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Biogen, a Cambridge, Massachusetts biotech company, announced last week that early tests of their new drug aducanumab, a monoclonal antibody, had shown impressive results in treating those with early stage Alzheimer’s disease. The drug significantly reduced the amyloid plaque buildup in the brain that is associated with Alzheimer’s.
In a very early stage safety test aducanumab slowed the cognitive decline and dementia associated with Alzheimer’s in people. On the Mini Mental Status Exam, a widely used measure of cognitive function, people at risk of Alzheimer’s who got a placebo lost around 3 points over a year. But those who got the lowest dose of aducanumab worsened by just two points and those who got a higher dose lost less than a point.
Biogen was so excited by the early results in 166 volunteers that it is going to try to go directly to a much bigger clinical trial of the drug. Wall Street was very excited too—Biogen’s stock price shot up 10 percent.
So this is all good news right? Finally, after decades, a next-generation bioengineered drug is showing real success against a dread disease for which nothing much currently exists. In fact, it is good news but success often brings ethical challenges in its wake. Here are five.
How safe must this drug be to release it?
Some subjects experienced inflammation of the brain at the highest doses that manifest as dangerous brain swelling. While lowering the dose seemed to control the problem, patients got better memory results on higher doses.
Getting the right dose figured out is crucial and so is calculating how to dose for patients of different body weight, genetic makeup and stage of disease. That takes time. And there may always be some risk.
Who gets it first?
Will those facing Alzheimer’s be willing to take a drug that might make them worse off—would Biogen be willing to sell it?Assuming some facing Alzheimer’s will jump at any promising drug and won’t want to wait, organizing a big clinical trial puts the company in a bit of a bind as patients or their families will beg the company to get the drug outside a trial.
If the company gives in, the trial will be slowed down as the drug is diverted for compassionate use. That could mean the answers to what is the right dose for patients could be delayed.
I don't know how much drug the company has but I cannot imagine it will have enough to supply a big clinical trial and also give it out to all those requesting aducanumab on a compassionate basis.
The company may just say no to requests but, given the push in many states to open up access to the dying, that may not be so easy to do.If the drug does work, demand, even for a drug that only buys time and is not a cure, will be off the charts not only in the U.S. but around the world.
How much will it cost?
Many people would pay anything to get it. And that means big bills for the payers – not just private insurance but the government programs such as the VA, Medicaid and Medicare.
If it clears the next clinical trial hurdle the cost for aducanumab will likely go through the roof. So will demand. Other promising monoclonal antibody drugs for various cancers and mental illnesses are rumbling through the research pipeline. If cost containment is a key goal for our health care system and those of other nations, success is bringing a whole lot of expensive trouble in its wake.
Will our Congress ever get around to holding hearings on prices to decide what financial incentives are strong enough to get companies to gamble on drugs but not so high as to limit access to the rich and well-insured?
Is living longer better?
If folks live a bit better for longer with Alzheimer’s, that slower decline means more challenges for families, caregivers and long-term care facilities.
While it is great to have grandma a bit more mentally with it for longer, issues about when to move those with the disease to institutional settings will not get any easier. Oddly, slowing mental decline may make it possible for some to stay at home or with families longer but that raises the burden on those families to manage those, usually elderly and frail, with Alzheimer’s. For a nation that hasn’t come close to figuring out how to handle home care, give help to caregivers and to improving institutional care a partially successful drug is a two-edged sword.
Will it make Alzheimer's less scary?
A partially effective drug against Alzheimer’s will tamp down some of the fear of dying of or with the disease – for a while. The Alzheimer’s Association just reported this week that 5.3 million Americans have the disease. The huge numbers of those facing Alzheimer’s in the years to come—one in nine over 65 and one in 3 over 85—is going to fuel a much louder societal debate about how to die with dignity if you lose your mind. A pill could buy some time to work out an answer but we need an answer. So do does the rest of the world.