Biotech startup hustles new Alzheimer's program to the clinic

Back in early 2012, a number of physicians around the U.S. began fielding some unusual requests from Alzheimer's patients for a prescription covering Targretin (bexarotene), a cancer drug sold by Eisai. They had heard that investigators at Case Western Reserve School of Medicine in Cleveland found that in mouse models, the drug had cut levels of soluble amyloid in the brain by 25% in a matter of hours. The treatment reversed signs and symptoms of the disease in rodents. And with no effective drug available, a lineup of patients were willing to try it on themselves.

Now Case Western has spun out the research into a new biotech company called ReXceptor, which plans to launch its first human study in a matter of months. Three of the top investigators involved in the animal research, including Gary Landreth, are running the upstart drug company. And they were happy to cite the early attention paid by The Wall Street Journal, CNN and others, which has helped whip up excitement for an unproven approach.

The company has $1.4 million in seed cash from a pair of Alzheimer's foundations as well as two anonymous donors. That money is being budgeted to support the first test in healthy subjects, with an eye to going on to see if the drug works in patients with the disease.

"This is an important proof-of-mechanism study that is a prerequisite for subsequent clinical evaluation of this drug in Alzheimer's patients," Landreth explained in a statement.

Animal studies, of course, are often notoriously unreliable indicators of the way a drug will act in humans. And in this case, there's also considerable uncertainty over what causes Alzheimer's and whether or not patients whose brains have already been damaged can truly benefit from any amyloid beta therapy. On the other hand, drug companies like Johnson & Johnson ($JNJ), Eli Lilly ($LLY), Roche ($RHHBY) and Pfizer ($PFE) have spent billions of dollars trying to find an effective drug that cuts levels of the toxic protein. And they haven't given up yet.

So count one more early-stage Alzheimer's study in the works which will test the amyloid beta hypothesis. No doubt any indication of success will trigger another big round of media attention, where anything positive on Alzheimer's--a terrible disease that afflicts 5.4 million Americans--is typically trumpeted as a breakthrough.

- here's the press release

Special Reports: The Alzheimer's pipeline: What's next? | The Top Phase III Disasters of 2012

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