Federal center pays good money for suspect medicine

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Thanks to a $374,000 taxpayer-funded grant, we now know that inhaling lemon and lavender scents doesn't do a lot for our ability to heal a wound. With $666,000 in federal research money, scientists examined whether distant prayer could heal AIDS. It could not.

The National Center for Complementary and Alternative Medicine also helped pay scientists to study whether squirting brewed coffee into someone's intestines can help treat pancreatic cancer (a $406,000 grant) and whether massage makes people with advanced cancer feel better ($1.25 million). The coffee enemas did not help. The massage did.

NCCAM also has invested in studies of various forms of energy healing, including one based on the ideas of a self-described "healer, clairvoyant and medicine woman" who says her children inspired her to learn to read auras. The cost for that was $104,000.

A small, little-known branch of the National Institutes of Health, NCCAM was launched a dozen years ago to study alternative treatments used by the public but not accepted by mainstream medicine. Since its birth, the center has spent $1.4 billion, most of it on research.

A Tribune examination of hundreds of NCCAM grants, dozens of scientific papers, 12 years of NCCAM documents and advisory council meeting minutes found that the center has spent millions of taxpayer dollars on studies with questionable grounding in science. The cancer treatment involving coffee enemas was based on an idea from the early 1900s, and patients who chose to undergo the risky regimen lived an average of just four months.

The spending comes as competition for public research money is fierce and expected to get fiercer, with funding for the NIH expected to plateau and even drop in coming years.

"Some of these treatments were just distinctly made up out of people's imaginations," said Dr. Wallace Sampson, clinical professor emeritus of medicine at Stanford University. "We don't take public money and invest it in projects that are just made up out of people's imaginations."

"Lots of good science and good scientists are going unfunded," said Dr. David Gorski, a breast cancer researcher at Wayne State University, who has been a vocal critic of NCCAM. "How can we justify wasting money on something like this when there are so many other things that are much more plausible and much more likely to result in real benefit?"

The director of the center and other advocates say it is worthwhile to use taxpayer dollars to study certain alternative treatments.

"They deserve scientific attention," said NCCAM Director Dr. Josephine Briggs, who noted that the center's $128 million annual allotment amounts to less than half a percent of the total NIH budget.

Briggs, a respected NIH researcher and physician who has headed NCCAM for nearly four years, said in an interview that she is dedicated to evidence-based medicine and that the center, under her leadership, is committed to rigorous scientific studies.

The center's recently adopted strategic plan focuses on studies of supplements and other natural products along with the effect of "mind and body" therapies like yoga, massage and acupuncture on pain and other symptoms. In fiscal years 2008-2011, NCCAM funded more than $140 million in grants involving mind and body therapies, including $33 million for pain research in fiscal 2011.

The new strategic plan "reflects real change or an evolution in our mission," Briggs said. "We are not your grandmother's NCCAM."

Studies of energy healing or distant prayer likely would not get funded by NCCAM today, she said.

Yet many mind and body treatments that are being studied, like qigong and acupuncture, also involve the purported manipulation of a universal energy or life force, sometimes called qi — metaphysical concepts unproved by science and incompatible with our modern understanding of how the body works.

In an email, Briggs wrote that it isn't necessary to invoke qi or other ancient concepts to study therapies that may benefit people with chronic pain, a significant health problem.

NCCAM's continuing interest in acupuncture comes even though many of its studies have found that acupuncture and similar therapies work no better than a sham treatment at easing symptoms like pain and fatigue.

To most scientists, that would mean the treatments are failures — drug companies cannot sell medicines that work no better than salt water or a sugar pill. But in the case of acupuncture and other mind and body medicine, the center and its supporters say it's unclear whether the benefits represent a placebo response or something more complicated.

Critics of the center say it's telling that NCCAM was conceived not by scientists clamoring to study alternative medicine, but by Sen. Tom Harkin, D-Iowa, a member of the powerful Senate subcommittee that helps oversee the NIH budget.

In a 1998 speech, Harkin described watching acupuncture and acupressure ease the pain and violent hiccups of a brother dying of thyroid cancer.

"These are things I have seen with my own eyes," said Harkin, who also lost three other siblings to cancer. "When I see things like this I ask, 'Why? Why aren't these things being researched?'"

A few months later, NCCAM was created through a dozen or so paragraphs added to a budget bill.

The center's main mission was clear: Study alternative therapies and how they could be integrated into conventional treatment.

Because of its origins and purpose, NCCAM has a duality not often found in scientific institutions.

"They are serving two very different masters," said Art Caplan, a bioethicist at the University of Pennsylvania. "At the end of the day, they don't want to turn themselves into 'the institute for showing complementary and alternative medicine is bogus.' Then no one will support them who is pro-complementary and alternative medicine."

Briggs said she has not been subjected to any political pressure in her tenure.

$34 billion business

Americans spend about $34 billion each year out of pocket on complementary and alternative therapies, according to a national survey conducted in 2007 by the U.S. Centers for Disease Control and Prevention.

The survey found that about 40 percent of American adults reported using some sort of alternative treatment in the previous year. Mostly they reported taking supplements; practicing deep breathing exercises; going to a chiropractor or osteopath for spinal manipulation; meditating; or getting massages.

Finding out through well-designed scientific studies whether these treatments work is a valuable service, said neurologist Dr. Steven DeKosky, who sits on the NCCAM advisory council and is dean of the University of Virginia medical school.

"I don't know who else would do that other than NCCAM," he said.

DeKosky headed a $36.5 million study, including $25 million from NCCAM, on ginkgo biloba, a popular supplement taken as a defense against dementia and Alzheimer's disease. DeKosky's study concluded that it did not lower the overall incidence rate of either condition in elderly people who were normal or already had mild cognitive impairment.

The Tribune found that when studying dietary supplements like ginkgo biloba, NCCAM evaluates the results in a way that is accepted within the medical research community.

It's well known that people who receive any treatment, even if it is inert or useless, are likely to report that it makes them feel better. Because scientists don't want to mistake that boost for a real treatment effect, well-designed clinical trials give some volunteers the real therapy and some a fake version of it, then compare the two groups.

NCCAM considers studies finding that a supplement does no better than a placebo to be evidence that it does not work. In an interview with the Tribune, NCCAM director Briggs cited a recent study of the fruit extract of the saw palmetto plant, which found that men who had difficulty urinating because of enlarged prostates reported relief from both the saw palmetto and the fake supplement.

Briggs described the study as one that came back "very convincingly negative" because "it did not demonstrate any benefit over placebo."

But when looking into "mind and body" medicine, NCCAM often argues that a treatment is valuable if patients report that it helped them, even if others receiving a sham treatment said the same. According to Briggs, "most in the mind and body area have actually shown impact."

For example, the center has spent millions of dollars on studies of acupuncture, in which tiny needles are inserted shallowly into the body. NCCAM's website states that the "vital energy" called qi "can be unblocked, according to (traditional Chinese medicine), by using acupuncture at certain points on the body."

Many studies, including those funded by NCCAM, find that true acupuncture performs no better than when a person is fooled into thinking he is getting acupuncture through the use of placebos like retractable needles or even toothpicks twirled on the skin.

People often report feeling less pain or less fatigued regardless of whether they receive real or fake acupuncture, suggesting a placebo effect at work. "I totally agree, in the broadest sense, that it is an effect of context and expectation and hope on pain," Briggs said.

And yet, instead of declaring these studies convincingly negative, NCCAM is pouring more research money into acupuncture.

"The intellectual dishonesty is just astounding," said Dr. Steven Novella, a neurologist at Yale School of Medicine and a critic of NCCAM. "They are just quietly changing the question and the rules."

Acupuncture researcher Dr. Brian Berman, principal investigator for $24 million in NCCAM grants since 1999, wrote in a paper published in the New England Journal of Medicine that a 2008 analysis of acupuncture studies involving 6,000 patients with lower back pain found no significant difference between true acupuncture and sham acupuncture, though both did better than usual care.

Berman, founder of the Center for Integrative Medicine at the University of Maryland School of Medicine, recommended in the paper that a hypothetical person with lower back pain who had not responded to standard medical treatments should receive 10 to 12 acupuncture treatments in addition to usual care. That series of sessions could cost hundreds of dollars.

In an email, Berman wrote that he based his recommendation on evidence that acupuncture is relatively safe and helps people. It's unclear, he said, why true acupuncture and sham acupuncture may produce similar effects.

Both Berman and Briggs said it is difficult to design trials of complex therapies like acupuncture.

"It is generally impossible to isolate a single element," Briggs wrote in an email. "A sham control in a mind-and-body study could easily miss answering the most important question of whether the patient experiences benefit (e.g., relief of pain) from the procedure as a whole."

Physicians who work with patients in pain say they welcome any new tools, especially ones without side effects of narcotics. If that means offering a treatment that may be a placebo, so be it.

"We have lots of people out there with a problem that isn't being addressed with conventional approaches," said Seattle researcher Dr. Daniel Cherkin, who also sits on the NCCAM advisory council. "What do we do with those people? To say we shouldn't do these things because it is a placebo denies them something safe and available and works. What do we replace that with?"

Critics respond that it's not right to charge people hundreds or thousands of dollars for treatments that amount to nothing more than an elaborate placebo without telling them so.

"There is another side of the coin. It is essentially deluding somebody," said Sampson of Stanford. "In other parts of our social life, it is a crime."

Coffee vs. cancer

In fiscal years 2002 and 2003, NCCAM helped fund a study with the National Cancer Institute of an arduous regimen for pancreatic cancer that is best known for frequent "detoxifying" coffee enemas. The study was troubled from beginning to end.

The research design pitted standard chemotherapy against a regimen developed by Dr. Nicholas Gonzalez, a New York City physician. In the study, volunteers on the Gonzalez protocol were to take dozens of supplements each day, including between 69 and 81 capsules of pancreatic enzymes; undergo twice-daily coffee enemas; maintain a strict diet; and engage in other "detoxifying" activities like "skin brushing."

There was little scientific evidence to suggest all of this would work other than a paper Gonzalez published in 1999 on a pilot study of 11 pancreatic cancer patients. Five were reported to have lived at least two years, a long time for pancreatic cancer, which usually kills swiftly.

The hypothesis behind his treatment — that pancreatic enzymes are the body's primary defense against cancer and can be used to fight it — is based on an unproven idea from the early 1900s.

"We have learned a lot since 1906 about cell transformation and how these cells change," said Dr. Mary Mulcahy, a gastroenterology medical oncologist at Northwestern University Feinberg School of Medicine.

Meanwhile, one of the protocol's components — coffee enemas — has been linked to infections and electrolyte imbalances that can be fatal.

Despite the risks and the lack of evidence that the regimen would help patients, the taxpayer-funded study enrolled 55 volunteers with pancreatic cancer. As the project continued, the U.S. Food and Drug Administration and the federal Office of Human Research Protections identified problems, including issues with the subjects' consent.

Researchers published their dramatic results in 2010 in the Journal of Clinical Oncology. Patients receiving standard chemotherapy had lived an average of 14 months. The Gonzalez patients lived an average of four months, and were in significantly more pain.

But some experts questioned the study's findings, saying it lacked a clear question and had a flawed design. For example, the volunteers were allowed to pick whether they received chemotherapy or the other regimen. Originally, they were to be randomly assigned to a group, but few patients were willing to volunteer under those conditions.

Gonzalez is a critic of the project, calling it a disaster. Gonzalez, who participated in the study but didn't run it, argues that the patients in his group were sicker than those receiving chemotherapy.

"It was a waste of taxpayers' money and 10 years of our lives," Gonzalez said. "It served no one and nothing."

Dr. John Chabot, professor of clinical surgery at Columbia University, led the study and said it was worthwhile. "Identifying treatments that don't work remains valuable," he said.

As for Gonzalez's criticism, Chabot said: "Dr. Gonzalez was an active participant and strong advocate of the study until the data started to come through and begin to direct us toward a conclusion."

NCCAM's Briggs declined to talk about the study, calling it "even more ancient history" and a study that would have little chance at receiving funding from her center today. "I think our advisory council would have lots of concerns," she said. NCCAM's website contains barely any mention of it beyond a link to the paper and links to the National Cancer Institute website, which provides more information.

Today, patients continue to stream in to see Gonzalez about his cancer treatment. In the end, the study changed few minds and put volunteers at risk for little benefit to them or to the greater good — at a cost to taxpayers of $1.4 million, with $406,000 coming from NCCAM.

Critics of NCCAM say the project demonstrates how difficult it can be to study complementary and alternative medicine, and that precious research dollars could be better spent elsewhere.

"We have to be good stewards of public money for science," said Gorski, the cancer researcher. "I don't view NCCAM as being a good steward of our public money at the moment. Even if they are doing rigorous science, they are still looking at incredibly implausible things."

ttsouderos@tribune.com

Twitter @chicagoscience

Copyright © 2014, Los Angeles Times
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