The 86 cancer patients all had tumors of the pancreas, prostate, uterus, or bone. One woman had cancer so rare there were no tested treatments. She was told to get her affairs in order.
All of these patients had a few things in common. All had advanced disease that had resisted every standard treatment, and all had genetic mutations that disrupted the ability of cells to fix damaged DNA. And all were enrolled in a trial of a drug that helps the immune system attack tumors.
The drug, Keytruda had such great results that the Food and Drug Administration has already approved the drug. It is the first time a drug has been approved for use against tumors that share a certain genetic profile, whatever their location in the body. Tens of thousands of cancer patients each year could benefit.
“This is absolutely brilliant,” said Dr. Jose Baselga, a physician in chief at Memorial Sloan-Kettering Cancer Center in New York, which has just hired the study’s lead investigator, Dr. Luis A. Diaz Jr.
After taking Keytruda, 66 patients had their tumors shrink substantially and stabilize, instead of continuing to grow. Among them were 18 patients whose tumors vanished and have not returned.
Since there was no control group, the results had to be significant. The study started in 2013 and is funded by philanthropies; the drug maker’s only role was to supply the drug. The study is continuing.
The drug, made by Merck, is already on the market for select patients with a few types of advanced lung, melanoma and bladder tumors. It is expensive, costing $156,000 a year.
A test for the mutations targeted by the drug is already available, too, for $300 to $600.
Just 4 percent of cancer patients have the type of genetic aberration susceptible to Keytruda. But that adds up to a lot of patients: as many as 60,000 each year in the United States alone, the study’s investigators estimated.
Clinicians have long been accustomed to classifying cancers by their location in the body – patients are diagnosed with lung cancer, for example, or brain cancer.
Yet researchers have been saying for years that what matters was the genetic mutation causing the tumors. At first, they were certain they would be able to cure cancers with drugs that zeroed in on the mutations, wherever the tumors were lodged.
But cancers were more complicated than that, said Dr. Drew M. Pardoll, director of the Johns Hopkins Bloomberg-Kimmel Institute and an author of the new paper.
The new study was based on the following understanding of the immune system. The immune system can recognize cancer cells as foreign and destroy them. But tumors deflect the attack by shielding proteins on their surface, making them invisible to the immune system.
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