gene therapy offers hope

[nadri]

Came across this and thought it was interesting.

Sarah

Chinese gene therapy offers hope to terminally ill cancer patients

By Adam Luck in Hong Kong

(Filed: 04/07/2004)

Terminally ill cancer patients from Europe and America are travelling to China for treatment with a revolutionary anti-tumour drug - the world's first officially licensed "gene therapy".

The Chinese government approved the manufacture and use of the drug at the end of last year after clinical trials found that it markedly improved the survival rate for patients with cancer of the head and neck. Doctors are now extending the treatment, named Gendicine, to patients with lung and stomach cancer.

Injected directly into the tumour, Gendicine works by, in effect, programming cancer cells to commit suicide. About 400 patients - 20 of them from abroad - have been treated with the drug in eight-week courses which cost the equivalent of £1,800.

Peng Zhaohui, a medical scientist who helped develop Gendicine, said: "I believe this revolutionary form of gene therapy represents the future for treating cancer patients.

"There is still a lot of work to be done, but the results have been very promising so far and - unlike experimental forms of gene therapy attempted elsewhere - there is no evidence of serious side-effects. We have treated foreign patients successfully and word is beginning to spread."

Mr Peng has been inundated with requests from overseas, including Britain, from patients desperate to try Gendicine. Arthur Winiarski, 48, a Polish-American businessman based in Warsaw, who was diagnosed with squamous cell carcinoma in his sinuses after a routine medical check-up 18 months ago, was one of them.

After operations, radiotherapy and chemotherapy in Poland, America and Germany, he was told by doctors at a hospital in Berlin that he had almost no hope. "They wanted to get rid of me. The only thing they could suggest was chemotherapy but they and I both knew this does not work with this kind of cancer," he said.

Then a friend saw a sentence about the new drug on the internet, which mentioned Mr Peng. Mr Winiarski emailed a short history of his case to the scientist. To his astonishment, within three hours came a reply: "I think I will be able to help you."

Still sceptical about his prospects, Mr Winiarski travelled to China and presented himself to Dr Niu Qi, an oncologist at Beijing's elite Tongren hospital, in April. He was told he needed surgery as well as the drug treatment.

He said: "They inject the drug into the tumour and it goes bananas: it invades itself and commits suicide. My tumour was so big - the size of a fist - that they had to use the drug to shrink it first."

After further doses of Gendicine Mr Winiarski was given the all-clear. He said: "To have a life like mine, to be a successful businessman, with everything going well and then all of sudden be told you have months to live - you cannot understand. This experience has been incredible and you could not believe the care I have had. I came here in desperation and they have treated me like a king."

Gendicine works by inserting a gene, called p53, into a virus, which is then injected into patients. The gene is naturally present in healthy cells but is "switched off" or mutated in many cancer patients. When reinserted into tumour cells by the virus, it triggers their self-destruction.

Mr Peng's company SiBiono, in Shenzhen, southern China, first tested Gendicine on head and neck squamous cancers which are relatively common in China.

In a clinical trial 120 patients were treated, some with the drug combined with radiotherapy, others with radiotherapy alone. All were then monitored for more than a year.

There was complete regression of the tumours in 64 per cent of those given the drug, three times the rate of those given radiotherapy alone. The only recorded side-effect was a mild fever.

Mr Peng said: "I don't think that there are likely to be any long-term effects because Gendicine cannot replicate in the patients' cells."

China's State Food and Drug Administration approved use of the drug in October and SiBiono was granted permission to begin manufacturing it in February. Prof French Anderson, director of Gene Therapy Laboratories at the University of Southern California, who is widely regarded as the father of gene therapy, has given the production plant his approval. He said: "It is an impressive manufacturing facility."

Prof Anderson said that the adenovirus being used by the Chinese to deliver the p53 gene to cells had largely been abandoned by Western scientists. He said, however: "Sometimes simpler is best."

Mr Winiarski's oncologist, Dr Niu, who trained at Harvard, said that his patient would need to return to China for further checks, but added: "So far this has been very good and I would be happy to use it with other patients."

Dr Niu said: "I plan to use it for liver cancer and gastric cancer because p53 is widely mutated in other forms of cancer, not just head and neck. My colleagues are excited by this and want to try it."

Mr Peng said: "This is no overnight success. It has taken 15 years of development and several years of clinical trials before we have reached this stage.

"There is still work to be done but we are proud of what we have achieved."

SiBiono has held "preliminary discussions" with Western pharmaceutical companies about possible manufacture and sale of the drug abroad, where testing requirements are far stricter.

8 June 2004[Health]: Deadly genes that run in families

10 November 2002: Cancer scientists closing in on key 'death gene'

Related reports

health.telegraph

External links

Cancer gene therapy is first to be approved [28 Nov '04] - New Scientist

Cancer Research UK

SiBiono (in Chinese)

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[Elaine]

Thanks for this information. Let us know any updates.

elaine