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Cancer's penicillin moment?

Tom Feilden | 09:56 UK time, Wednesday, 15 September 2010

It is so new it doesn't even have a name yet, but PLX 4032 is already being hailed as "one of the most exciting new cancer drugs in a generation".

That's partly a reflection of the promising results this experimental new drug has been producing in early stage clinical trials - successfully shrinking tumours in patients suffering from malignant melanoma - but also because of the way the drug was developed.

PLX 4032 is one of the first in a new class of drugs specifically designed to target the abnormal molecular activity resulting from an individual genetic mutation - in this case the B-RAF gene, which is stuck in the "on" position in malignant melanoma.

Its chemical structure is described in a paper in the journal Nature, which outlines the drug's discovery, development and functioning.

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We've known for some time that all cancers are the result of mutations in individual genes, but it's only been in the last few years that a phenomenal increase in the computational power of gene sequencing has allowed scientists to begin to sift the DNA code, comparing healthy and diseased cells and teasing out the subtle variations that give rise to cancer. That's given drug companies new targets for drug therapies like the mutated B-RAF gene in malignant melanoma, a previously incurable form of skin cancer.

"We really are at a remarkable moment," according to the Director of the Sanger Institute Professor Mike Stratton. "Modern sequencing techniques give us the potential to identify all of the genetic mutations that give rise to cancer. We've entered the end game in which we're going to complete our understanding of what causes cancer."

Of course getting to that end game is not the same thing as crossing the finishing line. Simply identifying all the genetic mutations that give rise to all the different types of cancer remains a colossal sequencing task, and even where that's achieved there's no guarantee that drug companies will be able to develop suitable treatments.

But the power of DNA sequencing does at least put us on a level playing field with cancer, and the hope, as one researcher put it, is that genomics may be about to deliver cancer's penicillin moment.


  • Comment number 1.

    Thank you *so* much for providing a link to the original research. All science writers should do that, but so few do!

  • Comment number 2.


    In your blog and your piece on today this morning you didn't mention the company who have developed PLX4032 or even hint that it had been developed in the commercial sector.

    Why do journalist persist with the convention that new drugs are discovered by "scientists", while stories that mention pharmaceutical companies invariably focus on the profits they make or the side-effects of an on-market drug.

  • Comment number 3.

    ...From which we divine that in clinical terms, this is an incremental step forward. Tumours regrew after a few months, having acquired resistance to the drug. In just two of 32 subjects, the tumours disappeared. So, we may see a small increase in survival, and a few months longer for more patients.

    I guess the exciting thing is that this does represent the breaking of a deadlock for this particular cancer, and that breakthrough came from the information provided by gene sequencing.

  • Comment number 4.

    This was a very interesting piece (on Thursday at about 08:20) about the possibility of a "Penicillin Moment" for cancer ...

    ... and so was the article today (Friday, at about 08:20) about the conference at the University of Arizona bringing together physicist, mathematicians, cosmologist and biologists to talk to each other to try to find new ways to address the *lack* of a "Penicillin Moment" for cancer.

    Hmmm, perhaps there needs to be some thinking about new ways for the BBC science editorial staff to talk to each other.


  • Comment number 5.

    My husband took part in this trial. Unfortunately he was one of the unlucky ones. He didn't make it. At first everything seemed to be going fine. All the tumours in his body shrunk by nearly 40%. Then it was dicovered the cancer was in his brain where the experimental drug was ineffective because it couldn't cross the blood brain barrier. Fifty per cent of advanced melanoma cases develop metastases in the brain and of course any malignant cancer that has spread to several sites within the body may also spread to the brain. I asked the doctors what research was being done to find treatments that could cross the blood brain barrier and the answer came back 'very little'. We will not have beaten cancer until we have an answer to this problem. He died earlier this year, six months after diagnosis. A year before that he had every appearance of being perfectly healthy and very fit for his age.


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