His patient, a spunky Italian-American woman in her 60s, was waiting in an exam room down the hall for the answer: Was the experimental drug stopping her deadly skin cancer?
But as Dr. Keith Flaherty read out the measurements of her tumors from the latest CT scan, he could not keep the distress from his voice.
“She’s worse,” he said to the clinical trial nurse at the University of Pennsylvania’s melanoma clinic.
Like the 17 other patients on the drug trial — the corporate lawyer, the receptionist with young children, the Philadelphia philanthropist — the woman known in the trial as Patient 18 was going to die, most likely within months.
In the exam room, her gratitude for his failed efforts to save her tore at his heart.
He had been so optimistic. A radical departure from standard chemotherapy, the drug was designed to reverse the effect of a genetic mutation particular to the patient’s tumors. The approach represented what some oncologists see as the best bet for attacking all types of cancer.
And as he returned to his office that autumn afternoon two years ago, Dr. Flaherty was already calculating the next step: he wanted to test the drug at a more potent dose before giving it to more patients in a larger trial. It would require retooling the drug in a costly and complicated task that might not work, and he would have to make his case to two companies that had already poured hundreds of millions of dollars into the drug and were eager to move it forward.
“This,” he insisted to colleagues, “is the best drug we’re going to get.”
Dozens of such “targeted” drugs are emerging from the laboratory, rooted in decades of research and backed by unprecedented investment by pharmaceutical companies, which stand to profit from drugs that prolong life even by weeks.
But putting them to their truest test falls to a small band of doctors committed to running experimental drug trials for patients they have no other way to heal.
At a time when cancer still kills one in four Americans, it is a job that requires as much hubris as heart. To chronicle the trial of the drug known as PLX4032 is to ride a roller coaster of breakthroughs and setbacks at what many oncologists see as a watershed moment in understanding the genetic changes that cause cancer.
Over three tumultuous years, Dr. Flaherty saw patients who drove hundreds of miles for their monthly dose, and one who arrived barely able to walk. Some took 32 pills a day. When it became clear they were not absorbing the drug, he asked them to take the pills with high-fat foods like hamburgers and eggs, which might help dissolve them.
At academic conferences, he clashed with other oncologists who warned that targeted therapy had almost never had long-lasting results. At Penn, he badgered laboratory researchers whose animal tests might provide early clues for how a drug would behave in his patients.
And always, he ended up on his BlackBerry, e-mailing, calling, cajoling the drug makers to commit even more resources to the new category of drugs he so deeply believed in.
A five-and-a-half-foot streak of outsize energy, Dr. Flaherty, 39, seemed buoyed by an innate optimism and a faith in the scientific logic underlying the approach.
But at his clinic, where he gave vials of pills to patients whose tumors were often erupting, black and bumpy across their arms and legs, he told them only what he believed to be true.
“This,” he said, “is our best shot.”
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