What Is Protollin? Nasal Alzheimer's vaccine to Be Tested in Human Trials

Researchers have announced on Tuesday the launch of a clinical trial to investigate the effectiveness of a drug to treat Alzheimer's disease.

The trial, which will take place at the Brigham and Women's Hospital in Boston, Massachusetts, is set to test a drug called Protollin, described as a "nasal vaccine" in a hospital press release.

Protollin is a type of immunotherapy drug, meaning it works by stimulating the body's immune system to help fight certain diseases.

Scientists are hoping that Protollin will be able to activate the body's immune system so that it targets beta amyloid protein plaques.

Beta amyloid protein plaques are essentially a build-up of proteins inside the brain that stop brain cells from working properly. Scientists think these protein build-ups are linked to Alzheimer's and research is ongoing to better understand how.

Protollin is given to patients via a spray that goes up the nose to help it reach the brain and stimulate the necessary immune response, according to I-Mab Biopharma, the company that makes Protollin alongside Jiangsu Nhwa Pharmaceutical.

According to a Brigham and Women's Hospital press release, the phase-1 clinical trial will involve 16 participants aged between 60 and 85 who have early symptomatic Alzheimer's disease and are in good general health. They will each receive two doses of Protollin one week apart.

The main aim of the study will be to see how safe the nasal medicine is. Scientists will also measure the effect the medicine has on patients' immune responses.

The trial, the first in which humans are given a nasal Alzheimer's vaccine, is being funded by I-Mab and Jiangsu Nhwa Pharmaceutical.

"The immune system plays a very important role in all neurologic diseases," said Dr. Howard Weiner in a press release, who has led research into treating Alzheimer's with this nasal medicine for around two decades.

"And it's exciting that after 20 years of preclinical work, we can finally take a key step forward toward clinical translation and conduct this landmark first human trial."

Alzheimer's disease is a brain disorder that causes a person's ability to think and remember things to slowly deteriorate. Eventually, people with the disease lose the ability to carry out simple tasks. It is the sixth leading cause of death in the U.S., according to the National Institute on Aging (NIA).

Alzheimer's disease leaves many features on the brains of people it affects. These features include the aforementioned amyloid plaques, bundled fibers called tau tangles, and loss of connections between neurons. In addition, affected parts of the brain will shrink.

Scientists don't yet fully understand what causes Alzheimer's disease, but significant progress has been made in recent years and causes probably include a combination of genetic, environmental, and lifestyle factors, the NIA notes.

Jigsaw puzzle in shape of head
A stock photo shows a jigsaw puzzle in the shape of a head with some pieces missing. A drug being tested as an Alzheimer's treatment is due to be trialed in patients. ThitareeSarmkasat/Getty