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