A genetic fingerprint behind the creation of brain-destroying amyloid plaques in Alzheimer’s patients is leading researchers on a path to treatment using drugs created to fight HIV infections.

The Alzheimer’s-linked gene, APP, creates genetic variants that give rise to the sticky plaques that damage brain cells.

Now a team from the Sanford Burnham Prebys Medical Discovery Institute has noted that APP wreaks its neural havoc using “reverse transcriptase,” the same type of enzyme in HIV, the virus that causes AIDS.

In turn, this discovery suggests that current, FDA-approved retroviral therapies already used to block HIV might present a new type of treatment for slowing the advance of Alzheimer’s.

Susan Gammon, director of communications at La Jolla, California, based SBP, told Considerable that while these HIV therapies’ effectiveness against Alzheimer’s are not yet proven, they could be prescribed immediately. “Because these are FDA-approved drugs, doctors can prescribe them for off-label use,” Gammon said.

“Meanwhile, we’re looking to set up clinical trials with an organization to evaluate the efficacy of anti-retroviral therapy as a treatment for Alzheimer’s.”

Recognizing the ability of Alzheimer’s-causing APP genes to create a wild variety of gene variations in the brain’s neurons is key to understanding the challenge of curing the disease.

“These findings may fundamentally change how we understand the brain and Alzheimer’s disease.”
Jerold Chun
Sanford Burnham Prebys Medical Discovery Institute

“These findings may fundamentally change how we understand the brain and Alzheimer’s disease,” said senior researcher Jerold Chun in a statement.

“If we imagine DNA as a language that each cell uses to ‘speak,’ we found that in neurons, just a single word may produce many thousands of new, previously unrecognized words. This is a bit like a secret code embedded within our normal language that is decoded by gene recombination. The secret code is being used in healthy brains but also appears to be disrupted in Alzheimer’s disease.”

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