Drug trials used genetic data to target tumors.

Scientists have hailed a ‘penicillin moment’ in cancer treatment following trials of a drug that uses genetic data to target the formation of specific tumors.  The study has raised hopes drug manufacturers will be able to tailor drugs to individual cancers that will halt them in their tracks and even reverse the growth of existing tumors.


The breakthrough is one of the most significant to use advances in our knowledge of DNA to tackle the root causes of disease.

For years scientists have been assembling vast amounts of genetic information provided through the human genome sequencing project. However, a number of prominent scientists had expressed disappointment that the data had not led to any major advances in treatments.

As part of the latest research, scientists in California developed a drug to block the effects of a specific gene mutation, B-RAF, linked to malignant melanoma – one of the deadliest cancers.

In one small clinical trial, tumors shrank by at least 30 per cent in 24 out of 32 patients with B-RAF mutations, and disappeared entirely in two other patients.

The drug cannot yet be declared a success: it comes with side-effects, can only treat the specific B-RAF mutation and there are no indications of its long-term usefulness.

However, a study of the chemical process behind the drug, detailed in the journal Nature, demonstrates the potential for speedy development of similar treatments targeting the particular genetic mutations that lie behind different types of tumor.

Professor Mark Stratton, Director of the Wellcome Trust Sanger Institute in Cambridge, which first linked B-RAF to malignant melanoma, said: “We’ve entered an end game in which we are going to complete our understanding of what causes cancer.”

Yardena Samuels, a cancer geneticist at the National Human Genome Research Institute in Bethesda, Maryland, told Nature News: “It’s a very important development, not just for melanoma, but for the entire cancer field.”

The company behind PLX4032, Plexxikon, is now working on a test that can diagnose which malignant melanoma patients have the B-RAF mutation and would therefore benefit from the drug.

Sir Mark Walport, director of the Wellcome Trust, agreed with suggestions that the breakthrough was a ‘penicillin moment’ for cancer researchers.

However, he added: “We have got to balance the hype and the hope. Cancer is complicated.”

Via Telegraph