It's a first-of-a-kind experiment that brings together five drug companies, the government, private foundations and advocacy groups. The idea came from the federal Food and Drug Administration, which has agreed to consider approving new medicines based on results from the study.
Its goal is to speed new treatments to market and give seriously ill patients more chances to find something that will help. Instead of being tested for individual genes and trying to qualify for separate clinical trials testing single drugs, patients can enrol in this umbrella study, get full gene testing and have access to many options at once.
The study, called Lung-MAP, is for advanced cases of a common, hard-to-treat form of lung cancer — squamous cell. Plans for similar studies for breast and colon cancer are in the works.
"For patients, it gives them their best chance for treatment of a deadly disease," because everyone gets some type of therapy, said Ellen Sigal, chairwoman and founder of Friends of Cancer Research, a Washington-based research and advocacy group that helped plan and launch the study. "There's something for everyone, and we'll get answers faster" on whether experimental drugs work, she said.
Cancer medicines increasingly target specific gene mutations that are carried by smaller groups of patients. But researchers sometimes have to screen hundreds of patients to find a few with the right mutation, making drug development inefficient, expensive and slow.
One of the leaders of the Lung-MAP study — Dr. Roy Herbst, chief of medical oncology at the Yale Cancer Center — said he once screened 100 patients to find five that might be eligible for a study, and ultimately was able to enrol two.
"It's just going to be impossible, in rare subgroups, for companies to find enough" people to try out a new medicine, said Dr. Richard Pazdur, cancer drugs chief at the FDA. He and others at the FDA suggested the Lung-MAP trial design to speed new treatments to market and "minimize the number of patients exposed to ineffective therapies," he said.
Everyone in the study will be screened for mutations in more than 200 cancer-related genes, rather than a single mutation as in conventional studies.
Then they will be assigned to one of five groups based on what these tumour biomarkers show. Each group will test a particular experimental medicine. Drugs can be added or subtracted from the study as it goes on, based on how each performs.
The initial round of testing involves Amgen, Genentech, Pfizer, AstraZeneca PLC, and AstraZeneca's global biologics partner, MedImmune. Up to 1,000 patients a year can be enrolled in the study.
It will cost about $150 million. The National Cancer Institute is paying $25 million, and the rest will come from foundations, charities and others in the public-private partnership.
About 500 hospitals that are part of a large cancer treatment consortium around the country will take part, and some private groups want to join as well, Herbst said.
"Nothing like this has ever been done before," where such comprehensive testing will be done to match patients to experimental drugs, he said.
Breyan Harris, a 33-year-old nurse from Sacramento, hopes to enrol. She's a lifelong non-smoker who was diagnosed with lung cancer on June 3.
"Since then I've pretty much been on the phone, seeing doctors, trying to figure out how do I get rid of this," she said. Harris expects to have one lung with a large tumour removed, "but if it comes back in my other lung I'm in real trouble," so finding a drug to attack any remaining, hidden cancer is crucial, she said.
Study sites and info: http://tinyurl.com/ovv4cnl
Marilynn Marchione can be followed at http://twitter.com/MMarchioneAP