Karen Lorne, diagnosed with Lou Gehrig’s disease in July, volunteers weekly with her certified therapy dog, Bailey, at the Ronald McDonald House in Chapel Hill, N.C.
Courtesy of Karen Lorne
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 communityidentified 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.