Brigham and Women’s Hospital has begun a clinical trial that will test the safety and efficacy of a new vaccine delivered nasally intended to prevent and slow the progression of Alzheimer’s disease (AD).
The trial represents the culmination of nearly 20 years of research led by Howard L. Weiner, MD, Co-director of the Ann Romney Center for Neurologic Diseases at the Brigham.
“The launch of the first human trial of a nasal vaccine for Alzheimer’s is a remarkable milestone,” said Weiner. “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 vaccine uses the immune modulator Protollin, an investigational intranasal agent that stimulates the immune system. Protollin is composed of proteins derived from bacteria and has been used safely in humans as an adjuvant for other vaccines. 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. I-Mab Biopharma (I-Mab) and Jiangsu Nhwa Pharmaceutical (NHWA) are responsible for the development, manufacturing and commercialization of Protollin.
The clinical trial is a single ascending dose (SAD) trial of 16 participants, all of whom will be enrolled from the Ann Romney Center. Trial participants will be between 60 and 85 years of age with early, symptomatic AD. Participants must be in good general health with no disease expected to interfere with the study and have had an amyloid-positive PET scan. Participants will receive two doses of the nasal vaccine one week apart.
The phase I trial’s primary objective will be to determine the safety and tolerability of the nasal vaccine. The research team will also measure the effect of nasal Protollin on participants’ immune response, including its effects on white blood cells, by examining cell surface markers, gene profiles, and functional assays.
The trial is funded by I-Mab, based in Shanghai, China, and Jiangsu Nhwa Pharmaceutical Co. (Nhwa). Brigham and Women’s Hospital and Inspirevax (formerly Biodextris) granted I-Mab and Nhwa global exclusive licenses to develop, manufacture, and commercialize Protollin, and Inspirevax will manufacture and supply Protollin for preclinical and clinical studies until the recruitment of the first patient in the Phase 1b Multiple Ascending Dose (MAD) study. I-Mab will develop and commercialize Protollin outside of the Greater China territory, while Nhwa will develop and commercialize the drug in the Greater China territory (i.e., mainland China, Hong Kong, Macau, and Taiwan).