Last updated on August 8th, 2023 at 11:53 am

The pharmaceutical company AC Immune SA has received FDA clearance to proceed with phase 1 and 2 clinical trials for its anti-amyloid beta vaccine for the treatment of Alzheimer’s disease. If the results of the clinical trial are positive, this may become one of the most important treatment options for Alzheimer’s disease.

Even though this new treatment is called a vaccine, it is not a preventative vaccine, it is used for the treatment of extant Alzheimer’s disease in a patient. It is an immunotherapy, much like immunotherapy for cancer, where the vaccine trains the immune system to attack the cancer. In this case, the immune system attacks one of the presumed biochemical causes of Alzheimer’s disease.

This article will discuss the key aspects of Alzheimer’s disease and how this vaccine can help treat it.

What is Alzheimer’s disease?

Before I proceed, it’s important to describe Alzheimer’s disease (AD). It is a chronic neurodegenerative disease that usually starts slowly and worsens over time. It accounts for 60-70% of dementia cases, even though the terms are sometimes used interchangeably. Alzheimer’s disease is a form of dementia.

Amyloid plaques, phosphorylated tau tangles, and neurofibrillary tangles are generally easily visible pathologies that can be observed by microscopic analysis of brain tissue from autopsies of those potentially afflicted by AD. These plaques and tangles seem to affect nerve functioning. Despite these observations, the precise pathophysiology, or development, of the disease is not known.

Since amyloid plaques are often identified in patients with Alzheimer’s disease, a large amount of research is focused on attacking those plaques as a way to reverse the effect on nerves which leads to AD.

The causes of AD are unknown (notice how much we do not know about this disease), although it is speculated that it is mostly genetically related, with a large number of genes that underlie this relationship. Despite the internet tropes, there is no rigorous evidence that aluminum causes AD – I wish this belief went away fast.

And since we have no clear understanding of the etiology and pathophysiology of AD, there are no treatments available today for the disease, though some drugs target the amyloid plaques but have not been shown to change the outcomes of AD.

There are a couple of medications that help manage some of the symptoms of the disease, but they are certainly not cures. There are several drugs at the very earliest stages of development that may hold out hope to treat the underlying disease.

One more thing that needs to be made clear. There are no biological tests for Alzheimer’s disease — usually, you can only find the amyloid plaques and other pathologies in post-mortem autopsies. Unfortunately. in the absence of an autopsy, clinical diagnoses of AD are “possible” or “probable”, based on other findings, such as memory tests and other methods.

The Alzheimer’s disease vaccine

AC Immune SA, a Swiss biopharmaceutical company, announced that it had received US FDA approval for its Investigational New Drug (IND) application, and it was going to begin clinical trials (NCT05462106, where you can find information about the clinical trial and email addresses to contact researchers to be included) in several locations in Spain and the United Kingdom.

The vaccine candidate, ACI-24.060, is an immunotherapy designed to generate antibodies targeting misfolded amyloid-beta, which will prevent accumulation and enhance the removal of amyloid plaques along nerve fibers. This vaccine candidate has the potential to inhibit plaque formation and increase amyloid plaque clearance efficiently, and this may reduce or prevent the progression of Alzheimer’s disease.

Interestingly, the clinical trial will not only test patients with Alzheimer’s disease but also with Down syndrome, a genetic disease that, in later stages, leads to dementia that also appears to be linked to amyloid beta accumulations in the nerve fiber.

Although we are very early in clinical trials (the researchers are just starting to recruit patients), it is exciting to know that vaccine-based immunotherapy may be the key to treating Alzheimer’s disease and Down syndrome.

Phase 1 and 2 clinical trials will not be completed until June 2026, and it will take a couple of years after that before data will be published. You know that I will be watching.

Summary

Once again, we have a new vaccine in development that will have a profound effect on humans by treating an insidious disease that affects families all over the world. Although we are a long way from having it available, it brings hope that scientific and medical research may have found an effective treatment for this disease.