A daily pill could halve the risk of death for lung cancer patients with a certain type of genetic mutation who have undergone surgery, according to long-awaited clinical trial results.
The drug is called osimertinib, which AstraZeneca Pharmaceuticals manufactures under the brand name Tagrisso. It has been used to treat late-stage lung cancer since the U.S. Food and Drug Administration granted it accelerated approval in 2015. The study’s findings were published June 4 in the New England Journal of Medicine and presented the same day at the annual meeting of the American Society of Clinical Oncology in Chicago.
The new study provides evidence that the same drug may improve outcomes for people with early stages of lung cancersaid Roy Herbst, deputy director of the Yale Cancer Center and co-lead author of the paper. “It really takes personalized therapy of advanced metastatic disease and moves it all the way to the earliest stages of lung cancer treatment,” says Herbst.
Lung cancer is one of the most common cancers in the US. The American Cancer Society expects more than 230,000 new cases to be reported this year. It is also the deadliest, responsible for nearly a quarter of cancer deaths in the US in 2020, according to the Centers for Disease Control and Prevention. Osimertinib is what scientists call a tyrosine kinase inhibitor, a compound that affects a class of enzymes involved in cell signaling, growth and division. The new findings will only be relevant to a small proportion of people with a specific type of genetic mutation. But for them, says Herbst, early treatment with the drug can make a big difference. “We expected this to work, but not as well,” he says.
The trial included 682 patients with the mutation, called T790M. Just under half of them took osimertinib once a day; the others took a placebo pill. All participants had had a tumor surgically removed and their cancer was considered stage 1, 2 or 3. (This scale goes up to stage 4, which is the most severe.) Some participants also had chemotherapy treatments. Each patient took the medicine for three years unless they had to stop, and they were followed for an average of five years (although some were followed for up to 6.8 years in total).
After five years, 88 percent of patients on osimertinib were alive, compared to 78 percent of the control population, meaning the drug roughly halved the risk of death over the five-year period. Osimertinib was also shown to reduce disease recurrence. There were about twice as many recurrences among the people taking placebos.
Of the patients in the study with more advanced stage 3 cancers, 85 percent of those taking osimertinib survived after five years, compared with 67 percent of those taking the placebo — an even greater reduction in deaths.
Those are compelling statistics, says Nagashree Seetharamu, a thoracic and head and neck oncologist at Northwell Health, who was not involved in the new study. Herbst’s presentation at the conference clarified many of the questions other researchers had, she says, noting that an earlier report from the group had left some observers unconvinced that the drug materially changed patient outcomes, rather than just a little more. buy time. She says it makes sense to treat eligible patients with this approach, though she would still like to see longer-term results.
Seetharamu also says the new study leaves important questions open about how osimertinib and chemotherapy work together, because some people in both the control and test groups had received chemotherapy before entering the study.
Because osimertinib only works for people with a specific genetic mutation, both Seetharamu and Herbst emphasize that the results underline how genetic engineering can improve outcomes by matching people with the most promising treatment options. “Genomic testing is extremely important,” says Seetharamu. “We knew it’s very important in patients with late-stage disease, but now, with studies like this one, we know it’s also important in earlier-stage disease.”
Herbst says he hopes the combination of genetic testing and the use of first-class drugs will work for other cancers as well. However, these drugs may incur costs. Multiple studies have questioned whether the improvements osimertinib can make are worth the staggering price tag of about $17,000 per month in the US
Nevertheless, Herbst is satisfied with the findings of the study. “When I started working in lung cancer, there was nothing to offer. There was no targeted therapy; there was no immunotherapy,” he says. “We brought new science-based medicines to the clinic; we learned how to use them; we learned how to personalize them.”
