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New Alzheimer’s Drug is Under FDA Review for the First Time in 20 Years

Over 5 million Americans are living with Alzheimer’s disease right now. The Alzheimer’s Association reports that the total number is expected to grow to nearly 14 million by 2050. 

Care costs for this disease and others like it rose to over $300 billion last year and is expected to rise to over $1 trillion by 2050.

Alzheimer’s and diseases like it are a growing problem that could create catastrophic economic and healthcare-related impacts. Medical professionals and researchers work to understand dementia and find better ways to treat it.

Progress is big news, and right now we may be on the verge of a new development. For the first time in two decades, an FDA panel is reviewing a new Alzheimer’s drug.

What Happens When a Patient is Diagnosed with Alzheimer’s Disease?

Alzheimer’s patients usually progress slowly, with gradual worsening over several years. The disease affects memory, judgment, thinking, personality, movement, and problem-solving skills.

There are five stages used to describe the progression of Alzheimer’s disease:

  • Preclinical Alzheimer’s Disease – The disease appears long before the patient notices symptoms. This is considered the preclinical stage. It is usually only discovered in research settings. The patient and those around them notice no changes in ability.

Preclinical Alzheimer’s disease can linger for years or even decades. New imaging technology is capable of revealing deposits of a protein called amyloid-beta, a tell-tale sign of Alzheimer’s. This information is important in clinical trials as researchers look for new treatment options. Patients can also undergo genetic testing to determine if they have an elevated risk due to family history.

  • Mild Cognitive Impairment (MCI) – Patients who are in the MCI stage of Alzheimer’s disease will notice smaller changes in their ability to think and remember. These usually don’t impact relationships. The patient may notice memory lapses when trying to recall information that should be easy to remember, like a recent event.

MCI can be an indication of other medical problems. A doctor should review symptoms to determine if Alzheimer’s is the cause.

  • Mild Dementia – Most patients are diagnosed during the mild dementia stage. Changes are more noticeable at this point. Friends, family, and doctors will likely begin to identify symptoms. The patient will have significant difficulty with thinking and memory, usually to the point that it impacts daily function.

Patients with mild dementia have problems handling complex tasks. Their problem-solving capability is reduced, and they will be more likely to make poor decisions. Their personality may also change with displays of uncharacteristic anger or irritability. The patient will also have reduced motivation and difficulty expressing thoughts. They are more likely to get lost or misplace belongings.

  • Moderate Dementia – The condition will progress at this stage, with the patient experiencing increased forgetfulness and increasingly poor judgment. They become more confused and memory loss is even more noticeable.  Loved ones may notice that the patient repeats stories or makes up stories to fill in memory gaps.

The patient will need increased assistance with daily activities like grooming, bathing, self-care, and using the bathroom. Some patients may lose control of their bowel or bladder. Their personality will further change. They may start to believe that caregivers are stealing from them, that a spouse is cheating on them, or they may see or hear things that aren’t there. Patients become agitated, restless, and more prone to aggressive outbursts.

  • Severe Dementia – Severe dementia describes the late stage of Alzheimer’s disease. Mental function will continue to decline. The patient will lose the ability to communicate. They may occasionally say words or phrases but will mostly be incoherent. They will require daily personal care. 

The patient will also experience a decline in physical ability. They may no longer be able to walk or sit up without support. Their muscles can become rigid and reflexes may respond abnormally. They eventually lose the ability to swallow and control bodily functions.

While dementia is a part of Alzheimer’s disease, it can also indicate other problems. A doctor can review symptoms and provide a diagnosis.

Alzheimer’s progression will vary. The average patient will live with the disease for three to 11 years after diagnosis. Some can survive for over 20 years. 

What Does a New Drug Mean for Alzheimer’s Patients?

The new drug, aducanumab, was developed by the Japanese pharmaceutical company Eisai Co. and Cambridge, Massachusetts-based Biogen Inc.

Aducanumab doesn’t cure the disease, but it can help slow the patient’s rate of decline. The jury is still out on whether the medication is effective or not. Two studies were halted stating that the drug wasn’t working. They then completely changed their view and reported that additional data indicated it was effective at a high dosage. Their results have not yet been published.

An FDA staff report was published that gave the medication a glowing review. While the news is uplifting, an FDA statistician highlighted inconsistencies and flaws in the results as well as potential safety concerns.

The consumer group Public Citizen warned that the drug is expected to be expensive and could “bankrupt our healthcare system” while only providing false hope for patients and their loved ones.

The Status of the FDA Panel’s Decision

FDA evaluations focus on effectiveness and safety. Advocates like the Alzheimer’s Association are pushing for approval, emphasizing the importance of need in the decision.

“There is a dire and drastic need” to assist people impacted “by the crushing realities” of Alzheimer’s, the association said. Others disagree, stating that need has no bearing on safety and effectiveness. 

Aducanumab is believed to clear clumps of the beta-amyloid protein from the brain. Other experimental treatments have been used in the past but made no difference in the patient’s ability to live independently.

The higher cost stems from how Aducanumab is made. It’s created from living cells. While neither company has given any indication of price, other Biogen drugs cost around $3,500 per month with additional fees for each infusion.

If approved, Aducanumab will most likely be covered by Medicare. Medicare and the FDA are not permitted to consider cost when reviewing new medications.

Even the qualification process could be costly. The drug has only been tested on people with mild dementia and mild cognitive impairment. Just verifying a diagnosis requires a $5,000+ brain scan that isn’t covered by Medicare or insurers due to its uncertain benefits. That could change if the treatment proves effective, but for now, nothing is certain.

The FDA used to require two studies that prove effectiveness and safety. That rule has relaxed in recent years. Both Aducanumab studies included around 1,650 participants. The first study showed positive results at high doses but the second was negative. 

The studies changed while underway, which throws the results into further doubt. The placebo group in the positive study worsened more than those in the negative study. That could be why those who took aducanumab looked better in comparison.

The FDA also dismissed safety concerns when as many as one-third of participants experienced brain swelling. 

The Mayo Clinic’s Dr. David Knopman expressed concern, stating that the FDA should require a third study to test aducanumab in ideal conditions. 

Dr. Eliezer Masliah with the U.S. National Institute on Aging also warns that the benefits are “relatively small.” The positive study showed that improvement of the rate of decline was only .39, measured using an 18-point thinking skills score. How much impact that small improvement would have on a patient’s ability to function is unclear.

Dr. Masliah also explained that medications that remove amyloid can have benefits if used early and with other treatments.

The American Academy of Neurology urged the FDA to consider a limited approval to avoid overwhelming the healthcare system and to better manage potential harmful side effects.

Anyone whose life has been affected by Alzheimer’s disease knows just how devastating it can be. The search for better treatments continues. In the meantime, Aducanumab is still under FDA review. A decision is expected by the first quarter of 2021.