Clinical Trial of Gene Therapy for Alzheimer’s Prevention Set to Begin

According to a publication from Impact Lab, clinical testing for a new kind of Alzheimer’s treatment is slated to begin at Weill Cornell Medicine (Cornell University’s medical school) in Manhattan this May.

About Alzheimer’s Disease

Alzheimer’s disease (AD) is a progressive disease characterized by the wasting and death of brain cells. Nobody knows what for sure causes Alzheimer’s, but the prevailing theory is that it’s caused by a combination of genetic, lifestyle, and environmental factors.

It’s the most common cause of dementia, a more loosely-defined condition that is defined by the type and severity of the symptoms experienced. Symptoms include declines in memory, thinking, and social skills.

Eventually, Alzheimer’s progresses to its most serious stages when the affected might not remember simple things like the names of objects or loved ones.

Scientists have been trying to pinpoint the cause of Alzheimer’s for years in order to hopefully, one day, develop an effective treatment.

Using Gene Therapy to Fight Alzheimer’s and Dementia

The Weill Cornell trial, to be helmed by the institution’s Dr. Ronald Crystal, represents a departure from the recent hopes of determining the cause of Alzheimer’s in favor of finding a way to prevent the condition from developing in already healthy individuals.

One of the key genetic links to Alzheimer’s scientists are aware of is the APOE gene. This gene, found on the 19th chromosome in humans, has three common types (types 2, 3, and 4) that vary in their relationship to Alzheimer’s. APOE type 2 is associated with a drastically reduced risk of Alzheimer’s, 3 is “normal,” and type 4 is associated with a radically increased chance of Alzheimer’s. In fact, the correlations can be so strong that doctors typically avoid testing for APOE to avoid upsetting their patients – since nothing can currently be done to change their genetic lot.

The function of the gene isn’t even fully understood just yet. Nonetheless, it remains one of the most most accurate ways of predicting the future development of Alzheimer’s in an individual.

Dr. Crystal and his team hope to change up the script by offering the first clinical trial for gene therapy in Alzheimer’s patients this May. The trial will involve “flooding” the brains of the most at-risk individuals with an injection of the hyper-resilient APOE type 2 gene. The gene will be introduced through billions of otherwise inert viruses that excel at infiltrating the incredibly selective blood-brain barrier that controls access to the brain from the blood stream.

The logic of the trial is based on the fact that some individuals inherit both a type 2 and type 4 copy of the APOE gene. These people experience Alzheimer’s at roughly the same rate as people with the type 3 APOE gene, suggesting that certain types of APOE can “cancel out.”

The trial could be a long shot, according to Professor Kiran Musunuru with the University of Pennsylvania’s medical school. The severity of the condition, however, begs for new trials and innovative thinking.

Dr. Crystal hopes that one day, even the most at-risk for Alzheimer’s might be able to lower their chances of developing the condition by “tuning up” their genes. The research, however, is still in its early infancy – the first step in the trial will be to determine if the added gene is functioning at a detectable level. But the high hopes of the team remain clear: that one day we might be able to massage our genes into better shape.


The technology relied upon for this trial is relatively new itself. How might returning to the search for a genetic link to Alzheimer’s, paired with this new technology, yield results we haven’t seen before? Share your thoughts with Patient Worthy!

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