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LINDEN, N.J. - On the last day of his life, Josia Cotto's parents gave him a choice.

The6-year-old boy had been fighting an inoperable brain tumor for 10months. When his mother, Niasia Cotto, found him in his bed,unresponsive and unable to open his eyes, "we knew there was nothingelse that we could do," she said.

An ambulance took Josia to ahospice room at a local hospital. His parents covered him in a soft,blue-and-white blanket, hugged him and held his small hand for the lasttime.

"We told him the choice was his, whether to keep fighting or be in peace with God," said his mother. "He chose."

Josia's parents would have paid any price to save him.

A Texas doctor, two months, earlier, had given them one: $25,000 upfront, by cash or check.

Clinging to hope, the Linden, N.J., couple took Josia to see Stanislaw Burzynski, a Houston doctor claiming to be able to do what no one else can: cure inoperable pediatric brainstem tumors.

Virtuallyany other doctor might have recited the same sad statistics: Althoughdoctors can now cure 83% of pediatric cancers in the U.S., there isusually no hope for kids with Josia's tumor. Perhaps 5% survive fiveyears.

Burzynski - an internist with no board certification or formal training in oncology - has said publicly that he can cure half of the estimated 200 children a year diagnosed with brainstem tumors. The Cottos were told that treatment could cost over $100,000, mostly out of pocket, because insurance plans often refuse to cover Burzynski Clinic treatments.

Burzynski, 70, calls his drugs "antineoplastons" and says he has given them to more than 8,000 patients since 1977.

Heoriginally synthesized these sodium-rich drugs from blood and urine -the urine collected from public parks, bars and penitentiaries. Althoughthey've been made in a lab since 1980, they still carry a distinctiveand unpleasant odor.And while the experimental drugs have not been approved by the Food andDrug Administration, Burzynski has described them like the holy grailof cancer therapy: safe, natural and highly effective. He has alsoprescribed them as a treatment for AIDS, lupus and other conditions.

STORY: Experts dismiss Burzynski's cancer claims

STORY: Families run out of hope, money after treatments

Some patients are convinced that he saved their lives.

MaryJo Siegel of Ventura, Calif., says she believes Burzynski cured herlymphoma. James Treadwell from Coronado, Calif., credits Burzynski withcuring his brain tumor. Jenny Gettino of Syracuse, N.Y., says Burzynskicured her daughter of an infant brain tumor.

Yet the National Cancer Institutesays there is no evidence that Burzynski has cured a single patient, oreven helped one live longer. He has not backed up his claims bypublishing results from a randomized, controlled trial - considered the gold standard of medical evidence - in a respected, peer-reviewed journal.

And Burzynski's drugs pose a risk of serious harm, including coma, swelling near the brain and death, according to the NCI and informed consentdocuments that patients sign before beginning treatment. WhileBurzynski has touted his treatments as an alternative to chemotherapy, a1999 NCI study foundthat antineoplastons can cause many of the same side effects asconventional chemo: nausea, vomiting, headaches, muscle pain, confusionand seizures.

Many blame the system for failing to protect patients.

"He'sa snake oil salesman," says pediatric oncologist Peter Adamson, aprofessor of pediatrics and pharmacology at Children's Hospital ofPhiladelphia. "This has gone on for so many years, it's reallyunbelievable."

For 36 years, critics say, Burzynski has beenselling false hope to desperate families at the most vulnerable time oftheir lives.

"When you want so hard to believe something, you end up listening to your heart and not your head," says Lisa Merritt of Armuchee, Ga., whose husband, Wayne, was treatedbriefly by Burzynski in 2009. The couple say that Burzynski misled themabout the type of treatment that would be offered, as well as the cost.Burzynski, she says, is "the worst kind of predator."

There aremany reasons why Burzynski has been able to stay in business so long. Hehas benefited from state laws that limit the Texas Medical Board'sauthority to remove his license, as well as the ability of terminallyill patients to collect damages. His devoted followers are willing tofight for him. He also has exploited the public's growing fascinationwith alternative medicine and suspicion of the medical establishment.

At times, Burzynski also has had an especially influential ally: the Food and Drug Administration.

FDA CHANGES COURSE

Although"there were some stormy relations with the FDA" in the past, Burzynskisaid in an interview, "now, we have a productive relationship."

For years, the FDA tried to prevent Burzynski from prescribing unapproved drugs.

In 1995, a federal grand jury indictedBurzynski on 75 felony charges, including criminal contempt, mail fraudand violations of the Food, Drug and Cosmetic Act. As a condition ofhis bail, a judge ordered him to stop prescribing antineoplastons. For atime, it looked as if Burzynski might never treat another patient.

Dozensof Burzynski's patients flocked to Washington to defend him, arguingthat taking away antineoplastons was akin to a death sentence. Siegel,who credits Burzynski with curing her lymphoma 22 years ago, hastestified on his behalf five times - once at his criminal trial and fourtimes at hearings on Capitol Hill.

Facing both a political andpublic relations firestorm, the FDA in 1996 abruptly changed course. Itoffered to allow Burzynski to continue treating patients, but onlythrough an official trial.

"Withone stroke of the pen, the FDA made legal what it had previously saidwas illegal," says Burzynski's attorney, Richard Jaffe.

Yet evenJaffe has acknowledged that the trial - now in its 17th year - was moreabout politics than science. In his 2008 memoirs, Galileo's Lawyer, Jaffe called it "a joke."

"It was all an artifice, a vehicle we and the FDA created to legally give the patients Burzynski's treatment," Jaffe said.

"With political help, you can get the FDA to say yes," says Siegel, 63.

The indictments led to two trials. In 1997, one of Burzynski's criminal trials ended in a hung jury; the other, an acquittal.

Today, the FDA refuses to comment on Burzynski.

NO NEW DRUG APPLICATION

Even his staunchest supporters wonder why Burzynski's drugs are nowhere close to receiving FDA approval.

"He'scuring cancer," says Siegel, who co-founded the Burzynski Patient Groupto spread the word about his therapies. "So why, why won't the FDAapprove it?"

Like many of Burzynski's supporters, Siegel suspects that the medical community and drug industry are aligned against him.

"Whydoes a doctor who can produce such extraordinary results continue to beattacked today?" Siegel asks. "The reason is because Dr. Burzynski andhis patented discovery pose the greatest threat to an entrenched medicalmonopoly."

In fact, the FDA hasn't had a chance to approve Burzynski's drugs. He has never officially asked.

Although Burzynski said he has completed 14 intermediate-phase studies, he has yet to file a new drug application, the final step toward getting a drug approved.

That hasn't stopped Burzynski from using his relationship with the FDA to recruit patients.

StaceyHuntington says she took her daughter to see Burzynski last year partlybecause the FDA's oversight made his therapies seem safer and morepromising. "My fear took us to Houston, and the hope he gave us made usproceed," says Huntington, of Chehalis, Wash.

In an interview, Burzynski said developing new drugs is complex and takes time.

Yet the FDA has approved 108 cancer drugs since Burzynski began his trial.

Curerates for one type of pediatric brain tumor - medulloblastoma - are now85%, according to St. Jude Children's Research Hospital in Memphis.Doctors can cure 95% of kids with Hodgkin lymphoma (a cancer of thelymph system), acute lymphoblastic leukemia (a blood cancer) andretinoblastoma (an eye tumor).

Fran Visco, president of the National Breast Cancer Coalition, describes the FDA's tolerance of Burzynski as "outrageous."

"Theyhave put people at risk for a long time," says Visco, an attorney andbreast cancer survivor. "That's completely unacceptable. How can anyonelook at these facts and believe that there is a real clinical trialgoing on ... rather than just using the FDA and the clinical trialsystem to make money?"

Burzynski dismisses criticism of his work, referring to his detractors as "hooligans" and "hired assassins."

Asfor criticism from former patients, Burzynski says, "We see patientsfrom various walks of life. We see great people. We see crooks. We haveprostitutes. We have thieves. We have mafia bosses. We have SecretService agents. Many people are coming to us, OK? Not all of them arethe greatest people in the world. And many of them would like to getmoney from us. They pretend they got sick and they would like to extortmoney from us."

History will vindicate him, Burzynski says, justas it has vindicated other persecuted medical "pioneers," such as LouisPasteur. In the future, Burzynski says, everyone will use his therapies,and the cancer treatments used today - such as surgery, chemotherapyand radiation - will be regarded as barbaric. "There will be a time whenpeople will see the light," he says, "and our treatments will be usedby everyone."

FDA IMPOSES NEW RESTRICTIONS

The FDA's patience with Burzynski apparently wore out after Josia died.

Ina report sent to the FDA after the boy's death, Burzynski's staffacknowledged that his last blood sample, taken the day he passedaway,showed a blood sodium level of 205 millimoles per liter, a levelthat is typically fatal. Burzynski's staff blamed that reading on a"false laboratory report based on a contaminated sample."

Yet hypernatremia is one of antineoplastons' most common side effects, known to doctors for two decades.

One of Burzynski's own informed consent documents - the form that patients sign before they begin treatment - put the risk at 21%.

On July 30, 2012 - six weeks after Josia's death - the FDA forbade Burzynski from giving antineoplastons to any new children.

Six months later, the FDA expanded its "partial clinical hold," forbidding Burzynski from giving the drugs to new adult patients, according to the Burzynski Research Institute's 2013 filing to the Securities and Exchange Commission. About 10 patients who were already receiving antineoplastons were allowed to continue, to avoid interruption of care.

According to FDA inspections performed after Josia's death, Burzynski has failed to report at least 18 hypernatremia cases.

The FDA publicly announced the restrictions on Burzynski's clinical trial for the first time in September.

Accordingto the FDA, the Burzynski institutional review board - an outside bodycharged with protecting patients - failed that most basic duty. In aletter announcing the restrictions, the agency said it has "no assurance" that the board was "adequately protecting the rights and welfare of the human subjects."

The FDA based its decision on "objectionable conditions" and a "continuing pattern of deficiencies found during the last three inspections," the letter said.

FDA inspectors also faulted Burzynski personally, as principal investigator of the study, according to inspectionsconducted from January to March. Copies of these reports were obtainedthrough a Freedom of Information Act request. Addressing Burzynski, theinspectors wrote, "you failed to protect the rights, safety and welfareof subjects under your care."

Inspectors charged Burzynski, as principal investigator, with a variety of other serious offenses, some dating to 2001. Among them:

• Inflating success rates in 67% of cases, by inaccurately reporting how tumors responded to treatment.

• Destroying patients' original records.

• Failing to report "unanticipated problems" to the institutional review board - sometimes for six or seven years.

Inthe inspections conducted this spring, officials noted four cases from1998 or 1999 in which patients were hospitalized for serious issues -such as pneumonia, lack of consciousness or bleeding in the skull - thatBurzynski researchers failed to reportuntil 2005. The FDA found similar problems in a 2001 inspection, whenofficials noted that Burzynski failed to report problems such aspneumonia, blood infections and pancreatitis, a life-threateninginflammation of the pancreas.

• Failing to protect patients from overdosing.

Forty-eight patients suffered a total of 102 drug overdoses from 2005 to 2013.

While the overdoses made some of these patients excessively sleepy, one had a seizure and another was hospitalized in intensive care with a breathing tube.

This represents a continuing problem, dating to reports of overdoses in inspections as early as 2001.

Burzynski'sreview board also repeatedly rubber-stamped his requests to givepatients antineoplastons outside of a clinical trial, the FDA'sSeptember letter suggests. In some cases, those decisions were madewithout consulting patients' medical records, or were made not byoncologists, but by a single member of the board, a "waterrehabilitation" specialist with no medical training.

Althoughresearchers do sometimes provide experimental drugs outside of clinicaltrials, exceptions should be rare, with perhaps one or two cases pertrial, Adamson says. In Burzynski's case, these "compassionate use"exceptions were common, FDA records show.

Enrolling patients forcompassionate use can be lucrative. Although researchers cannot chargefor experimental drugs, Burzynski does bill patients for relatedsupplies and services.

In Burzynski's defense, Jaffe notes thatinspection reports represent preliminary findings. The FDA has not yetissued final conclusions.

And Burzynski has taken issue with many of the FDA's findings.

In his written responseabout the FDA's claims that he inflated his success rates, Burzynskisaid that he "complied with all criteria for evaluation of response andmade accurate assessments for tumor response."

As for overdoses,Burzynski said in an interview that his staff works hard to trainpatients and their families to administer antineoplastons correctly.

None of the overdoses was fatal, he said.

"Theamount of medication that these patients receive is not dangerous,"Burzynski said. "At worst, they would sleep for a few hours."

Visco, the breast cancer advocate, says she's encouraged to hear that the FDA has put Burzynski's trial on hold.

"Itis about time that the FDA stepped in to stop Burzynski from subjectingmore patients to harm," she says. "I do not know why it took so long."

BURZYNSKI STILL HAS OPTIONS

The FDA can't put Burzynski out of business. No matter what happens to his trial, Burzynski holds a license to practice medicine in Texas.

Sodoes his son, Gregory Burzynski, a doctor who's helping to carry on hisfather's business. As vice president of the Burzynski Clinic, his son,34, works closely with his father and "oversees many operations" of theclinic, according to its website.

These days, doctors at the Burzynski Clinic are looking beyond antineoplastons. They mostly prescribe chemotherapy.

That'sa huge shift. During Burzynski's criminal trial in the 1990s, patientswho rallied to his defense carried signs reading, "Say No to Chemo."

Butthe Texas Medical Board, which has repeatedly tried and failed to putBurzynski out of business over the years, still questions Burzynski'scare.

The board charged Burzynskiin 2010 with violating state medical standards by prescribing legalcancer drugs in "random" and unapproved combinations, with no knownbenefits but clear harms.

Burzynski got those charges dropped in 2012, by successfully arguing that he didn't sign any of the prescriptions in question.

Burzynski is scheduled to go before the medical board again in January,based on a complaint filed by Stacey Huntington, whose daughter wastreated with antineoplastons for a brain tumor. At the meeting, a boardpanel "will hear the case and make recommendation to the full boardabout what disciplinary action, if any, is appropriate."

Huntington, who paid Burzynskinearly $34,000 for about six weeks of care, says she's concerned aboutboth billing irregularities and the quality of her daughter's treatment.Her daughter, Abra Hall,27, developed a life-threatening blood infection called sepsis afterleaving the clinic to continue treatment at home. The infectiondeveloped in a catheter in Hall's chest, which was used to administerthe antineoplastons, Huntington says. One month after developing sepsis,Hall was hospitalized again with a lung infection. Hall also developedserious complications from high doses of steroids, Huntington says.

Huntington says she decided to speak out to prevent other families from being taken advantage of.

"When you get a diagnosis of cancer, you are pretty vulnerable," she says. "I think they take advantage of that."

COTTOS NOT SURE WHAT TO THINK

No one told Josia's parents about any of this.

Not Burzynski. Not the FDA.

Joseand Niasia Cotto had no idea that their son's death prompted aninvestigation by the FDA, until they were contacted by USA TODAY.

TheCottos had long believed that Burzynski could have cured their son ifonly they had taken Josia to see him first, before giving him radiationand chemotherapy. They had even hoped to launch a non-profit, A Life forJosia Foundation, to help other children with cancer gain access toBurzynski's treatment.

Now, they don't know what to think.

Althoughmore than a year has passed since they lost their son, the Cottos saythey see reminders of him everywhere. Niasia, 32, says she feels hispresence in simple things, such as the light of a bright star on a darknight.

"He's still with us," says Jose, 33. "I know God had his plan and his purpose for Josia."

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