New Experimental Drug Shows 'Dramatic' Results in Lung Cancer Patients

Oct. 27, 2010— -- Evie Cogan sat in her car, waiting for the light turn to green. She grabbed the X-ray and CT documents that she had just picked up from the doctor's office moments before. Scanning the page, she saw the diagnosis: Right lung carcinoma.

Months earlier, Cogan, an assistant professor at LaSalle University in Philadelphia, had been trying to fight off a nagging cough that wouldn't let up for months. She dismissed it as bronchitis as she finished the 2005 spring semester in Basel, Switzerland, where she was teaching classes.

"You need to check out that cough," her father told her over the phone.

She finally did. After returning to the United States, she went to the doctor to get her first chest X-ray. Much to her relief, the X-ray came back negative.

Her doctors treated the cough as allergies, and put her on Claritin. But the cough worsened in the following weeks.

After another doctor's visit some time later, she received a voice mail from her physician. His voice sounded urgent and she was told to call him back as soon as possible.

The conversation is now a blur for Cogan, but she remembers the words, "fluid in the lungs."

Cogan had never smoked a cigarette in her life; she eats a healthy diet, and had a grandmother who lived past 100.

"It was surreal," said Cogan. "Everyone has secret fears that never happen, but this actually happened. It was so shocking."

And so Cogan began her journey through chemotherapy, radiation, and more chemotherapy. After all, Cogan said, she was in the battle of her life.

In November 2009, four years after her initial diagnosis, Cogan's friend called her about a medical piece she had seen on the ABC's World News, where Charlie Gibson discussed a new treatment for a specific kind of lung cancer.

Experimental Lung Cancer Drug: A New Option?

Cogan searched the Internet for the video, and watched the story of Bill Schuette, a lung cancer patient who, after exhausting all his treatment options, had qualified for a phase I clinical trial at Massachusetts General Hospital. He joined one other person in the trial after he tested positive for a genetic mutation in his lung tumor, known as EML4/ALK. Within weeks after taking the medicine, Schuette began to feel better and his tumor began to shrink.

I want this drug, Cogan said to herself.

She was hopeful, even though her chance of qualifying was slim. Each year, around 170,000 Americans are diagnosed with non-small-cell lung cancer. About two to seven percent of those patients have the genetic mutation in the tumor.

The experimental drug has now been described as "dramatic" and "exciting." With 82 patients now enrolled on the study, a new study published today in the New England Journal of Medicine shows that the targeted treatment, known as crizotinib, can halt or even reverse the growth of the non-small-cell lung tumors with the genetic mutation.

And Cogan qualified. She entered the clinical trial on Jan. 25.

"It was my miracle," said Cogan. "I have wonderful quality of life now."

"As physicians, it's worth prospectively identifying the genetic abnormalities in tumors to help guide patients to trial that will most like benefit them, even if it's a small number of people," said Dr. Eunice Kwak, lead author of the study and assistant in Medicine at Massachusetts General Hospital.

The study began as a phase 1 trial to determine the safety profile and tolerated dose of the drug. Because the researchers identified patients who responded positively to the drug, they wanted to find a larger group of patients who tested positive for the gene mutation.

The treatment reduced the tumor size in more than half of the 82 participants, and stopped tumor growth in one third. Historically, chemotherapy only halts tumor growth in 10 percent of non-small-cell cancer patients.

"About 60 percent of people with ALK mutations will have a good response with crizotinib, so for these people, this drug offers a 35 percent better chance for good disease shrinkage," said Dr. Gregory Kalemkerian, professor of Medicine and co-director of Thoracic Oncology at University of Michigan Health System. "That is a big benefit in the oncology world."

Present and Future of Genetic Screening

Genetic testing is the newest and most sophisticated of techniques to treat a variety of illnesses. The treatments require a direct examination of the DNA itself.

Dr. William Pao, an associate professor of medicine at Vanderbilt University, knows a lot about the topic because of his research on identification of genes in lung tumors.

"We are moving into the era of genetically-informed cancer medicine, where we use the genetic fingerprint of individual patient tumors to customize treatment plans," said Pao. "Many of us in the field are excited about this because we believe that the outcomes will be better and potentially less toxic than what we have done in the past."

Although the proportion of patients who carry the ALK mutation is small, the goal for such treatments is to personalize the delivery of care to patients with lung cancer.

"Though only three to five percent of patients harbor the particular mutation, those patients can receive dramatic benefit," said Dr. Martin Edelman, professor at the University of Maryland School of Medicine. "It must be recognized that a small percentage of a very common disease represents a fairly large number of people."

More Trials to Come

While the results of the early trials have been promising, researchers have already planned additional trials to confirm the long-term effectiveness of the treatment. The published study only reports the first six months of treatment results. In similar types of therapy, resistance can develop in patients within one or two years.

But for Cogan, the experience is worth the risk. She said she remains optimistic.

"I feel so fortunate to be in this clinical trial and have the wonderful care from the doctors at Mass General," she said.