The Last Word On Nothing

Van VanderMeer is about to celebrate an anniversary that he’d probably rather forget.

In December 2009, VanderMeer thought he had caught his annual winter cough; for a few years in a row, he’d developed a chest cold around this time of year. But this one lingered. VanderMeer was competing in a mixed doubles tournament in Virginia Beach, Virginia, where he lives, when he realized that he was too short of breath to continue the match.

His wife convinced him to see his doctor. An x-ray showed that VanderMeer’s left lung was almost entirely filled with fluid, prompting this observation from his radiologist: “This could be something not good.”

A diagnosis of lung cancer – stage 4 adenocarcinoma of the lung – followed in March 2010. VanderMeer learned that most people with his diagnosis lived for one year.

But VanderMeer, 64, felt hopeful when his doctor told him that his tumor had a genetic vulnerability to a treatment undergoing testing in clinical trials. This drug, crizotinib, is designed to destroy tumors bearing a particular glitch that turns on the activity of a cancer-causing gene called anaplastic lymphoma kinase – ALK.

For some cancers, and some patients, targeted treatments such as crizotinib have revolutionized cancer care. Leukemia patients who take imatinib, better known as Gleevec, for instance, can expect to live about as long as people never diagnosed with cancer. That’s truly remarkable, considering that before the drug was approved in 2001, these patients could expect to live for four to six years.

Patients with lung cancer badly need new treatment options like these. Fewer than one of every five patients diagnosed with lung cancer survive for five years, largely because it is not usually discovered until it has already spread and is therefore difficult to treat.

Less than ten percent of lung cancer patients have the genetic rearrangement that is targeted by crizotinib, and VanderMeer was one of them. He knew that the drug aims directly at the root of the cancer, rather than indiscriminately destroying cells, as chemotherapy does. “I refer to it as a stiletto, instead of the blunderbuss of chemotherapy,” he says. “I was convinced it was a silver bullet.”

So VanderMeer visited Johns Hopkins University in Baltimore, Maryland, to try to enroll in a clinical trial of the drug. But he wasn’t eligible for the trial because he hadn’t yet failed chemotherapy. And when he eventually did fail treatment this February, because he started developing kidney problems, those kidney problems barred him from participating in the crizotinib trials.

Then, in August, the same month that the US Food and Drug Administration approved crizotinib, VanderMeer learned that his cancer had metastasized – spread to both his lungs.

Finally, in September, a drug representative for Pfizer, which makes crizotinib, met VanderMeer and vowed to get him access to the drug. He started taking it on 21 September.

Two weeks later – on 4 October – the tumors had disappeared from VanderMeer’s lungs.

VanderMeer allows that is was frustrating not to be able to get access to crizotinib when he knew he might benefit from the drug. There is a major clinical trial in the works, funded by the US National Cancer Institute, that aims to test what happens when targeted treatments are used earlier in the course of patients’ lung cancer. I’ll be publishing more about that trial in Nature today. [Update: my story covers the first large clinical trial to test earlier use of targeted therapies in lung cancer.]

This is a crucial study, because most targeted treatments buy patients a few extra months, at most, of life, and their high cost has led regulators and insurers to make tough decisions about whether they are worth the expense.

As for VanderMeer, he’s just happy to be finally retiring the blunderbuss of surgeries, chemotherapy and blood transfusions in favor of the stiletto approach to treatment. Most patients who take crizotinib survive between 10 months and a year.

Having survived this long, VanderMeer is already beating the odds. Here’s hoping that the stiletto helps him continue to do that.

***

Image: 18th century blunderbuss, courtesy World Imaging/Wikimedia Commons.