Research Feature

Researchers Caution Against Premature ‘Off-Label’ Use

Researchers Caution Against Premature ‘Off-Label’ Use

The number of people living with Alzheimer’s, which stands at more than 5 million today, could hit 16 million by mid-this century, according to experts. “We have to avert this catastrophe,” says William H. Thies, PhD, chief medical and scientific officer for the Alzheimer’s Association, “to alleviate the immense human suffering” and lower skyrocketing costs that “could wreck our health care system and our economy.”

With no medications approved to reverse the mayhem that Alzheimer’s disease unleashes on the brain, early studies that open a window of hope are greeted with a pressing sense of anticipation. And, while “off-label” use—such as the use of a drug approved for one disease for a different condition—can be legal, bexarotene researchers Gary Landreth, PhD, and Paige Cramer, PhD, underscore that much more evidence must be gathered before the skin cancer drug could be ready for repurposing in Alzheimer’s disease.

As research experience has repeatedly borne out, drugs that work in mice often fall short when tested in humans. And even medications that have been deemed safe and effective in people with one condition can affect people with another disease differently, Landreth points out. “This drug has been used in cancer but has never been tested in a diseased brain,” he stresses. “Past history has shown that treatment strategies we think are innocuous can have devastating effects.”

The drug’s existing approval in skin cancer, coupled with the uniquely encouraging effects in animals, promises to compress the development timeline, but meanwhile, it would be jumping the gun to treat people who have Alzheimer’s with bexarotene. “We want to help as many people as we can, as quickly as possible, and for that we have to be incredibly conscientious moving forward,” says Cramer. “A cowboy attitude could jeopardize our research and would be a disservice to patients.”

Landreth and Cramer are dedicating themselves to minimizing the wait time while the drug is studied. Pledges Landreth, “We’ll move this research ahead as fast as is prudent, as we verify whether the drug works in the way we hypothesize.” For the time being, the researchers direct those interested in experimental therapies to the Alzheimer’s Association website,, for information about current clinical studies.