Drug a hopeful sign for Alzheimer's

An unexpected thing happened to some mice with Alzheimer's disease recently: They were cured, by a team of researchers at Case Western Reserve University. It's impossible to say yet whether the results can be duplicated in treating humans, but we need to find out as quickly as possible. More than 5 million Americans suffer from Alzheimer's, a number expected to double by 2050.

The drug that cured the mice, bexarotene, was approved more than a decade ago for humans, under the name Targretin. Intended to treat skin cancer, bexarotene has few side effects: fatigue, rash and high cholesterol. What's more, the bexarotene didn't merely block the progress of Alzheimer's; it reversed it completely. Cognitive and social skills reemerged, as did an improved sense of smell. Mice that had stared blankly at scattered pieces of tissue paper before receiving treatment began to gather them to make nests, just as healthy mice will.

The amyloid-beta protein on the mice's brains, believed to cause the disease, went away. It stayed away as long as the mice took their medicine and the experiment continued, for three months.

The theory that cancer drugs could help fight Alzheimer's has been around for at least 15 years, according to Dr. Peter Davies, director of the Litwin-Zucker Center at North Shore LIJ's Feinstein Institute. The problem is, no one really knows what it is about the bexarotene that so greatly improved things for the rats.

So now what?

The patents of the maker of the drug, Eisai, begin running out this year, and the company has not said it will do trials on using the drug to treat Alzheimer's, perhaps because it sees no profit in a drug that isn't protected. Scientists who conducted this study have formed a firm to seek patents on the drug for the Alzheimer's use, a tricky business when generic versions will soon be available for skin cancer. And Alzheimer's sufferers are begging doctors to give them bexarotene now, a legal practice known as "off-label use."

No current Alzheimer's drugs work well, or for long. There is already a popular off-label Alzheimer's procedure, intravenous immunoglobin, that many patients use at a cost of $50,000 per year. Like most off-label prescriptions, it is not covered by insurance.

Bexarotene would cost about $14,000 per year now, and far less when generics become available. And it's very difficult to persuade people enduring anything as awful as Alzheimer's to fear side effects.

But even if the drug works on humans, no one knows how much they should take, or how often, or for how long.

Studies should be initiated immediately, and since bexarotene is approved by the Food and Drug Administration to treat skin cancer, they can. President Barack Obama's proposed budget for next year includes an $80-million increase in spending on Alzheimer's research, and these studies and related ones should be launched at universities like Case Western and laboratories such as Feinstein on Long Island.

Patients and families are wondering whether to seek out this drug. Davies says the answer is, "Why not?" He's right. But we need to find a better answer than that, quickly, and there's no reason we can't.