The Latest on Alzheimer’s Disease Treatment and Vaccine Development

The number of adults in the U.S. living with Alzheimer’s disease is expected to reach 14 million over the next several decades. And with that, there’s a growing unmet need for effective treatment options. 

In June 2021, the FDA approved Aduhelm (aducanumab-avwa). It was the first new treatment for Alzheimer’s disease since 2003. It was also the first to target the underlying disease process, rather than temporarily manage symptoms. Many other similar treatments were studied before Aduhelm’s approval. But, most failed to show a benefit.

The good news is that there are now over 100 new Alzheimer’s therapies in clinical trials. And the first Alzheimer’s nasal spray vaccine is also being studied. Several medications are in phase 3 clinical trials. This is the last step before a company can apply for FDA approval. 

Let’s review the current progress that’s being made for Alzheimer’s disease treatment.

What clinical trials are underway for new Alzheimer’s disease treatment?

Many new medications are being studied for Alzheimer’s disease that target the underlying disease process. This includes lowering inflammation and/or removing beta-amyloid plaques (clumps of protein) in the brain. Beta-amyloid plaques and inflammation are thought to worsen Alzheimer’s disease progression.

Several of these medications are currently in phase 3 trials. The ones we’ll be covering can be broken down into two groups: biologics and small molecules.

Biologics

Biologics are medications made from living organisms or their components. That makes them harder to make than conventional medications. They’re typically given as injections, or as intravenous (IV) infusions into the vein.

There are currently at least four biologics being studied in phase 3 trials. Similar to Aduhelm, they work by targeting beta-amyloid plaques in the brain. 

• Donanemab is given as an IV Infusion every 4 weeks. It’s currently in a phase 3 trial for early Alzheimer’s disease that’s expected to be completed in 2023. But the manufacturer has already started submitting an application for FDA approval. That means the medication could be approved as early as next year. Donanemab is also being studied for Alzheimer’s disease prevention.

• Gantenerumab is the only medication of its kind in phase 3 trials that’s given as an injection under the skin. This could potentially make it an option that you can administer yourself at home. A phase 3 trial is currently recruiting participants with early Alzheimer’s disease. Initial results are expected mid-2022, and the study is expected to be completed by late 2023.

• Lecanemub is given as an IV infusion every 2 weeks. A phase 3 trial is studying the medication in people with early Alzheimer’s disease. The trial is expected to be completed in 2024. 

• Solanezumab previously made it to phase 3 trials. But it failed to show a significant benefit in people with mild Alzheimer’s disease. It’s now being studied for preventing memory loss in people without memory-related symptoms. Solanezumab is administered as an IV infusion every 4 weeks. This study is expected to be completed by mid-2023. It’s also being studied in another trial with gantenerumab.

Small molecules

Compared to biologics, small molecule drugs are made from chemicals. This makes them easier to make than biologics. Most medications are considered to be small molecule drugs.

There are several small molecule drugs in phase 3 trials for Alzheimer’s disease. Unlike the biologics being studied, these medications are given by mouth. They all work in different ways to target the underlying disease process. Below are a few examples:

• Blarcamesine (Anavex 2-73) activates a receptor (chemical binding site) in the brain that’s involved in several functions. These include removing dead cells and damaged proteins that can worsen Alzheimer’s disease. A phase 2/3 trial is studying the medication in people with early Alzheimer’s disease. Initial results are expected mid-2022.

• NE3107 works by targeting inflammation in the brain. A phase 3 trial is recruiting participants with mild to moderate Alzheimer’s disease. Initial results are expected by the end of 2022. 

• Oligomannate (GV-791) targets inflammation by altering bacteria in your gut. In 2019, the medication was conditionally approved for people with mild to moderate Alzheimer’s disease in China. The following year, the FDA cleared a phase 3 clinical trial that’s currently recruiting participants. The trial will study the medication in people with mild to moderate Alzheimer’s disease. It’s expected to finish in 2024. And if everything goes as planned, the manufacturer plans to apply for FDA approval by 2025.

Is there a vaccine for Alzheimer’s disease?

You may be surprised to learn that vaccines can be developed to prevent or treat certain conditions beyond an infection. Researchers have potentially found a way to apply this concept to Alzheimer’s disease. In fact, there are a few vaccines being tested in people. This includes both injectable and nasal spray vaccines.

Injectable vaccines

In 2019, United Neuroscience announced positive results from a phase 2 trial. Their vaccine works by triggering the immune system to remove beta-amyloid plaques in the brain. The vaccine is expected to enter phase 3 trials soon.

In June 2021, Axon Neuroscience published positive results from a phase 2 trial. Their vaccine works by targeting abnormal proteins that are thought to contribute to Alzheimer’s disease. Larger trials are needed to confirm a clinical benefit.

Nasal spray vaccines

In November 2021, Brigham and Women’s Hospital announced the launch of the first human clinical trial for a nasal Alzheimer’s disease vaccine. The vaccine contains an ingredient called Protollin. Protollin is thought to stimulate the immune system to help clear beta-amyloid plaques from the brain.  

The small trial will be testing the vaccine in people with early symptomatic Alzheimer’s disease. The vaccine will be given as two doses, one week apart. 

If everything goes as planned, researchers think the vaccine could not only be used to treat Alzheimer’s disease, but prevent it in people who are at high risk of developing it.

When will new Alzheimer’s disease treatments and vaccines be available?

The medications and vaccines being studied for Alzheimer’s disease are at various points in the clinical trials process. And they need to show they’re safe and effective before applying for FDA approval. But, there are a few ways they can become available faster.

Study medications that fill an unmet need for serious conditions can be granted different designations by the FDA. For example, Aduhelm was approved through the Accelerated Approval Program. Eli Lilly, donanemab’s manufacturer, is applying for the same pathway.

Accelerated approval allows medications to be approved before showing a clinical benefit. Instead, they’re approved based on different markers (e.g., less plaques in the brain) used to predict a clinical benefit. Effectiveness is confirmed after the medication is on the market.

Another common designation granted to Alzheimer’s medications is Breakthrough Therapy. This means the FDA is more hands-on during the trials process to make it as efficient as possible. Several medications we’ve covered have been granted Breakthrough Therapy designation.

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Written by:

Alyssa Billingsley, PharmD

Alyssa Billingsley, PharmD, is the director of pharmacy content for GoodRx. She has over a decade of experience as a pharmacist and has worked in clinical, academic, and administrative roles.

Reviewed by:

Joshua Murdock, PharmD, BCBBS

Joshua Murdock, PharmD, BCBBS, is a licensed pharmacist in Arizona, Colorado, and Rhode Island. He has worked in the pharmacy industry for more than 10 years and currently serves as a pharmacy editor for GoodRx.

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