People with ALS Face Tough Decisions About Experimental Medications

by Margaret Wahl on Tue, 2008-04-01 17:00

Deciding whether to take an experimental medication outside or inside a clinical trial can be tough for people with ALS.

With the excitement about lithium as a potential treatment for ALS and the proliferation of clinical trials, people with the disease are facing some tough decisions.

Although everyone hopes for benefit from an experimental medication, trials are conducted to gather broad data, not necessarily to help any individual. Risks of participating in a trial range from being assigned to a placebo (inactive substance) group, to being exposed to a compound that may worsen the disease, as was the case in the recent trial of minocycline.

On the other hand, bypassing a clinical trial to take an approved medication such as lithium, which lacks a track record in ALS, has its own risks, including exposure to a possibly harmful substance and exclusion from most clinical trials. It does, however, eliminate the guess work about placebo assignments and allows for individualized adjustments in dosage and timing of treatment.

Medical sociologists and other trial watchers worry that research subjects may not fully understand the differences between treatment and a clinical trial. Sociologist Gail Henderson at the University of North Carolina says most people are in a “gray zone” on this subject. They know they’re in a research study, but they may think of the research as more like an extension of treatment than it really is. This “therapeutic misconception” can be heightened if the investigator is also the patient’s doctor.

Would have asked more questions

Deb Wehrlin, 48, of South Burlington, Vt., begged her husband, Jeff, to drop out of the minocycline trial in 2005, when it was clear his ALS was worsening rapidly. He died in August of that year, at age 51. (Minocycline was later shown to have a harmful effect in ALS.)

Wehrlin says she wasn’t aware of the risks going into the trial, although Jeff might have been. “We were told it’s an antibiotic that’s been used for acne, and there didn’t seem to be any issues,” Wehrlin says, “but I also know my husband, and he could be very strong-willed, and it’s possible he didn’t share all the information with me.” Jeff, she says, was committed to finishing the trial because of his desire to help others, even when he realized he wasn’t going to survive.

If she had it to do over again, she says, “I think I would do more research on any product that offered hope, and be more careful, ask more questions. We all want a miracle, and who knows what that might be, but I think caution is needed.”

Might as well try it

Tim Kokojan of Euless, Texas, whose ALS was diagnosed in March 2005, isn’t concerned about the risks of taking lithium. He says he’s reassured by its long record in treating bipolar disorder.

Kokojan asked his doctor to prescribe lithium shortly after reading about the Italian trial on the Living with ALS listserv (

“When this came up about lithium, I thought I might as well try it,” Kokojan says. “I’ve noticed progression in the last year. My right arm and both legs are pretty weak now. In the last few months, I’ve noticed a big turn for the worse. I live alone. I’m in the middle of a divorce. I feel like I have nothing to lose.”

Kokojan, who says he’s now “willing to try anything,” says he wouldn’t want to enroll in a clinical trial of lithium if it meant the possibility of a placebo group assignment. “I don’t want to stop taking it [lithium] now that I’m getting the ball rolling,” he says.

Being a little cautious

Laura McWhorter, 37, of Bridgewater, N.J., says she’s not ready to jump on the lithium bandwagon just yet, even though her ALS, diagnosed in 2005, is progressing. “It started out just as a foot drop in the right leg,” she says, “but at this point, I really can’t walk at all. My arms and hands are starting to get weak, and that’s been making a big difference in what I can do for myself.”

McWhorter worked in the pharmaceutical industry, where she designed, monitored and interpreted clinical trials. Now she’s having to decide whether to enroll in a clinical trial herself.

As a professional, she’s skeptical of the lithium study. “It’s important to have replication to make sure something isn’t by chance,” she says. “I’m not really convinced that [the lithium findings] are not by chance. I also think, if it did work, there must have been some patients out there with ALS with bipolar disorder, and you’d think it would have been noticed.” Her search of the medical literature turned up nothing.

However, she says, “As a patient, I hold onto hope. I’m not completely doubting it. I’m watching the ALS space ( As people get out to 90 or 120 days [on lithium], I’ll see how it looks. I check that every week to see how they’re doing, because the flip side is, it’s not a picnic to take lithium. You need a lot of blood tests, you have to watch your diet, and your fluid intake is important. For those reasons I’m being a little cautious.”

Risks, rights, responsibilities

If you decide to enroll in a clinical trial, you’re taking on risks and responsibilities. In the United States, the Food and Drug Administration spells out the legal rights of trial participants by saying they should know what risks they’re facing, as well as what procedures they’ll undergo and for how long.

There are no legal responsibilities for trial participants, but it’s problematic if participants leave a trial for a trivial reason; fail to comply with study procedures or appointments for less than compelling reasons; are dishonest (for instance, about medication use); or take the study compound to a lab to determine whether it’s a placebo.

McWhorter has been in two previous trials. In one of them, she was assigned to what she later learned was creatine plus minocycline.

“There’s risk in doing everything,” she says. For her, the lithium jury is still out. “I would tell [people with ALS] that if they were doing relatively well to wait to join a study, but if they felt like they were getting toward the end of their rope, to try it. That’s probably what I’m going to do.”

Being a Co-adventurer” in the May-June issue of Quest will also address issues surrounding clinical trials.


Margaret Wahl
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