Researchers have launched an innovative medical experiment that’s designed to provide quick answers while meeting the needs of patients, rather than drug companies.

Traditional studies can cost hundreds of millions of dollars, and can take many years. But patients with amyotrophic lateral sclerosis, or Lou Gehrig’s disease don’t have the time to wait. This progressive muscle-wasting disease is usually fatal within a few years.

Scientists in an active online patient community identified a potential treatment and have started to gather data from the participants virtually rather than requiring many in-person doctor’s visits.

How is that possible?

In this case, doctors and patients alike got interested in an extraordinary ALS patient whose symptoms actually got better, which rarely occurs. He’d been taking a dietary supplement called lunasin, “and lo and behold six months later, [his] speech [was] back to normal, swallowing back to normal, doesn’t use his feeding tube, [and he was] significantly stronger as measured by his therapists,” said Richard Bedlack, a neurologist who runs the ALS clinic at Duke University.

Of course, it could just be a coincidence that the man who got better happened to be taking these supplements. To find out, Bedlack teamed up to run a study with Paul Wicks, a neuropsychologist and vice president for innovation at a web-based patient organization called PatientsLikeMe.

The study they came up with dispensed with many of the standard features of research that make it so expensive, time-consuming and often so hard to recruit patients:

• There’s no comparison group taking a placebo; instead the researchers match each patient with three to five people whose disease was on a similar course.
• The researchers could skip safety testing because the supplement is already on the market.
• Most important, they aren’t looking for subtle effects, like slower disease progression. That may be vital for a pharmaceutical company seeking approval for a new drug, but isn’t necessarily what patients want.

“I think what people are really looking for is to regain some function,” Wicks says. “So with limited time resources, limited patients available to take part in studies, perhaps we want to swing for the fences every now and then.”

Chances are the dietary supplement won’t help, but at least people will learn the outcome quickly and won’t waste their time and money if lunasin fails.

“If we find just one patient that has a reversal the size of the initial patient, that in itself is incredible, because these reversals are once in a generation,” Wicks said. He and Bedlack have identified just 24 patients, over many years, whose disease actually reversed course, at least temporarily.

The study recruited 50 volunteers at a record pace for ALS research, Bedlack said. That’s partly because it only requires three doctor visits. Those appointments are frequently challenging for people with this debilitating disease.

Most of the data are gathered virtually — patients post their own weekly assessments in a secure area of the PatientsLikeMe website.

Karen Lorne, a 58-year-old nurse practitioner from Chapel Hill, N.C., had cared for patients with ALS, so she knew about the inexorable nature of the disease. This spring, she noticed she could no longer hold medical instruments securely in her left hand, and her speech started to slur. She was diagnosed with ALS in July.

“I was pretty shocked that we know so little and that we have no idea how to fix it — because that’s what we do in medicine,” she says.

After considering her options, Lorne decided to sign up for the low-hassle trial with the supplement. She reports her own symptoms once a week, by typing them in on the PatientsLikeMe website. She can track her own progress, as well as that of the other patients in the study.

And it also serves as a support group. “You can type in, ‘I’m having a bad day,’ and somebody will give you a list of pointers to help you keep in the center, which is where you really want to live,” she said.

And the study doesn’t consume her precious days. She still can focus on her family and life’s daily pleasures.

“We try to live in the present and enjoy every moment as thoroughly as possible,” she says. “And actually, some of my friends who do not have the disease, seeing me having it has helped them shore up their lives and recognize what’s important.”

She is also helping advance knowledge about ALS, even if this trial doesn’t end up helping her.

Patients are a valuable resource, yet only 10 percent ever end up in a study.

“If this infrastructure works, in two years could you imagine 20 trials like this run in parallel?” Wicks asks. “You know, I have a question for the field: Why isn’t every patient in a study?”

One reason that’s not the case is that very ill patients are not likely to recover under any circumstances, so they don’t usually provide a lot of value in scientific studies, says Jeffrey Rothstein, a neurologist who runs ALS studies at the Johns Hopkins University School of Medicine. “That’s why almost no trial is open to all-comers,” as this one was, he says.

He agreed, though, that the study would show whether the supplement has a truly dramatic effect, which is the main purpose of the experiment. “The value of getting patients involved is fantastic,” he says.

If a virtual study like this identifies a promising lead, scientists could quickly pursue that through laboratory studies and more traditional studies with volunteers. Clearly that approach, of starting with animal studies and building on that knowledge, has been a huge disappointment so far with ALS.

And if the approach is successful, it could apply to other diseases as well.

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