The move, the hospital said in a press release, represents the culmination of 20 years of research at the institution.
“The launch of the first human trial of a nasal vaccine for Alzheimer’s is a remarkable milestone,” Dr. Howard Weiner, co-director of the Ann Romney Center for Neurologic Diseases at Brigham and Women’s Hospital, said in a statement. “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 first human clinical trial for the intranasal vaccine is intended to prevent and slow the progression of the disease.
In an interview with Fox News, Weiner discussed the mechanism of Alzheimer’s disease, pointing out that people in their 50s and 60s with no symptoms of Alzheimer’s are developing it.
“Understanding the mechanism of Alzheimer’s … you have these toxic substances in the brain: amyloid, tau, other things like that. Also, as we get older, and the immune system usually acts to fight these things off. As we get older, the immune system isn’t as strong in doing that. And through a whole series of experiments, we discovered that we could give a nasal vaccine – which is actually a type of bacteria that’s used in other vaccines – and it stimulates the immune system and then the cells go into the brain and fight against the disease,” he explained. “So, we’ve been working on this [for] a long time. A lot of challenges – understanding the mechanism, how to manufacture it, working with the FDA – and we finally made it.”
The vaccine uses the immune modulator Protollin, an investigational intranasal agent that stimulates the immune system.
Protollin is made up of proteins derived from bacteria and has been used safely in humans. It 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, which the hospital noted is one of the hallmarks of Alzheimer’s disease.
The study is funded by I-Mab Biopharma (I-Mab) and Jiangsu Nhwa Pharmaceutical (NHWA), which are responsible for the development, manufacturing and commercialization of Protollin.
The trial will study 16 people between the ages of 60 and 85 with early, symptomatic Alzheimer’s, and all participants will be enrolled from the Ann Romney Center.
The patients must have had an amyloid-positive PET scan and be in good general health.
“We’ll be doing blood testing to see which dose stimulates the immune system the best,” Weiner said. “This will last about six months, after which there’ll be a trial of 150 patients that will be treated for years. So, right now, we’re just getting two doses, two a week apart. And then, the next trial when we treat 150 patients, they’ll be treated for a year probably getting it once a month … That’s what we’re aiming toward.”
The Phase I trial’s objective will be to determine the safety and tolerability of the nasal vaccine, in addition to measuring the effect of nasal Protollin on participants’ immune response, including its effects on white blood cells.
“The immune system plays a very important role in all neurologic diseases,” wrote Weiner. “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.”