IN WHAT is arguably the most significant use of genetic knowledge to tackle disease, scientists in California have created a drug that prevents the effects of a gene mutation linked to malignant melanoma.
The London Daily Telegraph reports that researchers have built on advances made by the human genome project to develop a drug that specifically targets the deadly cancer.
In a small clinical trial, melanoma tumors shrank by nearly a third in 24 of 32 patients with a mutation known as B-RAF – and in two other people, the tumors disappeared completely – according to the research, published in the journal Nature.
Plexxikon, the company behind the the drug – preliminarily called PLX4032 – has also begun working on a test to determine whether individual melanoma sufferers have the mutation and thus would respond to the treatment.
While the drug’s long-term effects are unknown, and it does cause unwanted side effects, its initial results gave hope to scientists and cancer patients alike that specific chemicals can be used to target specific diseases.
Sir Mark Walport, director of UK cancer charity the Wellcome Trust, said 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.”
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