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Trials of new drug offer hope to cancer patients
Amy Harmon, NYT News Service, Feb 24, 2010, 12.52am
For the melanoma patients who signed on to try a drug known as PLX4032, the clinical trial was a last resort. Their bodies were riddled with tumours, leaving them almost certainly just months to live.
But a few weeks after taking their first dose, nearly all of them began to recover.
Lee Reyes (30) of California, who had begun using a feeding tube because of a growth pressing against his throat, bit into a cinnamon roll. Randy Williams, 46, who drove 600 miles from his home in Arkansas to the M D Anderson Cancer Center in Houston for the experimental drug, rolled out of bed. "Something's working," he thought, "because nothing's hurting.
It was a sweet moment, in autumn 2008, for Dr Keith Flaherty, the University of Pennsylvania oncologist leading the drug's first clinical trial. A new kind of cancer therapy, it was tailored to a particular genetic mutation that was driving the disease, and after six years of disappointments, his faith in the promise of such a targeted approach finally seemed borne out. His collaborators at five other major cancer centres, melanoma clinicians who had tested dozens of potential therapies for their patients with no success, were equally elated.
In a kind of pinch-me exercise, the six doctors sent one another before and after CT scans of their patients. The trial of PLX4032 offers a glimpse at how doctors, patients and drug developers navigate a medical frontier as more drugs tailored to the genetic profile of a cancer are being widely tested on humans for the first time.
Throughout the fall, the only two patients on the trial whose tumours continued to grow were the ones who did not have the particular gene mutation for which the drug had been designed. They were removed from the trial. By late December, tumours in the 11 patients who did have the mutation had shrunk. Those involved in the trial held their collective breath waiting to see how long the remissions would last.
One of them was Mark Bunting, 52, an airline pilot from Utah. His initial scan in early October showed the cancer in his bones, an incursion considered virtually impossible to reverse. After two months on the drug, it had all but disappeared. From New York, Paul B Chapman of Memorial Sloan-Kettering Cancer Center, perhaps the most determined sceptic of the group, acknowledged, "This looks impressive."
It was a far cry from where they had been a year earlier, when a previous incarnation of the drug had no effect. Urged on by Flaherty and Chapman, the companies that owned it had spent months devising a new formulation that could be absorbed at higher doses.
But the new drug, still in the earliest phase of testing, had to pass several more hurdles before federal regulators would determine whether it was safe and effective enough for widespread use.
In December, as the doctors added more patients to the Phase 1 trial, looking for the highest dose they could give without intolerable side effects, they scrambled to prepare slides with graphs and statistics to convince the Food and Drug Administration that the drug should be tested in a larger Phase 2 trial.
The agency required a summary of any and all side-effects there had been only a few and any deaths of patients on the study; thankfully, there had been none since the drug was reformulated. In a matter of days they needed to submit their findings for a prestigious meeting of clinical oncologists in June.
Amy Harmon, NYT News Service, Feb 24, 2010, 12.52am
For the melanoma patients who signed on to try a drug known as PLX4032, the clinical trial was a last resort. Their bodies were riddled with tumours, leaving them almost certainly just months to live.
But a few weeks after taking their first dose, nearly all of them began to recover.
Lee Reyes (30) of California, who had begun using a feeding tube because of a growth pressing against his throat, bit into a cinnamon roll. Randy Williams, 46, who drove 600 miles from his home in Arkansas to the M D Anderson Cancer Center in Houston for the experimental drug, rolled out of bed. "Something's working," he thought, "because nothing's hurting.
It was a sweet moment, in autumn 2008, for Dr Keith Flaherty, the University of Pennsylvania oncologist leading the drug's first clinical trial. A new kind of cancer therapy, it was tailored to a particular genetic mutation that was driving the disease, and after six years of disappointments, his faith in the promise of such a targeted approach finally seemed borne out. His collaborators at five other major cancer centres, melanoma clinicians who had tested dozens of potential therapies for their patients with no success, were equally elated.
In a kind of pinch-me exercise, the six doctors sent one another before and after CT scans of their patients. The trial of PLX4032 offers a glimpse at how doctors, patients and drug developers navigate a medical frontier as more drugs tailored to the genetic profile of a cancer are being widely tested on humans for the first time.
Throughout the fall, the only two patients on the trial whose tumours continued to grow were the ones who did not have the particular gene mutation for which the drug had been designed. They were removed from the trial. By late December, tumours in the 11 patients who did have the mutation had shrunk. Those involved in the trial held their collective breath waiting to see how long the remissions would last.
One of them was Mark Bunting, 52, an airline pilot from Utah. His initial scan in early October showed the cancer in his bones, an incursion considered virtually impossible to reverse. After two months on the drug, it had all but disappeared. From New York, Paul B Chapman of Memorial Sloan-Kettering Cancer Center, perhaps the most determined sceptic of the group, acknowledged, "This looks impressive."
It was a far cry from where they had been a year earlier, when a previous incarnation of the drug had no effect. Urged on by Flaherty and Chapman, the companies that owned it had spent months devising a new formulation that could be absorbed at higher doses.
But the new drug, still in the earliest phase of testing, had to pass several more hurdles before federal regulators would determine whether it was safe and effective enough for widespread use.
In December, as the doctors added more patients to the Phase 1 trial, looking for the highest dose they could give without intolerable side effects, they scrambled to prepare slides with graphs and statistics to convince the Food and Drug Administration that the drug should be tested in a larger Phase 2 trial.
The agency required a summary of any and all side-effects there had been only a few and any deaths of patients on the study; thankfully, there had been none since the drug was reformulated. In a matter of days they needed to submit their findings for a prestigious meeting of clinical oncologists in June.
