by Isadora B. Stehlin
FDA Consumer

Medical treatments come in many shapes and sizes. There are "home remedies" shared among families and friends. There are prescription medicines, available only from a pharmacist, and only when ordered by a physician. There are over-the-counter drugs that you can buy--almost anywhere--without a doctor's order. Of growing interest and attention in recent years are so-called alternative treatments, not yet approved for sale because they are still undergoing scientific research to see if they really are safe and effective. And, of course, there are those "miracle" products sold through "back-of-the-magazine" ads and TV infomercials.

How can you tell which of these may really help treat your medical condition, and which will only make you worse off--financially, physically, or both?

Many advocates of unproven treatments and cures contend that people have the right to try whatever may offer them hope, even if others believe the remedy is worthless. This argument is especially compelling for people with AIDS or other life-threatening diseases with no known cure.

Clinical Trials

Before gaining Food and Drug Administration marketing approval, new drugs, biologics, and medical devices must be proven safe and effective by controlled clinical trials.

In a clinical trial, results observed in patients getting the treatment are compared with the results in similar patients receiving a different treatment or placebo (inactive) treatment. Preferably, neither patients nor researchers know who is receiving the therapy under study.

To FDA, it doesn't matter whether the product or treatment is labeled alternative or falls under the auspices of mainstream American medical practice. (Mainstream American medicine essentially includes the practices and products the majority of medical doctors in this country follow and use.) It must meet the agency's safety and effectiveness criteria before being allowed on the market.

In addition, just because something is undergoing a clinical trial doesn't mean it works or FDA considers it to be a proven therapy, says Donald Pohl, of FDA's Office of AIDS and Special Health Issues. "You can't jump to that conclusion," he says. A trial can fail to prove that the product is effective, he explains. And that's not just true for alternative products. Even when the major drug companies sponsor clinical trials for mainstream products, only a small fraction are proven safe and effective.

Many people with serious illnesses are unable to find a cure, or even temporary relief, from the available mainstream treatments that have been rigorously studied and proven safe and effective. For many conditions, such as arthritis or even cancer, what's effective for one patient may not help another.

Real Alternatives

"It is best not to abandon conventional therapy when there is a known response [in the effectiveness of that therapy]," says Joseph Jacobs, M.D., former director of the National Institutes of Health's Office of Alternative Medicine, which was established in October 1992. As an example he cites childhood leukemia, which has an 80 percent cure rate with conventional therapy.

But what if conventional therapy holds little promise?

Many physicians believe it is not unreasonable for someone in the last stages of an incurable cancer to try something unproven. But, for example, if a woman with an early stage of breast cancer wanted to try shark cartilage (an unproven treatment that may inhibit the growth of cancer tumors, currently undergoing clinical trials), those same doctors would probably say, "Don't do it," because there are so many effective conventional treatments.

Jacobs warns that, "If an alternative practitioner does not want to work with a regular doctor, then he's suspect."

Alternative medicine is often described as any medical practice or intervention that:

• lacks sufficient documentation of its safety and effectiveness against specific diseases and conditions
• is not generally taught in U.S. medical schools
• is not generally reimbursable by health insurance providers.

According to a study in the Jan. 28, 1993, New England Journal of Medicine, 1 in 3 patients used alternative therapy in 1990. More than 80 percent of those who use alternative therapies used conventional medicine at the same time, but did not tell their doctors about the alternative treatments. The study's authors concluded this lack of communication between doctors and patients "is not in the best interest of the patients, since the use of unconventional therapy, especially if it is totally unsupervised, may be harmful." The study concluded that medical doctors should ask their patients about any use of unconventional treatment as part of a medical history.

Many doctors are interested in learning more about alternative therapies, according to Brian Berman, M.D., a family practitioner with the University of Maryland School of Medicine in Baltimore. Berman says his own interest began when "I found that I wasn't getting all the results that I would have liked with conventional medicine, especially in patients with chronic diseases.

"What I've found at the University of Maryland is a healthy skepticism among my colleagues, but a real willingness to collaborate. We have a lot of people from different departments who are saying, let's see how we can develop scientifically rigorous studies that are also sensitive to the particular therapies that we're working with."

Anyone who wants to be treated with an alternative therapy should try to do so through participation in a clinical trial. Clinical trials are regulated by FDA and provide safeguards to protect patients, such as monitoring of adverse reactions. In fact, FDA is interested in assisting investigators who want to study alternative therapies under carefully controlled clinical trials.

Some of the alternative therapies currently under study with grants from NIH include:

• acupuncture to treat depression, attention-deficit hyperactivity disorder, osteoarthritis, and postoperative dental pain
• hypnosis for chronic low back pain and accelerated fracture healing
• Ayurvedic herbals for Parkinson's disease. (Ayurvedic medicine is a holistic system based on the belief that herbals, massage, and other stress relievers help the body make its own natural drugs.)
• biofeedback for diabetes, low back pain, and face and mouth pain caused by jaw disorders. (Biofeedback is the conscious control of biological functions such as those of the heart and blood vessels normally controlled involuntarily.)
• electric currents to treat tumors
• imagery for asthma and breast cancer. (With imagery, patients are guided to see themselves in a different physical, emotional or spiritual state. For example, patients might be guided to imagine themselves in a state of vibrant health and the disease organisms as weak and destructible.)

While these alternative therapies are the subject of scientifically valid research, it's important to remember that at this time their safety and effectiveness are still unproven.

Avoiding Fraud

FDA defines health fraud as the promotion, advertisement, distribution, or sale of articles, intended for human or animal use, that are represented as being effective to diagnose, prevent, cure, treat, or mitigate disease (or other conditions), or provide a beneficial effect on health, but which have not been scientifically proven safe and effective for such purposes. Such practices may be deliberately deceptive, or done without adequate knowledge or understanding of the article.

Health fraud costs Americans an estimated $30 billion a year. However, the costs are not just economic, according to John Renner, M.D., a Kansas City-based champion of quality health care for the elderly. "The hidden costs--death, disability--are unbelievable," he says.

To combat health fraud, FDA established its National Health Fraud Unit in 1988. The unit works with the National Association of Attorneys General and the Association of Food and Drug Officials to coordinate federal, state and local regulatory actions against specific health frauds.

Regulatory actions may be necessary in many cases because products that have not been shown to be safe and effective pose potential hazards for consumers both directly and indirectly. The agency's priorities for regulatory action depend on the situation; direct risks to health come first.

Unproven products cause direct health hazards when their use results in injuries or adverse reactions. For example, a medical device called the InnerQuest Brain Wave Synchronizer was promoted to alter brain waves and relieve stress. It consisted of an audio cassette and eyeglasses that emitted sounds and flashing lights. It caused epileptic seizures in some users. As a result of a court order requested by FDA, 78 cartons of the devices, valued at $200,000, were seized by U.S. marshals and destroyed in June 1993.

Indirectly harmful products are those that do not themselves cause injury, but may lead people to delay or reject proven remedies, possibly worsening their condition. For example, if cancer patients reject proven drug therapies in favor of unproven ones and the unproven ones turn out not to work, their disease may advance beyond the point where proven therapies can help.

"What you see out there is the promotion of products claiming to cure or prevent AIDS, multiple sclerosis, cancer, and a list of other diseases that goes on and on," says Joel Aronson, director of FDA's Health Fraud Staff, in the agency's Center for Drug Evaluation and Research. For example, he says, several skin cream products promise to prevent transmission of HIV (the virus that causes AIDS) and herpes viruses. They are promoted especially to health-care workers. Many of the creams contain antibacterial ingredients but, "there is no substantiation at all on whether or not [the skin creams] work" against HIV, says Aronson. FDA has warned the manufacturers of these creams to stop the misleading promotions.

People at Risk

Teenagers and the elderly are two prime targets for health fraud promoters.

Teenagers concerned about their appearance and susceptible to peer pressure may fall for such products as fraudulent diet pills, breast developers, and muscle-building pills.

Older Americans may be especially vulnerable to health fraud because approximately 80 percent of them have at least one chronic health problem, according to Renner. Many of these problems, such as arthritis, have no cure and, for some people, no effective treatment. He says their pain and disability lead to despair, making them excellent targets for deception.

Arthritis

Although there is no cure for arthritis, the symptoms may come and go with no explanation. According to the Arthritis Foundation, "You may think a new remedy worked because you took it when your symptoms were going away."

Some commonly touted unproven treatments for arthritis are harmful, according to the foundation, including snake venom and DMSO (or dimethyl sulfoxide), an industrial solvent similar to turpentine. FDA has approved a sterile form of DMSO called Rimso-50, which is administered directly into the bladder for treatment of a rare bladder condition called interstitial cystitis. However, the DMSO sold to arthritis sufferers may contain bacterial toxins. DMSO is readily absorbed through the skin into the bloodstream, and these toxins enter the bloodstream along with it. It can be especially dangerous if used as an enema, as some of its promoters recommend.

Treatments the foundation considers harmless but ineffective include copper bracelets, mineral springs, and spas.

Cancer and AIDS

Cancer treatment is complicated because in some types of cancer there are no symptoms, and in other types symptoms may disappear by themselves, at least temporarily. Use of an unconventional treatment coinciding with remission (lessening of symptoms) could be simply coincidental. There's no way of knowing, without a controlled clinical trial, what effect the treatment had on the outcome. The danger comes when this false security causes patients to forgo approved treatment that has shown real benefit.

Some unapproved cancer treatments not only have no proven benefits, they have actually been proven dangerous. These include Laetrile, which may cause cyanide poisoning and has been found ineffective in clinical trials, and coffee enemas, which, when used excessively, have killed patients. (See "Hope or Hoax? Unproven Cancer Treatments" in the March 1992 FDA Consumer.)

Ozone generators, which produce a toxic form of oxygen gas, have been touted as being able to cure AIDS. To date this is still unproven, and FDA considers ozone to be an unapproved drug and these generators to be unapproved medical devices. At least three deaths have been connected to the use of these generators. Four British citizens were indicted in 1991 for selling fraudulent ozone generators in the United States. Two of the defendants fled to Great Britain, but the other two pleaded guilty and served time in U.S. federal prisons.

The bottom line in deciding whether a certain treatment you've read or heard about might be right for you: Talk to your doctor. And keep in mind the old adage: If it sounds too good to be true, it probably is.

Isadora B. Stehlin is a staff writer for FDA Consumer.