Investigators have turned in intriguing animal data suggesting that an orphan drug used to treat lymphoma may be effective in countering Alzheimer's disease. And with the news grabbing headlines around the world, researchers say they need to race to a more conclusive understanding of how the drug works in humans as physicians field demands for the treatment.
Any animal data can only offer clues to what investigators can find in human studies. In this case, the drug--bexarotene, an Eisai drug sold as Targretin--lowered levels of beta amyloid, a long-suspected culprit behind Alzheimer's, and improved the memories of the rodents in the study.
In most cases, that might inspire years of research and clinical studies to see if the treatment works in humans. There's no real consensus on what causes the disease, which afflicts millions. And companies like J&J ($JNJ) and Eli Lilly ($LLY) are devoting hundreds of millions of dollars to major trials of late-stage products in a race to a new approval that would be worth a megablockbuster return.
In this case, though, the intense interest in finding any new therapy is complicated by the fact that bexarotene is already on the market. And the lead investigator is being queried by physicians who are being asked for a prescription now by patients willing to test it on themselves.
"We've got to work fast, and we have got to be right. We can't screw this up," Gary E. Landreth, a neuroscientist at Case Western Reserve University School of Medicine, tells The Washington Post. "It has to work in humans like it works in mice or we can pick up and go home."
Special Report: Making sense of the Alzheimer's drug pipeline