FDA agrees to let patients get controversial drug

The Food and Drug Administration has agreed to allow a handful of cancer patients to receive unapproved drugs from a controversial Texas doctor, but only if they can find another physician to administer them.

The drugs are made by Houston doctor Stanislaw Burzynski, who was the subject of a USA TODAY investigation last year. While his supporters consider him a medical maverick, mainstream doctors describe him as a snake-oil salesman. Burzynski has claimed for more than 36 years to be able to cure certain hard-to-treat brain tumors with drugs he calls antineoplastons. The National Cancer Institute notes that Burzynski has never published definitive evidence that his drugs cure cancer or even help people live longer.

Burzynski has been unable to give these drugs since 2013, when the FDA placed his experiments on hold after the death of a 6-year-old New Jersey boy taking antineoplastons. In December, the FDA sent Burzynski a warning letter, noting that he inflated his success rates and failed to report side effects and to prevent patients from repeatedly overdosing. The Texas Medical Board also charged Burzynski last year with false advertising.

None of that has deterred a group of eight families whose loved ones are suffering from incurable brain tumors and who would like to try Burzynski's patented therapy. Those families include the parents of 12-year-old McKenzie Lowe of New Hampshire; the parents of 6-year-old Refael Elisha Cohen of Texas; and 47-year-old Liza Cozad-Lauser of California.

All contacted the FDA to ask for "compassionate use" of antineoplastons.

The FDA acknowledged Friday that it has agreed to allow them to use the experimental drug, but only if they can find a qualified, independent physician to administer the drug. Beyond infusing the drug and overseeing their care, the doctor would have to formally apply for expanded access to an "investigational new drug," as well as get approval from an institutional review board, an independent panel that reviews safety and ethical issues involved in clinical trials.

The FDA grants an average of more than 1,000 requests a year for expanded access.

Burzynski will supply antineoplastons for free, says his attorney, Richard Jaffe.

While Cozad-Lauser has found a doctor willing to administer the antineoplastons, McKenzie's family members say they have contacted dozens of doctors, but have been unable to find a qualified oncologist to agree to participate.

"I'm an adult. I should be able to make this decision," says Cozad-Lauser, who was diagnosed with an inoperable brain stem tumor, diffuse intrinsic pontine glioma, or DIPG, 18 months ago. Although the tumor is diagnosed in 200 to 300 people a year, there are few long-term survivors. "My own government is telling me I can't choose a medicine. I should have the right, when there is no standard of care available to me and every doctor had told me I have no chance of survival."

Refael's family posted an update on their Facebook page, asking friends to help them find a doctor in the New York area, where the child is in the hospital. Because Refael is in a coma, however, he's unlikely to benefit from antineoplastons, says attorney Antonio Martinez, a lobbyist working with the ANP Coalition, a group advocating for antineoplaston access.

"It's the FDA's job to protect the public, but when does the FDA's authority go too far?" asked Martinez. Addressing Refael's case, he asks, "Why couldn't they (the FDA) have allowed him to try ANP six months ago? Now, he has almost no chance."

McKenzie's grandfather, Frank LaFontain, says he hopes to get treatment for his granddaughter, who has had chemotherapy and radiation, while she's still healthy. Today, he says, "she runs, she plays basketball for her school, there's no stopping her. She just keeps going."

Burzynski has attracted a large following over the years from supporters of alternative medicine and critics of the FDA, with whom he has often done battle. A new study in JAMA Internal Medicine finds that about half of Americans believe in medical conspiracies, such as the notion that government and industry are hiding cures from the public.

Others have been critical of the FDA's tolerance for Burzynski.

"I have no idea why the FDA would do this after finally showing signs of doing its job with respect to Burzynski," says oncologist David Gorski, whose blog, Respectful Insolence, has long been critical of Burzynski. "It's a major propaganda victory for Burzynski."

In a statement, the FDA says the agency has a duty to protect patients from unproven products that could do more harm than good.

"Patients, including those who are terminally ill, deserve this protection when taking a product that has not been found to be safe and effective," the FDA said in its statement. "We recognize that there is a range of opinion about the extent to which individuals should be able to decide for themselves how much risk they are willing to tolerate from an experimental therapy."

Richard Saunders, whose daughter Amelia was treated by Burzynski, says he welcomes the FDA's decision. Amelia, from the United Kingdom, died of cancer at age 4, in 2013.

"If there is any way for patients to get access to antineoplastons, and have the choice to do so — but under controlled, professionally guided circumstances — then that has to be a good thing," says her father, Richard, who wrote a book about his experience, called Saving Amelia.

Some doctors and patient advocates say they're disappointed by the FDA's decision.

Tina Cowles, whose husband died of lung cancer after being seen by Burzynski in 2012, says antineoplastons may deserve further study. But she's glad that it won't be done by Burzynski.

"I would agree with not letting Burzynski anywhere near any cancer patient," says Cowles, who said she felt "duped and disappointed" after going to Burzynski's clinic. "He strikes me now as egotistical and paranoid. And oh, what a financial empire he as built on the backs of the desperate and dying."

Jan Buckner, chair of oncology at the Mayo Clinic in Minnesota, says he wouldn't advise patients to take antineoplastons.

"There have not been well-conducted studies that demonstrate efficacy of antineoplastons for patients with any malignancy," says Buckner, who tried to conduct a rigorous clinical trial of antineoplastons in the 1990s, until Burzynski withdrew support for it. "There are known toxicities. Since there is not clear benefit, I would not advocate treatment with antineoplastons outside the setting of a well-designed clinical trial."

Oncologist Howard Ozer says he's disappointed that the FDA is allowing patients to take antineoplastons outside of a rigorous clinical trial.

"This is a cop-out," says Ozer, director of the University of Illinois Cancer Center, who studied Burzynski's research results in the 1990s.

"Because it can be toxic and cause life-threatening sodium problems, patients (who take antineoplastons) are at risk and could die," Ozer says.

The scientific community will learn nothing by giving these patients antineoplastons, because there will be no comparison group, Ozer says. Any doctor who agrees to give antineoplastons could be biased in favor of them, which could skew the results.

Oncologist Henry Friedman says the FDA made a savvy political decision in granting compassionate use of the drugs.

"It will deflect criticism from anybody who says the FDA is unilaterally denying dying patients something that could help them," says Friedman, deputy director of the Preston Robert Tisch Brain Tumor Center at Duke University in North Carolina, who also reviewed Burzynski's studies in the 1990s. "It will get FDA off the hook."

Burzynski declined to comment.

Sandy Smith, who took her son Andrew to see Burzynski, says she approves of the FDA's decision, because it could allow scientists to learn if antineoplastons have any value.

"With true science, if something is working, then someone else should be able to come up with the same results," says Smith, a breast cancer survivor from Michigan, whose son died of a DIPG at age 8 in 2009.

Smith says she felt misled by Burzynski, who seemed to wash his hands of their case when her son developed complications. Their family spent $24,000 in two weeks at Burzynski's clinic — more than her husband, the minister at a small church, makes in a year. They paid Burzynski's fees with donations from other churches.

Smith says the FDA plays an important role in protecting vulnerable families from opportunists who prey on people suffering from life-threatening diseases.

"We can't just have a free-for-all where everyone feels entitled to do anything and there is no consideration of safety," she says.

Yet Smith says she can understand the desperation of families.

"I wish it were easy to say, 'This doesn't work. Don't waste your time on this,'" she says. "You don't want to take away someone's hope."


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