Within hours after sales of a powerful new leukemia drug were stopped last month because of potentially lethal side effects, loosely organized groups of doctors and patients — determined to keep getting a medicine they depend on — sprang into action across the country.
They began making calls and writing letters to regulators, seeking assurance that people already taking Iclusig, developed by Cambridge’s Ariad Pharmaceuticals Inc., would have uninterrupted access to the treatment while Food and Drug Administration officials pored over new clinical data on its toxic side effects and haggled with Ariad about revised wording on a warning label.
“I was terrified because this drug is my lifeline,” said Karen Linscott, a 48-year-old teacher from Eden Prairie, Minn. Her form of blood cancer — chronic myeloid leukemia — has been in remission for two and a half years since she began taking Iclusig as part of a clinical trial in Ann Arbor, Mich.
Soon after the drug was pulled from the market on Oct. 31, Linscott and other patients appealed to an FDA patient liaison in an hour-long conference call. “They had to hear from the patients and the doctors,” she said.
Meanwhile, Dr. Richard M. Stone, director of the adult leukemia program at Boston’s Dana-Farber Cancer Institute, joined 22 other blood cancer specialists to sign a Nov. 1 letter to FDA oncology chief Richard Pazdur. It called on the agency to speed up a process allowing patients who had been buying the drug commercially to get approval to keep receiving it.
Within three days, the new program was in effect.
The successful lobbying effort underscores the growing role of patient advocates in a drug monitoring process once seen as the exclusive province of regulators. In this and other recent cases, advocates have contended that physicians should be permitted to prescribe drugs with serious side effects to willing patients who have few — or no — other options to treat diseases that are often fatal.
Some patients using Iclusig — which cost about $115,000 a year — say their health improved dramatically because of the treatment, and are not deterred by recent evidence linking it to increased incidence of blood clots, heart attacks, and strokes.
“The risks outlined by the FDA are real,” said Stone, a Harvard Medical School professor who chairs the Alliance’s leukemia committee, a group of doctors and health professionals studying new treatments. “But there are certain patients for whom there are no good alternatives. So we think there’s a risk-benefit ratio that favors continuing giving them the drug.”
Similar arguments have arisen around other medicines that carry significant risks, but also work remarkably well for some people. In March, breast cancer patients and supporters picketed a Cambridge hotel where FDA commissioner Margaret Hamburg spoke, calling for faster approval of new drugs.
Outside the Biotechnology Industry Organization’s annual convention in Boston last year, a group demonstrated against long delays in approving the breast cancer therapy Kadcyla.
And last week, more than a half dozen patients and advocates testified at an FDA advisory panel meeting, contending that severely ill multiple sclerosis patients and their doctors should be able to determine whether the benefits of the experimental drug Lemtrada — made by Cambridge biotech Genzyme — outweigh its safety risks.
“If one of your family members had MS, wouldn’t you want them to have a choice?” Connecticut patient Melissa Burdick asked medical specialists and FDA staffers at the Silver Spring, Md., meeting. “We, as patients, deserve the right to have a choice of therapy.”
Iclusig patients echo that sentiment. “I believe in this drug, it saved my life,” said Beth Galliart, an executive assistant at a Silicon Valley financial firm who was diagnosed with leukemia in 2008 and began taking Iclusig in a clinical trial the following year. “It means I got to meet my nephew. But I could wake up tomorrow morning and it could be taken away.”
FDA officials are often torn between their dual missions of assuring that lifesaving treatments get to patients and guarding against potentially deadly health risks. More than a dozen patients have died after taking Iclusig in clinical studies involving more than 500 patients worldwide, according to the FDA.
In a statement accompanying its drug safety alert Oct. 31, the agency cautioned that “at this time, the FDA can not identify a dose level or exposure duration that is safe for patients.”
But responding to the rising tide of advocacy, they also have designated employees to listen to — and try to resolve — patients’ concerns. They also are quick to respond to feedback from doctors.
“We understand the need for access to products for the treatment of serious and life-threatening diseases and that there are some patients who are currently receiving [Iclusig] who will require continued drug access,” Pazdur, director of the hematology and oncology products office at the FDA Center for Drug Evaluation and Research, wrote Nov. 5 in response to the letter from 23 leukemia specialists.
Dr. Harry P. Erba, professor of internal medicine and director of the hematologic malignancy program at the University of Alabama in Birmingham, who wrote the letter signed by the specialists, said it was not intended to dispute the FDA safety warning. But, he said, “there was a sense of urgency that patients who are on the drug now not be allowed to relapse” because they were suddenly denied access to Iclusig.
Ariad executives are working with FDA regulators to come up with a strategy for reducing the drug’s risks and an amended label restricting Iclusig prescriptions to patients who can most benefit and for whom other drugs have failed.
More than 1,000 people in the United States were treated with Iclusig obtained commercially from January through October, when sales were suspended. Since then, Ariad’s stock has plunged and the company has laid off 160 workers, about 40 percent of its US staff, in a bid to conserve cash until the drug can be returned to the market.
“The most important thing for us is to make sure there’s access to this drug for patients who need it,” said Ariad chief scientific officer Timothy P. Clackson. But while Iclusig remains on the market in some European countries, Ariad is no longer being paid to distribute it in the United States under the FDA’s emergency program.
Hans Loland, a 45-year-old computer systems analyst from Woodinville, Wash., who has organized an Internet group for those diagnosed with chronic myeloid leukemia, said patients are hoping Ariad and the FDA can quickly reach agreement.
“We support the FDA and the job they have to do,” Loland said. “But we want a balanced approach. Our concern is the longer this is dragged out, there are patients who are going to lose access to the drug and there are doctors who may not prescribe it to patients who could benefit.”Robert Weisman can be reached at firstname.lastname@example.org. Follow him on Twitter @Globe-RobW.