Charlotte researchers and patients participated in a clinical trial that has demonstrated the effectiveness of targeted gene therapy for treating advanced lung cancer.
The study, published this week in the New England Journal of Medicine, was co-authored by Dr. Daniel Haggstrom, a medical oncologist at Carolinas HealthCare System’s Levine Cancer Institute.
The experimental drug, called AZD9291, targets a sub-set of lung tumors that have mutated, making patients resistant to therapies they had been taking with good results. When patients took the pill once a day, about 80 percent experienced tumor shrinkage or stability with manageable side effects, Haggstrom reported.
“We are seeing amazing results with this treatment,” Haggstrom said in a statement. “Patients are living longer, with an improved quality of life compared to traditional chemotherapy… It is an exciting time in cancer care… Through research and clinical trials, we are tailoring our therapies to improve outcomes and limit adverse side effects.”
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Targeted therapy, also called personalized medicine, has been heralded as one of the great hopes for cancer patients. President Barack Obama called for an initiative on “precision medicine” during his State of the Union address in January.
“This is a smart war now,” said Dr. Edward Kim, chair of solid tumor oncology at Levine Cancer Institute. “This is a smart missile versus just dropping a bomb out of a plane. We’re being very precise.”
The journal article described the results of a Phase I trial of AZD9291, which has been designated by the Food and Drug Administration as a “breakthrough” drug, meaning it could get expedited approval in coming months.
Phase I trials are not intended to cure anyone, but to evaluate safety, determine the correct dose and identify side effects. The studies usually include a small number of patients whose cancer is so advanced they don’t really benefit. But the results provide the basis for larger studies that compare the new therapy to standard treatment.
The AZD9291 trial enrolled 253 lung cancer patients in 33 medical centers around the world, including four sites in the United States. The Charlotte site enrolled 12 patients.
Lung cancer is the leading cause of cancer death in the United States, and until recently treatment has been limited to chemotherapy or radiation if surgery was not an option. In this country, 10 to 15 percent of lung cancer patients have tumors with a particular genetic mutation. Although there are several approved targeted therapies for those patients, they become ineffective after awhile because the tumors mutate again.
“This drug (AZD9291) can overcome that resistance in half the patients,” Kim said. “We’re targeting mutations coming from mutated tumors. That’s why this is so cool.”
And it’s all accomplished with a daily pill, which is easier to take than infusions of chemotherapy. “There are some people that could literally be on pills for advanced lung cancer for years,” Kim said.
Edwina Edgeworth, 57, of Monroe, is one of the patients whose health improved on the experimental drug.
Three years ago, the former school bus driver was diagnosed with advanced lung cancer even though she has never smoked. Initially she was treated with one of the approved targeted drugs, but after a year and a half, it stopped working. A biopsy showed her tumor had mutated, making her eligible for the AZD9291 trial. She began taking the drug in February 2014, and showed improvement in just a few weeks. Today, her tumor has shrunk by nearly 60 percent.
“Life has been great” since taking the new drug, Edgeworth said. “No more side effects.… I feel close to living a normal life. My next step is to continue to educate people about lung cancer awareness and the benefits of trying study drugs.… I only take one pill a day, and I am a living testimony.”