New prostate cancer drug helps victims live five months longer

This is of course encouraging but the real question is not the mean survival time conferred by the drug -- which could be seen as trivial -- but the variance. Do some patients live a LOT longer? That would be impressive

An experimental drug extended the lives of men with advanced prostate cancer by almost five months in a new trial. Independent monitors halted the trial early because of the clear benefit to men taking the new pill.

An interim analysis of the trial of 1,199 men who were previously treated with chemotherapy found that the drug – MDV3100 – improved overall survival by 4.8 months compared with a placebo. Average survival for men treated with the drug was 18.4 months, while those who were treated with a placebo survived for 13.6 months.

The findings were assessed after 520 men taking part in the trial had died, and have boosted market ratings for the U.S. makers Medivation, which is developing the drug with Japanese partner Astellas Pharma.

An independent data monitoring committee recommended the trial be halted early so men who were not taking the drug could be offered it.

Professor Johann de Bono, Professor in Experimental Cancer Medicine, The Institute of Cancer Research, The Royal Marsden Hospital and the co-principal investigator of the study, said ‘MDV3100 has a novel mechanism of action and it is encouraging to see these positive survival data from the interim analysis. ‘There is a real need for new treatments in advanced prostate cancer that target the cancer in different ways.’

The new drug is designed to target prostate cancer cells that become resistant to standard hormone-deprivation therapies, which are supposed to cut off supplies of testosterone which allow tumours to grow.

The drug is supposed to hit three different molecular targets which the company hopes will give it an advantage over other new agents in the pipeline.

Around 10,500 British men have advanced prostate cancer that has become resistant to standard hormone treatments, meaning they have few options left.

David Hung, chief executive officer of Medivation, said ‘We’re very excited because of the survival benefit. 18 months ago, once a man with prostate cancer failed chemo, he went to a hospice. 'Now these men will have another treatment option.’

Full analysis of data from the trial, including side effects, will be released at a medical meeting in February in the US.

The drug’s manufacturers plan to meet the US Food and Drug Administration early next year, with the possibility the drug might be licensed as early as 2013.

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We can breathe again: Cystic fibrosis sisters enjoy dramatic health boost thanks to pioneering drug

Only applicable to 5% of sufferers, though

Two sisters who have struggled with cystic fibrosis their whole young lives have had their lives transformed by a pioneering drug.

Laura Cheevers, 13 and her sister Cate, 10, have seen a dramatic improvement in their conditions after they started taking ivacaftor. They can both breathe far more easily, have gained weight and no longer need regular antibiotics.

Up till now the sisters from North Andover in Massachusetts had struggled with the genetic condition, which blocked up their lungs with sticky mucus and left them vulnerable to infections.

Both were in and out of hospital as simple colds could turn into life-threatening lung infections. They also had to consume thousands of calories to keep them from losing weight as they couldn't absorb nutrients effectively.

Then in April 2010 the girls were both accepted onto trials for a new twice-daily pill, which had been developed by the cystic fibrosis Foundation and Vertex Pharmaceuticals. Unlike most previous drugs, ivacaftor was developed to target the cause of cystic fibrosis rather than relieve the symptoms.

After a few months it became clear that while Cate was on the drug, older sister Laura was probably receiving a dummy pill.

'She (Cate) began growing like a weed, and her first lung tests showed almost a 30 per cent bump, which blew us all away,' her mother Kim Cheevers said. Cate added to ABC News: 'She (Laura) would be coughing a lot more. I would feel bad because I wouldn't be coughing.'

Then in April this year, scientists from the University of Washington put all the trial participants on the drug and Laura took a turn for the better. The teenager, who usually found it difficult to gain a pound in a year, has gained eight pounds in seven months.

Her mother Kim, who is an intensive care nurse, said: 'She is not coughing at night anymore.' She added that her daughters were no longer plagued with thick mucus and they found it far easier to clear their lungs.

Cystic fibrosis is caused by a mutation in the gene for the protein that controls the balance of salt and water in the body's mucus membranes. The drug ivacaftor, also known as VX-770, restores the balance in five per cent of CF patients with a particular mutation.

Dr Ronald Crystal, at Weill Cornell Medical College, told ABC News: 'I would predict for those individuals who have this mutation, that it will prolong their lives.'

Scientists believe its success paves the way for tackling other mutations.

Scientists have published the study findings in the latest New England Journal of Medicine. It found the drug improved lung function, helped with normal growth and weight gain and had fewer side-effects than the placebo - suggesting it is safe.

Last month Vertex asked the Food and Drugs Administration and European Medicines Agency for a priority review of the drug.

As for Cate and Laura, they are both continuing on the drug in a trial extension and will continue on it for eight years. 'We're on it until it gets FDA approval,' Ms Cheevers said.

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