Brigham and Women's Hospital will test the safety and efficacy of a nasal vaccine aimed at preventing and slowing Alzheimer's disease, the Boston hospital announced Tuesday. The start of the small, Phase I clinical trial comes after nearly 20 years of research led by Howard L. Weiner, MD, co-director of the Ann Romney Center for Neurologic Diseases at the hospital.
The trial will include 16 participants between the ages of 60 and 85, all with early symptomatic Alzheimer's but otherwise generally healthy. They will receive two doses of the vaccine one week apart, the hospital said in a press release. The participants will enroll from the Ann Romney Center.
A Phase I clinical trial is designed to establish the safety and dosage for a potential new medication. If it goes well, a much larger trial would be needed to test its effectiveness.
The vaccine uses a substance called Protollin, which stimulates the immune system. "Protollin is designed to activate white blood cells found in the lymph nodes on the sides and back of the neck to migrate to the brain and trigger clearance of beta amyloid plaques — one of the hallmarks of AD [Alzheimer's disease]," the hospital explains. It notes that Protollin has been found to be safe in other vaccines.
"The launch of the first human trial of a nasal vaccine for Alzheimer's is a remarkable milestone," said Weiner in the hospital's press release. "Over the last two decades, we've amassed preclinical evidence suggesting the potential of this nasal vaccine for AD. If clinical trials in humans show that the vaccine is safe and effective, this could represent a nontoxic treatment for people with Alzheimer's, and it could also be given early to help prevent Alzheimer's in people at risk."
The researchers say they aim to "determine the safety and tolerability of the nasal vaccine" in the trial and observe how Protollin affects participants' immune response, including how it affects their white blood cells.
"The immune system plays a very important role in all neurologic diseases," Weiner added. "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."
"Research in this area has paved the way for us to pursue a whole new avenue for potentially treating not only AD, but also other neurodegenerative diseases," said Tanuja Chitnis, MD, professor of neurology at Brigham and Women's Hospital and principal investigator of the trial.
I-Mab Biopharma and Jiangsu Nhwa Pharmaceutical are responsible for developing, manufacturing and commercializing Protollin.
Medical researchers around the world have been working for years to develop new drugs to treat or slow Alzheimer's, the most common cause of dementia, which affects an estimated 6 million Americans. But many of the experimental medications that once seemed promising in early studies have not panned out, leaving patients and their families with few good treatment options.
In June, the FDAthe first new drug for Alzheimer's in 20 years, Biogen's . However, the decision sparked controversy following warnings from independent advisers that the much-debated drug had not been shown to help slow the brain-destroying disease and carries the risk of serious side effects.
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