Emory is investigating the effectiveness of gene therapy in CERE-110, an experimental medication that may help alleviate the symptoms of Alzheimer’s disease.

Alzheimer’s disease causes the degeneration of multiple thought processes.

“We are gaining insight into these processes, but certainly don’t understand them entirely,” said Nicholas Boulis, assistant professor of neurosurgery. “The CERE-110 approach is really an attempt to compensate for this underlying process and protect against it rather than stopping the cause.”

Emory is conducting the research in conjunction with 12 other institutions and universities.

The testing process is about to enter into its second phase. During this phase, scientists will surgically administer injections of CERE-110 into a cluster of neurons that create acetylcholine (ACh), a chemical that is vital to processes necessary for learning and memory.

Boulis said that patients with Alzheimer’s disease have lost a disproportionate amount of the cells that create ACh. The injections of CERE-110 will be able to bolster ACh-producing cells, he said.

Patients will be separated into two groups, those receiving placebos and those receiving the actual drug. A patient from one group will then be paired with a patient in the other group based on the severity level of their Alzheimer’s symptoms.

The patients are not told whether they are receiving the placebo or the drug, and those administering the drugs are unaware of which group each patient was placed in.
This process, Boulis explained, is the best way to test the effectiveness of the drug.

The first phase, Boulis said, was meant to demonstrate that the dosage to be used for each patient in Phase II was safe.

“We hope that phase two will prove efficacy,” Boulis said.

Boulis said the patients to be selected to participate in the second phase will be people who have minor to moderate symptoms of Alzheimer’s.

CERE-110 makes use of a stripped-down virus, called the adeno-associated virus (AAV). The virus itself is unable to replicate without the help of other viruses such as herpes. Because of this inability to replicate, the virus has been deemed safe to use in CERE-110.

AAV can be used to generate nerve growth factor (NGF). NGF, Boulis explained, is a critical element in protecting the survival of ACh-producing cells.

“AVV vectors like the one used in this study have proven safe in extensive animal testing, as well as several other human trials for neurodegenerative diseases,” James Lah, associate professor of neurology and lead investigator, said in a Feb. 1 University news release.

The National Institutes of Health and Ceregene Inc., collaborated to fund this project.
Boulis has worked with Ceregene on other projects that investigated gene therapies for Parkinson’s Disease and Amyotrophic Lateral Sclerosis (ALS), which is more commonly known as Lou Gehrig’s Disease.

Lah is currently a paid consultant for Ceregene.

— Contact Andrew Hull.