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Desperate patients seek access to drugs in testing stages

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MIAMI - By early 2002, after little more than a year of treatment, Jennifer McNeillie had tried all of the approved drugs to fight her colon cancer, but her tumors had not disappeared.

McNeillie's husband, Steve Walker, researched cancer treatments and found two experimental drugs - Erbitux and Eloxatin - he thought could help his wife. Both had shown promise in early trials, but neither had yet been approved by the Food and Drug Administration.

Walker tried to enroll his dying wife in clinical trials for the two drugs, but she didn't fit the specific criteria for any of them.

So Walker found himself at what one expert called ``the intersection of desperation and regulation'' - unable to get his hands on two drugs he thought might help save his wife's life.

The number of people in Walker's position has grown in recent years, as patients have increasingly taken treatment decisions into their own hands and the Internet has dramatically expanded the data available to the public.

In some cases, a doctor, drug company and the Food and Drug Administration will jointly agree to give a patient an experimental drug, a term known as compassionate use.

But compassionate use poses difficult ethical questions for doctors, drug companies and regulators. Giving patients the untested drugs they want can be dangerous and can drain resources from clinical trials, which are the way drugs get approved and become available to the masses.

As a company, our ethical obligation is to ensure ultimate access to the drug, and ultimate access is FDA approval,'' said Mary Lynn Carver, a spokeswoman for the pharmaceutical company AstraZeneca, whose drug Iressa was in great demand before its approval in 2003.And the way to get FDA approval is through clinical trials.''

But the clinical trials process is long and complex, involving at least three rounds of testing and often stretching over five years or more. When a new treatment shows promise early on, desperate patients often want access long before the treatment is approved - or proven effective.

Dr. W. Jarrard Goodwin, director of the Sylvester Comprehensive Cancer Center at the University of Miami, cited the recent history of bone-marrow transplants for breast cancer patients.

Bone-marrow transplant for patients with recurrent or extremely serious breast cancer was something that came into practice because patients wanted it so badly and doctors began to believe in it,'' Goodwin said.It came into common clinical practice before it was proven to be of benefit, and then it was proven not to be of benefit. So there were lots of women who went through stem-cell bone marrow transplant - a very involved, debilitating and expensive procedure - and then it was found not to be effective.

``That was more of a watershed point in both investigators and patients realizing the importance of clinical trials,'' Goodwin said.

There is no comprehensive database of experimental drugs, so even learning what exists can be a daunting process.

``I was calling doctors all over Florida, I was calling the companies, I was calling the FDA, I was calling major medical centers,'' Walker said of his search for experimental drugs for his dying wife.

Marshall Gilula's training as a physician helped him track down a promising drug when he was fighting throat cancer. He searched the medical literature and found an experimental drug called Onyx 015 that targeted a genetic deficiency common to some cancers - including his.

Finding a promising drug is only the first step, though. A patient still has to persuade a doctor to apply for compassionate use. This often requires both a pushy patient and a compassionate doctor.

In Gilula's case, the doctor was Goodwin.

``Egged on by this unique individual, my staff and I jumped through a lot of hoops'' to get Gilula the drug he wanted, Goodwin said.

Goodwin and his staff did about 100 hours of extra work - much of it paperwork - to procure Onyx 015. Gilula said he owes his life both to the drug and to Goodwin.

The FDA says it gets hundreds of applications like Gilula's - one doctor, one patient - every year. Each one is reviewed individually.

``We have to know about every human being who is getting an unapproved drug,'' said Patty Delaney, associate director of the FDA's cancer liaison program.

Rarer are so-called large-scale, expanded access programs, Delaney said. If demand for a particular experimental drug becomes unusually strong, pharmaceutical companies occasionally establish ``expanded access'' programs that dispense the drug to hundreds or thousands of patients.

The largest of these programs in recent years was for Iressa, a cancer drug that showed great promise in early trials for treatment of lung cancer.

We had calls from congressmen, we had calls from leading businessmen, heads of state in different parts of the world,'' Carver said.They wanted access to this drug under compassionate use.''

In response, the company established an expanded access program in 2000. By the time the drug was approved in May 2003, more than 20,000 people had received the drug through the program, Carver said.

But not everyone who wanted the drug got it.

In 2001, Frank Burroughs' daughter Abigail was dying of throat cancer, and Burroughs believed Iressa might help. But the drug had only been tested in lung cancer, and AstraZeneca wouldn't give the drug to patients with other cancers.

It was an extremely difficult decision for us,'' Carver said.We would have loved to have been able to allow access, but we also had to look at the bigger group'' of present and future lung cancer patients.

Abigail Burroughs died in June 2001, and five months later her father founded the Abigail Alliance, a nonprofit organization, to lobby for broader access to experimental drugs.

Steve Walker's wife died in June 2003. He had managed to get her onto Eloxatin and Erbitux at various times, but he could never get a steady supply of the experimental drugs.

``We never got what we needed when we needed it,'' he said.

Since his wife's death, Walker has helped Burroughs lobby the FDA to expand dying patients' access to experimental drugs. But other cancer advocacy groups have been wary, arguing that the proposal could slow the clinical trials process, hampering both the scientific understanding of new drugs and their ultimate approval by the FDA.

Walker thinks the Abigail Alliance's proposal has not been more popular because those who might benefit from it are in no position to lobby for the cause.

These terminal cancer patients are completely underrepresented in the system and they die,'' he said.They don't live long enough to form a politically powerful group.''



There is no single database that lists all the experimental drugs, but here are a few resources:, a Web site run by the federal government, lists thousands of ongoing clinical trials., run by a nonprofit patient-advocacy group, lists some expanded access programs and experimental drugs in advanced trials., the Web site of the National Cancer Institute, has a searchable database of clinical trials on cancer drugs.


(c) 2004, The Miami Herald. Distributed by Knight Ridder/Tribune News Service.


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