Is This Experimental Drug A Promising Alzheimer's Treatment?
If you're someone who is at high risk of developing dementia or Alzheimer's disease, it can be pretty emotionally and mentally draining, especially if you feel like there's very little you can do to delay its onset (aside from following a healthy diet and lifestyle, and getting enough sleep). But new research on an experimental drug suggests that it could potentially help lower the odds of developing Alzheimer's in people who are genetically predisposed to the devastating condition. Of course, Alzheimer's is a complex disease, and there's no silver bullet treatment.
The findings, which were published in The Lancet Neurology, are a little complicated and highly specific to a certain group of people. They also raise a lot of questions from experts about the potential impact of this treatment plan in the general population. Here's what you should know, and what doctors think of the drugs.
Meet the experts: Clifford Segil, DO, is a neurologist at Providence Saint John's Health Center in Santa Monica, CA; Amit Sachdev, MD, MS, is the medical director in the Department of Neurology at Michigan State University. Randall J. Bateman, MD, is a study co-author and professor of neurology at Washington University in St. Louis
For the study, researchers recruited 73 people with rare and inherited genetic mutations that cause the overproduction of a protein called amyloid in the brain. In case you're not familiar, amyloid is found, but in the brain it clumps together to form "plaques" between nerve cells. Too much build-up of these amyloid plaques in the brains can lead to issues with brain function and is linked to Alzheimer's, per the National Institute on Aging (NIA). But we'll get to more of that later.
For the study, the researchers gave the study participants an experimental anti-amyloid drug. They found that 22 participants who had no cognitive problems at the study's start and who took the drug the longest (an average of eight years) had a 50 percent lower chance of developing symptoms.
The researchers didn't study the impact of the treatment on people who already have Alzheimer's disease. Instead, they looked at people with a very high likelihood of developing it who were within 10 to 15 years before they were expected to develop the disease based on their family history.
So, the treatment is more geared towards delaying or halting onset rather than curing an existing disease.
Amyloid plaques are areas where amyloid (again, a protein) has built up, explains Amit Sachdev, MD, MS, medical director in the Department of Neurology at Michigan State University.
'You can think of it a lot like a scab on your skin,' he says. 'The scab is rough, it isn't supposed to be there and, if it gets too thick, it causes a problem.'
But there is some debate about whether targeting amyloid will help lower the risk of Alzheimer's disease, says Clifford Segil, DO, a neurologist at Providence Saint John's Health Center in Santa Monica, CA. 'Targeting brain amyloid is not a slam dunk for Alzheimer's prevention as we do not have any tests that are in use clinically to predict Alzheimer's onset,' he says.
'Most neurologists liken [amyloid plaques] to skin freckles, which are normal aging phenomena and extremely infrequent[ly] became a skin cancer,' Dr. Segil continues. 'Many Alzheimer's scientists liken them to unexploded land mines which, if left alone, will cause older people to lose the ability to tie their shoes and all but surely get Alzheimer's disease.'
The treatment uses anti-amyloid antibodies to break down the plaques, Dr. Sachdev explains.
'The anti-amyloid antibodies attach to amyloid and use the immune system to remove it,' he says. 'This is very effective, because the immune system is excellent at getting rid of things that do not belong.'
But Dr. Sachdev also says that the drug relies on inflammation to clear the plaques. (Think of it like this: Inflammation is a natural bodily response to kickstart healing in the body, bringing various cells and biological tools to the site of injury to repair it. The treatment seems to trigger inflammation.) 'More than 50 percent of patients who were exposed in this study had changes to their brain [including potential for swelling and bleeding] that could be related to inflammation,' he says.
As such, these medications come with the risk of brain bleeding, brain swelling, and brain shrinkage, making them risky to use, Dr. Segil points out.
Not yet, says Randall J. Bateman, MD, study co-author and professor of neurology at Washington University in St. Louis. This is partly because the study was quite niche. "Our trial tested this in rare families with mutations that cause Alzheimer's disease," Dr. Bateman says.
This trial originally ended in 2020. Right now, there are ongoing trials that will wrap up in a few years, and if those are successful, the treatment could potentially be made available to the public, per Dr. Bateman.
But some doctors aren't convinced these treatments should be used. One of the medications in the treatment (gantenerumab) is no longer made. 'The company developing it has decided to go into go in a different direction,' Dr. Sachdev says.
Anti-amyloid medications also come with a high risk of side effects, including death—and that's concerning to many doctors and families, Dr. Segil says. 'Like most neurologists in the U.S. today, we are not using this family of medications,' he says. 'Clinical neurologists like me continue to be skeptical that the benefits outweigh the risks of anti-amyloid medications for dementia.'
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