Alzheimer's disease discovery: Cold Spring Harbor research with key protein improves memory in mice with symptoms

A study from Cold Spring Harbor Laboratory on Long Island is showing promise in delaying memory loss associated with Alzheimer’s disease, a form of dementia that’s a leading driver of death in the United States.

The research found that mice with Alzheimer’s disease symptoms showed "improved cognitive behaviors" when the protein PTP1B was suppressed.

In the brain, the growth of amyloid-β, or plaque, is a key factor in the development of Alzheimer’s disease, researchers said. The study found that reducing PTP1B brought down the amyloid-β burden and enhanced cognitive function.

Mice with Alzheimer’s symptoms that were treated with PTP1B showed a 30% to 50% reduction in amyloid burden, according to the study, published last week in the Proceedings of the National Academy of Sciences.

Now, researchers hope the study will lay the groundwork for developing additional Alzheimer's treatments that can slow the disease.

"The goal is to slow Alzheimer’s progression and improve quality of life of the patients," Cold Spring Harbor Laboratory Professor Nicholas Tonks, who is the research’s lead author, said in a news release.

According to the Alzheimer's Association, more than 7 million people age  65 and older in the United States are estimated to have the disease in 2025.

Researchers used standard behavioral tests on mice with Alzheimer's disease symptoms, wild-type mice and Alzheimer's disease model mice treated with the PTP1B inhibitor.

One of the tests used a small pool with a submerged platform. The mice learned to locate the platform when they no longer wanted to swim. When the platform was removed, the wild-type mice searched the area, whereas the Alzheimer's mice were unable to learn or remember its location.

But the Alzheimer's mice treated with the PTP1B inhibiter remembered the platform’s location similarly to wild-type mice, the research showed.

In another test, the treated mice showed curiosity about a new object similar to wild-type mice, whereas the untreated Alzheimer mice didn't recognize it as new.

"So this is why we got so excited about it. It's a clear impact on the learning and memory phenotypes that are disrupted in the Alzheimer's mouse model," he said in a phone interview.

Despite the promising results, Tonks notes it takes years, clinical trials and much more funding before this type of treatment can reach people. 

"So I think what the mission of academia should be is to come up with new, exciting basic science observations, validate new exciting approaches to treatment of the disease, and provide a sufficient validation to engage the pharmaceutical companies, then to come on board and help to set up clinical trials that can realize the potential of what the basic research has done," he said.

Dr. Nikhil Palekar, director of the Stony Brook Center of Excellence for Alzheimer's Disease, said the results presented in the paper are "exciting," but an actual treatment is a long road ahead.

Currently, he said, there are a few medications to reduce amyloid accumulations in people who have early Alzheimer's disease, slowing the progression of the disease by roughly 30% over 18 months.

A successful treatment derived from the lab’s research could be another treatment option, allowing patients to maintain their level of functioning for much longer, he said.

 "It's a progressive illness, and people continue to get worse with time. So if you're able to slow it down, people will be able to continue to work, if they're working, continue to be able to, you know, drive their car, you know, function socially, with their families, friends, travel," he said in a phone interview. 

Palekar noted there were two other issues in the brain related to Alzheimer’s: a protein called tau that destroys brain cells and inflammation. Still, Palekar called the lab’s research "novel."

"Hopefully it will translate also in humans, and we might be able to develop medications based on this finding that will inhibit this protein," he said.

By Tiffany Cusaac-Smith

tiffany.cusaac-smith@newsday.com@T_Cusaac

Tiffany Cusaac-Smith is a general assignment reporter for Newsday. She previously worked at USA TODAY and is an alum of Howard University.