FDA Forces Fatal Chemo on Kids

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FDA Forces Fatal Chemo on Kids 

 

By Kristen Philipkoski  |   Also by this reporter Page 1 of 2
 

02:00 AM Oct. 15, 2002 PDT

 

Michael and Raphaele Horwin never imagined that trying to save their son's life might mean he'd be taken away from them.

Two-year-old Alexander Horwin was diagnosed in 1998 with an aggressive form of malignant brain cancer called medullablastoma, which almost always kills children.

Alexander underwent two surgeries to remove the tumor. The first one left him temporarily unable to walk and with optic nerve damage. After the second, he was tumor-free, but the disease threatened to return if he didn't receive treatment.

Doctors recommended a "state-of-the-art" chemotherapy treatment. It was the best they had to offer -- but it could also damage Alexander's heart, lungs, liver and kidneys, and lead to loss of hearing, small stature, infertility, another cancer, intellectual decline and death.

It wasn't until after Alexander died that the Horwins discovered that in addition to those horrors, various medical journals reported that it was also ineffective for young children.

"After Alexander passed away, my wife and I wanted to find out why our 2-1/2-year-old son died while receiving 'state-of-the-art' chemotherapy at a prestigious children's hospital," Michael Horwin said. "We researched the medical literature and what we discovered astonished and sickened us."

Throughout the '70s, '80s and '90s, doctors reported in various journals that the drug Alexander received caused seizures, dementia and death, and that it is a carcinogen. One experiment was shut down early because the children's tumors spread within five months.

Still, according to FDA regulations, this treatment trumped an experimental treatment the Horwins wanted to use simply because the chemo (called CCG-9921) is FDA-approved -- for adult cancer. It is not, however, approved for the treatment of medullablastoma in small children.

Oncologists are still recruiting children with medullablastoma to test CCG-9921.

It's called "off-label" use of a drug, and it's common practice among physicians.

Why, then, couldn't the Horwins choose a different treatment -- one that wasn't linked to reports of devastating effects but did seem to have added years to some children's lives and possibly cured others?

Frenzied research had led the Horwins to Dr. Stanislaw Burzynski. His treatment seemed to be the most promising and least harmful. The Horwins found stories online of Burzynski's medullablastoma patients who were now healthy and had suffered few if any side effects.

But even though Burzynski's treatment was part of an FDA-approved clinical trial, the doctor had to turn them away. According to the FDA, only patients who had tried an approved treatment that had failed (meaning the tumor had returned) qualified for Burzynski's treatment.

Burzynski's controversial "antineoplaston" treatment is made of peptides originally discovered in human urine and now manufactured synthetically. The Polish-born doctor -- the only doctor in the world who offers this therapy -- claims that these protein fragments "turn off" cancer genes.

"When we were reluctant to bring in Alexander for chemo, the oncologists wrote in his chart that a court order might be necessary so that they could forcibly take our son from us," Horwin said. "That scared us."

The Horwins felt chemo was their only choice, and they were devastated. After eating healthy foods during her pregnancy, and making all of his baby food with organic ingredients, Raphaele Horwin held Alexander as doctors injected him with medication his parents knew would poison him.

After only three months of treatment, Alexander reported feeling pain in his lower back and neck. A CT scan revealed he had 30 new tumors, and doctors gave him a few days to live. He died three weeks later.

The FDA regulation that prevented Alexander from receiving Dr. Burzynski's treatment is part of the Food, Drug, and Cosmetics Act. It has been upheld by various lawsuits and state codes.

Critics of the requirement say the Horwins' experience is emblematic of a larger problem: Americans don't have the freedom to choose the health care that they and their physicians believe is best.

"Few Americans are aware that their treatment options, indeed their most personal medical choices, are regulated by the government and are seriously limited if they become ill," said Peter Chowka, an investigative journalist who has reported on alternative medicine for 25 years and served as a consultant for the National Institutes of Health's Office of Alternative Medicine. "Quite simply, there is very little freedom of therapeutic choice and only a half-hearted commitment to medical pluralism in this country."

Burzynski's and some other so-called "alternative" cancer treatments show encouraging signs in some patients, but they are effectively banned in favor of FDA-approved treatments that have gone through the rigorous and very expensive approval process.

 

The FDA says the rules are in place for good reasons, including the need to protect Americans from charlatans who want to take advantage of desperate, dying patients, as well as to protect children from parents who would fall for such schemes.

If Burzynski had disregarded the rule and treated Alexander, he would have faced prison time, a hefty fine and loss of his medical license.

"I am basically happy to treat all of these patients because we see good results of treatment," Burzynski said. "Unfortunately, my hands are tied up, and I certainly can't do anything about it unless the FDA will change their mind."

The FDA does have two programs to allow terminal cancer patients to access non-approved therapies currently in trials: the Investigational New Drug Application (IND) and a "single-patient," "emergency" or "compassionate IND."

"The FDA doesn't necessarily decide based on the drug; it is based on the protocol of the scientific research," said Laura Bradbard, a spokeswoman for the agency.

But in most cases, patients can participate in these programs only if no comparable treatment alternative is available.

"What does this mean? There is no case law on point, so currently it means whatever the FDA decides," wrote Michael Horwin in his article "War on Cancer": Why Does the FDA Deny Access to Alternative Cancer Treatments? published in the California Western Law Review.

Horwin, previously a health-care administrator, was so outraged by his son's and other children's treatment that he became an attorney, hoping to change the law.

Critics say patients and physicians, not the FDA, should have the power to make decisions about personal health care.

"If we're talking about protecting the public at large, that's quite a different matter from one sole party who is going to die by all accounts and really needs the greatest sympathy and protection from the government," said Jonathan Emord, a principal at the law firm Emord and Associates in Washington, D.C.

Dr. Thomas Wagner, head of cancer research at the Greenville Hospital System in South Carolina and a professor of molecular biology at Clemson University, described a scenario in which one of his patients died in pain and penniless because of a so-called "natural healer."

"The FDA is a much more benevolent organization than people seem to understand. In every case with every protocol there are exceptions," Wagner said.

In his experience, Wagner said, the FDA has been efficient about approving experimental drugs in special circumstances, although he said traditional therapies tend to get approved more often.

The Horwins and others have lobbied for change, partly in the form of the Access to Medical Treatments Act (AMTA).

"The AMTA must be passed by Congress, though -- the same Congress that over the years created most of the problems in the first place," Chowka said. "It's not surprising therefore that the AMTA has been languishing in Congress for almost a decade."

According to the National Cancer Institute, 1.3 million Americans will be diagnosed with cancer this year.

"Alexander lived only five months after being diagnosed and yet his medical bill totaled nearly $250,000," Horwin said. "If you take even one-half of $250,000 and multiply it by 1.3 million people, the dollars are staggering."

However, between 1973 and 1991, the cancer death rate increased, according to the National Cancer Institute. From 1991 to 1998, the death rate decreased, but there was no overall percentage change in the death rate between 1973 and 1999. Researchers predict that the overall cancer burden in the United States will increase.

"Our son is dead, but he generated over a quarter of a million dollars in revenue for the cancer industry within five months. As my wife and I said when we testified in front of Congress on June 7, 2000, cancer victims, especially children, should not be used as profit centers," Horwin said.

Jack and Mariann Kunnari faced the same tragic situation as the Horwins four years earlier.

Their son Dustin was diagnosed in 1994 with medullablastoma. Doctors recommended surgery, radiation and chemotherapy, and told them about the devastating side effects.

Since doctors said Dustin had only four months to live, the Kunnaris wanted to keep him as comfortable as possible in that short time.

"We just wanted him to have a quality of life," Mariann Kunnari said. "If he lived a year we would be happy with that -- even if he lived six months."

Like the Horwins, the Kunnaris found Dr. Burzynski. When they told their doctors they were thinking about this option, they were warned there was a chance social workers would step in and force Dustin to receive the traditional therapies.

"As if it isn't stressful enough to choose the best possible treatment for your child," Kunnari said. "Then to be threatened with having him taken away so they could give him a treatment that could kill him -- it was unbelievable."

But the Kunnaris were lucky. The FDA tightened its regulations in 1997, saying patients had to try approved therapies without success before going to Dr. Burzynski. But in 1994 the Kunnaris were not bound by this rule.

"You would never know he was sick," his mother said.

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