanced, could definitely lead to cures."
Kirn, who is now based at the Imperial Cancer Research Fund in London, added: "This is a very exciting step. It is going to be built on by everybody else out there."
The treatment, called ONYX-015, was developed by US biotech company Onyx Pharmaceuticals. Phase II trials were carried out on patients in the US and in Britain.
Phase III trials involve a larger number of volunteers and are the final phase aimed at determining whether a treatment or drug actually works.
Gene therapy pioneer Dr William French Anderson, of the University of California, said: "It is very encouraging because this is the first time there has been a Phase II trial where the tumors have gone away in a significant number of people and have not come back."
One weakness of the treatment is that it has to be injected into the growth to work, meaning it is of no apparent use against tumors that are difficult to reach or those that are too small to see.
It is hoped it may be possible to give ONYX-015 to patients intravenously, which would affect the whole body.
Dr Anderson, who conducted the first gene therapy experiment in 1990, said the experiment was one of several helping to rehabilitate the troubled field of gene therapy.
Recent setbacks have included the death last September of an 18-year-old US patient who was being treated for a rare liver disease among males. "With all its twists and turns, gene therapy seems to be turning the corner after a very bad year," Dr Anderson added.
He noted that French scientists had used gene therapy to cure two baby boys of a rare, inherited immune disorder called severe combined immunodeficiency, and said there had been successes against haemophilia, heart disease and now cancer.
Reuters contributed to this report.
Heart doctors report promising results with gene therapy
Nature Medicine magazine
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