An orphan drug that has already passed FDA safety tests turns out to reverse (yes, reverse!) Alzheimer's - in mice. Human trials will start soon and should produce results within the year.
Scott Turner, director of the Georgetown University Medical Center's Memory Disorders Program, who was not involved in the research, welcomed the findings.
"This looks very exciting," he said. "This is a brand new way to move forward in human trials of Alzheimer's disease and it works great with mice."
Turner, a neurologist and leading expert in Alzheimer's disease, however cautioned that more study was needed to see if the same results can be seen in humans.
"One obstacle is that the mice may not be a good model of Alzheimer's disease. We have so many things that work in mice and we try them in humans and they just completely fail," he said.
Trials should begin in the next month or so, Landreth said.
"Perhaps the most important thing is to ask the question: Does this drug work in human beings as it does in mice? Does it get into the brain? And does it have an effect on amyloid levels and increase ApoE levels?
"We need to do that in normal human beings and see if humans are like mice."
And the drug:
Bexarotene was initially made by US-based Ligand Pharmaceuticals under the brand name Targretin.
It gained orphan drug status in the United States -- approval by the US Food and Drug Administration -- in 1999 as a treatment for cutaneous T-cell lymphoma, a rare cancer of the immune system that manifests in the skin and liver.
The Japanese pharmaceutical giant Eisai bought the worldwide rights for it in 2006. Bexarotene is now available in 26 countries in Europe, North America and South America.
The study abstract is at Science Magazine; the article is available to subscribers.