A Clue in RNA
Dr. Wartman’s doctors realized then that their last best hope for saving him was to use all the genetic know-how and technology at their disposal.
After their month of frantic work to beat cancer’s relentless clock, the group, led by Richard Wilson and Elaine Mardis, directors of the university’s genome institute, had the data. It was Aug. 31.
The cancer’s DNA had, as expected, many mutations, but there was nothing to be done about them. There were no drugs to attack them.
But the other analysis, of the cancer’s RNA, was different. There was something there, something unexpected.
The RNA sequencing showed that a normal gene, FLT3, was wildly active in the leukemia cells. Its normal role is to make cells grow and proliferate. An overactive FLT3 gene might be making Dr. Wartman’s cancer cells multiply so quickly.
Even better, there was a drug, sunitinib or Sutent, approved for treating advanced kidney cancer, that inhibits FLT3.
But it costs $330 a day, and Dr. Wartman’s insurance company would not pay for it. He appealed twice to his insurer and lost both times.
He also pleaded with the drug’s maker, Pfizer, to give him the drug under its compassionate use program, explaining that his entire salary was only enough to pay for 7 ½ months of Sutent. But Pfizer turned him down too.
As September went by, Dr. Wartman was getting panicky.
“Every day is a roller coaster,” he said at the time, “and everything is up in the air.”
Desperate to try the drug, he scraped up the money to buy a week’s worth and began taking it on Sept. 16. Within days, his blood counts were looking more normal.
But over dinner at a trendy St. Louis restaurant, he picked at his chicken and said he was afraid to hope.
“Obviously it’s exciting,” he said. “But Sutent could have unanticipated effects on my bone marrow.” Maybe his rising red blood cell counts were just a side effect of the drug. Or maybe they were just a coincidence.
“It’s hard to say if I feel any different,” Dr. Wartman said.
And the cost of the drug nagged at him. If it worked, how long could he afford to keep taking it?
The next day, a nurse at the hospital pharmacy called with what seemed miraculous news: a month’s supply of Sutent was waiting for Dr. Wartman. He did not know at the time, but the doctors in his division had pitched in to buy the drug.
Two weeks later, his bone marrow, which had been full of leukemia cells, was clean, a biopsy showed.
Still, he was nervous. The test involved taking out just a small amount of marrow. Cancer cells could be lurking unseen.
The next test was flow cytometry, which used antibodies to label cancer cells. Again, there were no cancer cells.
But even flow cytometry could be misleading, Dr. Wartman told himself.
Finally, a yet more sensitive test, called FISH, was done. It labels cancer cells with fluorescent pieces of DNA to identify leukemia cells. Once again, there were none.
“I can’t believe it,” his awe-struck physician, Dr. John DiPersio, told him.
Dr. Wartman, alone in his apartment, waited for his partner, Damon Berardi, to come home from work. That evening, Mr. Berardi, a 31-year-old store manager, opened the door with no idea of Dr. Wartman’s momentous news. To his surprise, Dr. Wartman was home early, waiting in the kitchen with champagne and two flutes he had given Mr. Berardi for Christmas. He told Mr. Berardi he should sit down.
“My leukemia is in remission,” he said. The men embraced exultantly, and Dr. Wartman popped open the champagne.
“I felt an overwhelming sense of relief and a renewed vision of our future together,” Mr. Berardi said. “There were no tears at that moment. We had both had cried plenty. This was a moment of hope.”
Hunches and Decisions
Dr. Wartman and his doctors had fateful decisions to make, with nothing but hunches to guide them. Should he keep taking Sutent or have another bone-marrow transplant now that he was in remission again?
In the end, Dr. DiPersio decided Dr. Wartman should have the transplant because without it the cancer might mutate and escape the Sutent.
Meanwhile, Pfizer had decided to give him the drug. Dr. Wartman has no idea why. Perhaps the company was swayed by an impassioned plea from his nurse practitioner, Stephanie Bauer.
Dr. Wartman’s cancer is still gone, for now, but he has struggled with a common complication of bone-marrow transplants, in which the white blood cells of the transplanted marrow attack his cells as though they were foreign. He has had rashes and felt ill. But these complications are gradually lessening, and he is back at work in Dr. Ley’s lab.
His colleagues want to look for the same mutation in the cancer cells of other patients with his cancer. And they would like to start a clinical trial testing Sutent to discover whether the drug can help others with leukemia, or whether the solution they found was unique to Lukas Wartman.
Dr. Wartman himself is left with nagging uncertainties. He knows how lucky he is, but what does the future hold? Can he plan a life? Is he cured?
“It’s a hard feeling to describe,” he said. “I am in uncharted waters.”