Clinical trials are driven by hope

"It's clear that the placebo effect is powerful," said Harker, who noted that patients on placebo may experience the known side effects of a drug. They also experience some of the positives. "They have more energy, feel better, more upbeat, hopeful. They're more positive about the treatment, whether or not the drug is helping them."

And there's another good-bad aspect to clinical trials. Patients in clinical trials tend to be more closely monitored. But they often also are subjected to more tests, more restrictions, more time requirements. Some clinical trials pay participants for their time or cover travel expenses, while others don't. In Phase I, where there's no benefit to the participant at all, payment is likely to be higher.

Vance has wrestled with the extra time commitment. But he won't argue with this result: After the fourth treatment, whether placebo or medication, his tumor was half the size. And a lesion on his lung found when he started chemo disappeared after eight weeks. Liver tumors are shrinking.

He battles fatigue but continues to work as a computer specialist with the Utah Valley Emergency Communication Center.

"Things have gone pretty well. I have convinced myself I am getting the drug in the study and I have a positive outlook. But I'm not where I thought I would be this time in my life or ever. It's changed a lot of my perspective on things."

What about after?

After the clinical trial, but before final FDA approval, some investigational drugs may be available to patients through "expanded access" protocols, which may also make the substance available to patients who don't actually qualify for a particular study. It only happens with serious or life-threatening illnesses that have no good alternative treatment. It can also be done to get more information about the drug, especially safety. But clinical investigators must monitor the new treatment in a well-controlled study or the completed study must have shown clear positive results.

What happens after is a concern for Grant, who believes she's received the real drug Ampligen for chronic fatigue. She had the side effects for a day or so after every infusion of the powerful drug, which has antiviral and immunomodulator properties. After about six weeks, those faded. After 10 weeks, "my brain came on," she said.

Participants who were convinced they were getting the medication felt sorry for anyone receiving placebo. But the "carrot" for the study was that at the end, everyone would receive the real drug, which is expensive, for a period of time, after which they could pay for it. Five of the 12 participants Grant knew have continued on, though not everyone can afford it.

Ampligen is on the "fast track" because it's the only drug to demonstrate improvement for those with chronic fatigue. Predictions are it will win the FDA nod quickly.

Still, not all participants will have access to study medication in the time between completion of a study and approval — if approval ever comes.

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E-mail: lois@desnews.com