Vaccines, gene therapy and other novel approaches to future Alzheimer’s treatments | Alzheimer's Prevention Registry

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March 24, 2021

Vaccines, gene therapy and other novel approaches to future Alzheimer’s treatments

By Alzheimer's Prevention Bulletin


Alzheimer’s researchers continue to explore new therapies to prevent and reverse the path of the disease. Researchers have been met with disappointing clinical trials along the way but have held steadfast in their fight to end this memory-stealing disease. Now, scientists are exploring new and novel approaches to treatment and prevention.

“New approaches like gene therapy and new ways of delivering drugs already used for other neurologic diseases may give us a road map for developing new approaches to treat Alzheimer’s disease and other dementias,” said Robert Alexander, MD, Chief Scientific Officer at the Alzheimer’s Prevention Initiative.

These new modalities include vaccines, gene therapy, drugs that effect messenger RNA, and methods to promote protein degradation and removal (PROTACS). Most are not in trials yet but are being studied by Alzheimer’s researchers for their potential. Let’s explore a few of them.

Vaccines – Previous trials have looked at vaccines to prevent Alzheimer’s. They were not successful for a number of reasons, but now there is renewed interest in this method. Anti-amyloid vaccines have shown good immune response and safety in previous trials, and an anti-tau vaccine is also currently being studied.

Gene Therapy - Imagine if a treatment could overcome the presence of the APOE4 gene that increases risk for Alzheimer’s by inserting into the brain the APOE2 gene that is known to protect the brain. It could be used to counteract the expression of APOE4 in the brain and perhaps prevent the development of Alzheimer’s disease.

RNA Drugs – These drugs bind to RNA and can either greatly decrease the amount of a particular protein that a cell produces or modify the protein in a way that limits its potential to cause dementia. For example, it may be possible to deliver a therapy to the brain that would decrease the level of APOE4 by blocking the messenger RNA that codes for this protein. This concept is not in clinical trials yet.

PROTACS – This technology proposes to use small proteins to “hijack” the cells machinery that gets rid of old and damaged proteins to, for example, eliminate tau buildup in the brain.

“In addition, to these exciting new methods of effecting genes and proteins, there are a variety of new treatments under study that can decrease the brain inflammation associated with Alzheimer’s” said Dr. Alexander. Other studies are exploring whether noninvasive brain stimulation, such as using transcranial magnetic stimulation (TMS), or surgical implants, including deep brain stimulation (DBS) are effective treatments for people with mild cognitive impairment or dementia due to Alzheimer’s.

Interested in participating? Check out these studies using novel approaches: