BWH Researchers Launch First-Ever Human Trial for Alzheimer’s Nasal Vaccine


Brigham and Women’s Hospital researchers will begin treating patients this week as part of a first-of-its-kind human trial for a nasal vaccine to prevent and slow Alzheimer’s disease progression.

The trial is the culmination of 20 years of preclinical research led by Howard L. Weiner, a Harvard Medical School professor of neurology and co-director of the Brigham’s Center for Neurologic Diseases, per a Nov. 16 press release.

The clinical trial involves 16 participants between the ages of 60 and 85 experiencing early, symptomatic Alzheimer’s disease. Over a five- to six-week period, each participant will receive a total of four doses of the nasal vaccine, according to Tanuja Chitnis, an HMS professor of neurology and the trial’s principal investigator.

The first phase of the trial focuses on assessing the safety and tolerability of the vaccine among its participants. The research group anticipates having results by next summer, Chitnis said.


Administered intranasally, the vaccine utilizes Protollin, a drug that stimulates the immune system by activating white blood cells. Protollin clears the brain of clumps of protein pieces called beta-amyloid, which are associated with Alzheimer’s disease.

“It’s working to activate macrophages and microglia — which are normal cells that phagocytose — and help to clear abnormal proteins or materials and we’re activating those cells to clear amyloid-beta from the brains of Alzheimer’s patients,” Chitnis said.

Weiner said that this nasal vaccine is a “totally unique approach” compared with other treatments for Alzheimer’s disease currently in development, such as drugs that intravenously give antibodies or work to prevent clumping by inhibiting the breakdown of amyloid protein.

He added that the nasal vaccine does not appear to have any major clinical side effects.

When used in tandem with available screening methods, the vaccine may help those at high risk for developing Alzheimer’s, according to Weiner.

“You measure people for high blood pressure, and if you treat them, they have less heart attacks and strokes,” Weiner said. “There’s now biomarkers and blood tests that you can do where people who are at risk for Alzheimer’s, and you could treat them with this vaccine and you could prevent them from getting Alzheimer’s.”

Weiner also said that if this clinical trial is met with success, he believes doctors will be able to prescribe it to individuals within the next five or six years.

“It could be really something that’s used widely in the population and all over the world to help slow down or prevent Alzheimer’s,” he said. “It really has the potential to be a major vaccine.”

Chitnis said she believes that this vaccine, following further development and research, may have the potential for preventing and treating other neurodegenerative diseases.

“What we are seeing are a number of neurodegenerative diseases that are due to misfolded proteins, which likely are not being eliminated correctly by the body and especially in the brain,” Chitnis said.

“I think it will be interesting to see if a similar approach could be utilized in diseases like Parkinson’s, ALS, and other tauopathies,” she added.

—Staff writer Ariel H. Kim can be reached at

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