Robert Bazell writes: Tonight we report on exciting, early results with an experimental drug for advanced melanoma. The drug is still only known by its experimental name PLX4032. It targets a genetic mutation called BRAF which is present in about 60 per cent of melanomas. That means that only people who have the BRAF mutation in their cancer could even possibly benefit. But in the latest results 80 per cent of those with the mutation and very advanced melanoma had their tumors shrink. Some are alive and well as long as two years after the treatment. You can read the research paper in the New England Journal of Medicine and editorial about it in the Journal.
These results come from the early stage trials called Phase I and II. Researchers are enrolling patients in a pivotal Phase III trial which will lead to FDA approval if the results continue to go well. The drug is made by a small biotechnology company called Plexxicon in Berkeley, California which is partnering with Roche to test and sell the drug. Click here to find out about enrolling in clinical trials
The hope is that ultimately doctors will be able to give this drug, not just to people with advanced melanoma, but in what is called adjuvant therapy immediately after surgery. That should keep the cancer from spreading in the first place and bring about many long-term cures
For years melanoma was easily treatable if found early and surgically removed, but if it spread the outlook was bleak. These results follow three months after a completely separate drug Ipilimimab was shown to cure 20 to 30 per cent of people with advanced melanoma. Ipilimimab works by stimulating the immune system to attack the melanoma. The FDA just received the results for that drug and granted it accelerated approval status which means it could be on the market within a year. There seems to be no correlation between which patients benefit from which drug. Some could benefit form both.
But sadly –even if both drugs end up being widely available, manyh patients will not be helped by either. This is how progress against cancer is beginning to take shape. Scientists and doctors have learned that even one type of cancer – such as melanoma – is not one disease, but several. And the still very distant goal is to find treatments for all forms of all cancers.