This story was published on on February 8, 2024.

UAMS announced that a team of researchers led by Sue Griffin has discovered a potential new drug to block the harmful effects of the so-called Alzheimer’s gene.

The findings, published Jan. 8 in Communications Biology, include discoveries of a druggable target and a drug candidate made by researcher Meenakshisundaram Balasubramaniam, the paper’s first author. The research is supported by a five-year, $2.4 million grant from the National Institutes of Health National Institute on Aging.

Balasubramaniam, co-principal investigator on the grant with Griffin, said UAMS built the first known full-length structure of the Alzheimer’s gene protein in 2017. The work led to the discovery of a druggable site on the protein.

Most Alzheimer’s research nationally has focused on treatments that can clear away the brain’s plaques and tangles associated with the disease. But people with mild Alzheimer’s symptoms have already lost about half or more of the neurons responsible for memory and reasoning, which led Griffin to focus on prevention. 

An estimated 50%-65% of people with Alzheimer’s disease have inherited the Alzheimer’s gene from one or both parents, UAMS said in a news release about Griffin’s research. About 25% of people have one copy of the gene and are three times as likely to develop the disease.

In a statement, Griffin said the findings on the Alzheimer’s gene protein and its role in the development of the disease are “among the most impactful of my 50 years as a research scientist.” Her research team plans to conduct larger-scale preclinical research on the drug candidate, CBA2, as well as test other potential drug candidates. A provisional patent has been awarded on the CBA2 drug candidate, and full patent approval is pending.

“Our hope is that people who have one or two copies of [the Alzheimer’s gene] will one day take the drug regularly throughout their life and significantly reduce their risk of developing Alzheimer’s disease,” Griffin said.