The dizziness didn’t much worry Neil Fachon. Neither did the double vision. He was recovering from concussions he had sustained while playing sports, and the symptoms weren’t enough to keep him from his engineering classes at Northeastern University in Boston.

Then came some inexplicable struggles with handwriting and swallowing.

Doctors scheduled an MRI. On March 4, his life came unhinged.

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Oncologists at Massachusetts General Hospital told Neil, 20, that he had a rare and incurable form of cancer known as DIPG, or diffuse intrinsic pontine glioma, which attacks the brain stem and usually kills its victims in a year or less.

The diagnosis dropped the college sophomore into a maelstrom. Doctors laid out one course that seemed of little use. Herbalists recommended dietary changes. Neil and his family grew excited about an experimental therapy offered by a renegade Texas physician, Dr. Stanislaw Burzynski, and he flew to Houston to get infusions — but got them for just for a single day before federal regulators abruptly ordered the treatments to stop.

Soon, Neil’s family was suing the Food and Drug Administration for the right to keep trying the new therapy. Late last week, the Fachons won, and Neil resumed the treatments.

The family drama comes amid growing tension — in Congress and across the US — about how far terminally ill patients can, and should, go in search of a miracle.

The FDA tries to protect patients from unproven remedies that might do more harm than good. But when a child has an incurable brain tumor, does the same cold calculus of risk and benefit apply?

“Neil wanted to be part of this research,” Wendy Fachon, Neil’s mother, told STAT. “What did he have to lose — his life?”

For his part, Neil said in an interview Tuesday that he has been focusing on staying upbeat, even amid the “crazy stress” of fighting the FDA for the right to try the infusions. “I’m thinking ‘What can I still do?’” he said. “I can hold my head up. I can keep the most positive spirit I can, and help my parents get through this the best they can. And that’s precisely what I intend to do.”

Neil Fachon
Neil Fachon after having a catheter inserted in his chest in April. Courtesy Wendy Fachon

No time to wait

Modern medicine is a brutal battlefield: hope spars with fear. Risk with caution.

It’s the FDA’s job to ensure that treatments work, that trials are conducted safely, that potential for gain outweighs potential for harm. But regulators are under constant pressure from patients — and, increasingly, politicians — who feel the agency moves too slowly, demands too much data, and won’t let dying patients take a chance on experimental drugs.

Just this month, for instance, mothers of children with Duchenne muscular dystrophy, which is fatal, raged against regulators who have delayed approval of a drug for the disease. Parents call the treatment promising, but the FDA has expressed doubts about the drugmaker’s data, based on a 12-patient study.

“We’ll never have a perfect drug, but we’re trying to find a way to get the best of both worlds: minimize risk and maximize benefits,” said Karen J. Maschke, a political scientist at the Hastings Center and editor of the journal IRB: Ethics & Human Research.

“It’s a hard conversation to have,” Maschke added. “But we need to keep trying to have it, and have it ongoing so we can have it at times we’re not in crisis.”

The Fachons didn’t have the luxury of waiting for a good time to talk.

They knew that mainstream medicine had little to offer Neil beyond radiation to tamp down the tumor and perhaps buy him a few more months. Neil’s oncologists also said they could biopsy the tumor to see if he might qualify for experimental treatments. But a biopsy on the brain stem can be risky.

As they considered clinical trials, Wendy Fachon plunged into consultations with practitioners of naturopathic medicine.

Neil was soon eating heaps of kale and downing an herbal concoction. He took vitamins and high doses of curcumin, a substance in tumeric, prescribed by a woman with a degree in naturopathic medicine who studied with Cherokee elders.

The family had known about Stanislaw Burzynski, who promotes an experimental treatment made from amino acids and peptides that he says cancer patients lack. Burzynski has tangled for decades with federal and state regulators; they have accused him of unethical conduct, of sloppy record keeping — and of repeatedly jeopardizing his patients with risky treatments and drug overdoses.

Despite his record, the Food and Drug Administration authorized Burzynski to run a clinical trial of his treatment on a brain stem tumor. The clinical trial meant the family would not have to pay for the treatment, which can run in the tens of thousands of dollars and is not covered by insurance.

In mid-April, Neil became the one and only patient enrolled.

The Fachon family, who live in East Greenwich, R.I.,  had read critiques of Burzynski’s treatment on the websites of the National Cancer Institute (which says there’s no known benefit), and Memorial Sloan Kettering Cancer Center (which says it could be useful as a supplemental therapy for breast cancer). They’d read testimony, too, from Burzynki’s many impassioned, though less credentialed, fans. Neil and his sister, Evie, a senior at Northeastern, had even held a Skype conversation with a woman in Argentina who credited Burzynski with curing her fatal brain tumor.

“Neil wanted to be part of this research. What did he have to lose — his life?”

Wendy Fachon

So Neil and his parents flew to Houston, where they were later joined by Evie. There, Neil had an intravenous catheter inserted in his chest; it delivers the treatment every four hours, around the clock.

Camped out in hotel rooms in Houston, the family passed the time playing board games such as Settlers of Catan, Carcassonne, and Pandemic. More recently, Neil has spent more time on the computer, writing about his experience.

“He’s doing OK,” his mom said last week. “He’s very tired. We’re all very tired. But he does feel he’s improving.”

Everyone was shocked when the FDA abruptly ordered the trial shut down, without making public the reason. The agency reversed course just as abruptly, and just as mysteriously, last week, after the Fachon family filed their lawsuit. A federal court order dated May 27 indicated that the FDA would let Neil Fachon continue his therapy.

“I can’t put into words the stress and the mental trauma that the FDA put us through,” Wendy Fachon said. “How they treated us was absolutely abysmal.”

The FDA declined to comment. Burzynski also turned down requests for interviews.

Clinical trials do get stopped midstream occasionally, for various reasons, but Richard Jaffe, an attorney who represented Burzynski for years, said in general such action is taken only when there’s “a strong likelihood” that a participant in the trial will get harmed.

Jaffe has sued Burzynski for nearly $250,000 in unpaid legal fees, but says he still believes in the doctor and his experimental blend of compounds normally found in blood and urine.

“It may not be a perfect treatment and it’s not approved yet for marketing, but there’s some evidence” of benefit, Jaffe said.

Fachon said she understood the side effects of the experimental treatment to be minimal: imbalances in sodium or potassium. “But if you’re aware of that, and you stay away from salty foods and drink lots of water and eat bananas and take potassium supplements, you’re fine,” she said. “I think it’s brilliant.”

Neil, too, said he felt he was in good hands. Asked how he liked Dr. Burzynski, he responded: “I love him.”

Burzynski has “a twinkle in his eye,” Neil added. “But he cares very deeply about what he’s doing. He has a very real intent to help people.”

A doctor under fire

Burzynski has been promoting his experimental treatment, which he calls “antineoplaston therapy,” since the 1970s. He lists a string of scientific papers on his website, but he hasn’t published any data from randomized controlled trials testing the efficacy of his treatment against placebos or more conventional therapies.

Antineoplaston therapy is not approved by the FDA for the treatment of any illness.

Yet Burzynski pitches it as a “cutting-edge,” personalized cancer treatment. His clinic’s slogan: “Tomorrow’s cancer treatment today.”

Two documentaries about the doctor paint him as a visionary persecuted by the medical establishment. Dr. Mehmet Oz, the medical-doctor-turned-TV-personality, has promoted him. But regulators have long been skeptical.

In the 1980s, the Texas Medical Board reviewed Burzynski’s use of antineoplaston therapy on about 60 patients to see if it was safe. The results were inconclusive. In 1988, the board accused him of violating the Medical Practice Act, in part because he had given patients the therapy after the FDA had put a hold on clinical trials. A Texas trial court later threw the case out. The board has called for suspending his license for violating medical standards.

The FDA has come down on Burzynski, too. Of particular interest to the agency: the clinic’s Institutional Review Board, which is charged with approving the protocols of research trials and protecting patient safety. The FDA concluded in 2008 that the board didn’t do enough to minimize risk to trial participants, failed to ensure patients gave informed consent, and otherwise neglected its responsibilities.

Strikingly, the FDA also said that when a member of the review board had a conflict of interest, non-IRB members were allowed to step in as substitutes. FDA regulations prohibit that practice. The FDA sent several warning letters but did not shut down the clinic.

In 2012, a young boy died in one of Burzynki’s trials, and both the Texas Medical Board and the FDA again investigated. The results were troubling for Burzynski.

In a harsh warning letter to Burzynski, dated Dec. 3, 2013, the FDA said the doctor had “failed to protect the rights, safety and welfare of subjects under your care.”

Dr. Stanislaw Burzynski has “failed to protect the rights, safety and welfare of subjects” under his care in clinical trials.

Food and Drug Administration

The agency also noted that Burzynski had failed to report adverse reactions that patients suffered in the clinical trial, as required by law, and neglected to obtain informed consent, especially regarding additional fees for the treatment. He was also cited for failing to maintain accurate case histories — which are crucial to figuring out whether a treatment actually works.

And that wasn’t all. “Overdoses have been reported to you on a weekly basis during your Monday, Wednesday and Friday staff meetings. There is no documentation to show that you have implemented corrective actions during this time period to ensure the safety and welfare of subjects,” reads one federal inspection report from 2013.

At the same time, the Texas Medical Board revved up its pursuit of the Houston doctor.  In November 2014, the board filed another case against him, accusing him of violating ethical standards and the standard of care, among other charges. That case is pending.

Through it all, his fans have remained loyal. Rita Starr, who said the doctor successfully treated her mother in 1994, cofounded the Burzynski Patients Group to rally support. The doctor is now treating her husband for lymphoma. And she said she “personally knows” patients who have overcome brain malignancies after being treated by Burzynski.

Starr visited the clinic last week, gave Neil Fachon a new backpack to carry his treatment supplies in, and showed him photos of Burzynski patients who have survived cancer.

“The family knows they’re in a good place,” she said.

Fachon family
Dean, Evie, Neil, and Wendy Fachon at the Burzynski clinic last month. Courtesy Wendy Fachon

‘One of the worst tumors’

DIPG “is one of the worst tumors we have in pediatrics,” said Dr. Peter Manley, who directs the pediatric neuro-oncology outcomes clinic at the Dana-Farber Cancer Institute. There are about 300 cases a year in the US, mostly in  younger children.

The standard treatment is radiation to the brain stem. Manley said patients usually improve as a result, but the tumor tends to grow again within six to nine months. Most patients die within a year of diagnosis.

Research on alternative therapies is actively underway, however — including a clinical trial at the Translational Genomics Research Institute and the University of California, San Francisco, and another at St. Jude’s Children’s Research Hospital in New York.

In addition, Manley said researchers at Memorial Sloan Kettering and Weill Cornell Medicine are studying methods of injecting chemotherapy directly into the brain stem. The idea is to overcome a big weakness of standard chemotherapy — that it often does not effectively cross the blood-brain barrier.

“We’re hoping that we’ll learn more about these tumors and target them directly,” Manley said.

“What can I still do? I can hold my head up. I can keep the most positive spirit I can. … And that’s precisely what I intend to do.”

Neil Fachon

Wendy Fachon, who works for a magazine called Natural Awakenings Rhode Island, said she and her husband, Dean, a freelance writer, considered other clinical trials, but none of them appealed as much as Burzynski’s, given how many people had testified about positive experiences with the Houston doctor. She also continues to pursue herbal and naturopathic remedies.

Dr. Naoki Umeda, a staff physician at the Cleveland Clinic’s Center of Integrative and Lifestyle Medicine, said herbal treatments can effectively support — but should not replace — traditional cancer treatments, because they can boost the body’s immune system.

Remembering to smile

As the drama has unfolded, Neil Fachon has remained positive.

It comes easily to him.

In high school, Neil spent his summers in a student leadership program in Massachusetts — the last two as a volunteer, training the younger participants to hold fundraisers in which they read a particular book to elementary school students.

The book was “The Jester Has Lost His Jingle,” written by David Salzman, who died of Hodgkin’s lymphoma in 1990 at age 22.

In it, a jester regains his lost humor with the help of a girl who is battling brain cancer.

After Neil’s diagnosis in March, friends from his program sent him a copy of the book and a jester doll.

Neil said he wants to give copies to two young patients with DIPG who recently checked into Burzynski’s clinic. He wants them to know, he said, that “no matter how bad it seems, how down they are, there’s always, just around the corner, a smile.”

“Especially for kids going through this kind of thing — myself included — it’s something we need to be reminded of, because the whole situation can become so oppressive at times,” he said. “So to have that message of positivity and laughter and love for life … that’s the message I’m trying to bring to people.”

Meanwhile, Wendy Fachon said she is considering supporting legislative initiatives that might help patients in similar situations gain more control over their medical treatment. She has already heard of at least one other child who was blocked by the FDA from joining her son on Burzynski’s trial.

“My heart goes out to those families, because I know what it’s like,” she said.

As for Neil’s future, she said: “We are very optimistic for his recovery. His story can’t end now. I think his story is just beginning.”

Neil agreed.

“I’m feeling pretty good,” he said. “I don’t plan to stop feeling pretty good any time soon.”

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  • I feel thw FDA ahould stay out of our personal business when it comes to making medical decisions concerning our own bodies. Who are they to tell us what we can and can’t do? If we’re aware of the risk it should be our decision, not theirs. After all it is a clinical trial and it’s our life and money at stake. The FDA is going on about this the wrong way, cancer is never going away but the more people that are actually possibly healed by this treatment encourages lots more people to consider/participate and pay for this treatment. Is the FDA so indebited to big pharma that they can’t or won’t even consider the value or importance of peoples lives? There’s still money to be made here if that’s what there main interst is about, I’m pretty sure it’s not all about the patients safety and well being! Part of the medical othe is “to do no harm”, but that’s what the FDA and Dr’s are doing, they are putting us in harms way by restricting us of our choice of medical care and alternatives. There’s clinical trials that they approve of for all kinds of things, like ecigs, which could actually lead to cancer and they want to prevent a treatment that could possibly irradicate the disease, that’s so HYPOCRITICAL!!! I would risk my life by going out of this country for treatment for cancer just for the possibility of being cured. When are LIVES REALLY going to MATTER?

    • Hi, Pamela:
      The cruel reality is that almost no one (of health care system) is interested in seeing any kind of cheap and effective solutions. 3 trillion dollars (and up) annually must be spent to cover health care of chronic diseases. If you come up with a solution to cut that spending in just 5% then there will be more trouble than benefits coming to you. But if you (big pharma?) afford to pay clinical trials and get FDA approval, then you may charge a high price, without disturbing the whole health care system. See http://www.forbes.com/sites/matthewherper/2017/05/05/fda-approves-first-new-drug-to-treat-als-in-22-years/#66dcbea57fb3
      Well it doesn´t matter much if it actually can or cannot help ALS patients, since the disease modifying effect is so low it is almost impossible to verify.

      The above mentioned drug is used to slow the progression of ALS disease.
      You can achieve more than that with hydrogen which has the problem of too cheap to get and not protected by patent. By applying hydrogen you can stop and reverse the symptom meaning that ALS patients may recover their ability to work and get their lives prolonged.
      The same happens to COPD, lupus, Parkinson´s disease, cancer and many more. Most chronic diseases can be prevented/treated/cured. Patients are not well informed because their doctors are not well informed even though many studies have been published. Take COPD as an example. Researchers know about the benefits of hydrogen for treatment of COPD. But who cares? It is more profitable selling devices and drugs constantly until the patients die. If they cure a COPD patient in two weeks they would put their business in danger. (We cure the majority of late stage COPD patients in less than two weeks, by applying hydrogen gas inhalation).
      So in the end the question is not the right to try (investigational drugs) but to get well informed. NIH ( https://nccih.nih.gov/ ) should do a better job to collect, analyse, test/prove and disseminate information about alternative and complementary therapies. But this approach seems too difficult to take. Because the whole system is not in favor of cheap cure which really works.

  • Chemo injecting directly into the the brainstem? Sounds madness to me. Suffering daily because of a FQ antibiotic CIPRO that crosses blood brain barrier. The pains are from HELL when your CNE is on fire. I praid for death many times.

    • All of the other doctors my best friend saw told her to go home and get her affairs in order that they couldn’t help her. A few days afterward, Dr. Burzinsky accepted her as a patient (she weighed 85 pounds and was bleeding from one of her tumors at that point and several her organs were cancerous) because she had only had 1 treatment of chemo prior to her first visit to his offices. She’s now living in Mexico with a home in San Miguel de Allende and a condo in Playa Del Carmen with no signs of cancer. She improved drastically after each treatment and those were almost completely without side effects. On a side note, after 2+ years of her medical ordeal, I myself have symptoms of PTSD because of my fears for her and frustration with what she went through trying to survive her illness. If only she had gone to Dr. B in the beginning. Also, my brother, a firefighter who drove an ambulance for the Houston Fire Department, and on occasion transported some of Dr. B’s patients to the hospital, told me that all of them spoke very highly of Dr. B and raved about how he treated them so well. Most of said hospitals refused to treat his patients and my brother had to drive them around Houston until one would accept them. I immediately called Dr. Burzynski’s office for an appointment when I noticed a physical anomaly that I feared was a sign of cancer. I saw his son, who is physician and works with Dr. B. His son listened to my concern and then assured me that he knew of nothing that would indicate I had cancer. He sent me on my way (at that appointment) with no tests and said to come back if I had any additional concerns of if symptoms appeared. He could have run all kinds of tests but was professional and kind and made me feel at ease and I walked away knowing that he would have treated me if it was necessary.

  • I would simply like to thank Neil for his strong spirit and Dr. Burzynski for his magnificent ability to withstand so much negativity and trouble from the relentless attacks of government.
    Thank you both so much,
    Rob Lowis

  • I just watched the documentary. There are a few things I’d like to point out. First, this article stated the FDA was citing incomplete patient history but confiscated files. Hard to keep a consecutive narrative when your paperwork is “lost”.
    Second, it is my opinion that anyone diagnosed terminal should be given the choice of treatmemts.
    And third, watching the effects of both chemo and radiation aside, 72,000 people in the USA died of overdoses on opioids yet that medication is still widely available, over prescribed, and( from personal experience with many doctors in several states)NOT warned against! The medical community takes an oath to protect human life but shouldn’t each individual do that REGARDLESS OF VOCATION?!

    • Hi Heather! What a powerful impact your comment made on me. Perfectly said. People like you have to keep speaking, I live in Australia and your voice was heard here loud and clear.

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