Interventional clinical trial of a therapeutic agent for Alzheimer’s disease

AC Immune and UC San Diego School of Medicine (UCSD)

Principal Investigators: 

  • Dr. Wolgang Barth, PhD
  • Dr. Andrea Pfeiffer, PhD
  • Dr. William Mobley, MD, PhD
  • Dr. Michael Rafii, MD, PhD

Phase 1B Clinical Trial

This landmark study by AC Immune and UCSD represents the first major clinical trial by a pharmaceutical company for Alzheimer’s in the Down syndrome population. The study is focused on developing a vaccine that targets the misfolded βAmyloid protein, in order to decrease plaque accumulation and promote plaque removal. It is believed that this would decrease the risk of Alzheimer’s disease. This represents the first ever private-public partnership in Down syndrome research (National Institutes of Health, LuMind RDS, and AC Immune).

One of the most established underlying factors of Alzheimer’s disease is the misfolding, aggregation, and eventual deposition of a protein known as beta amyloid, which is controlled by the amyloid precursor protein (APP) gene on chromosome 21. The accumulated beta amyloid plaques harm the brain.

The prevalence of Alzheimer’s disease is increasing, due in part to the increased average lifespan. The prevalence is also greater in those who have a higher amount of APP. This is the case in people with Down syndrome, who have over expression of the 21st chromosome. Virtually all adults with Down syndrome develop the neuropathology (amyloid beta plaques and tau tangles) consistent with Alzheimer’s by their 40s.

Clinical dementia in DS estimates:

  • 50% by age 55
  • 70% at age 55 – 60
  • 95% by age 68

Efforts to provide therapies for Alzheimer’s are critical to improve independence and length of life for people with Down syndrome.





Target Life-stage

Phase 1B to test Safety, Tolerability, and Immunogenicity
Estimated completion time is June, 2020
Double-blind, randomized, placebo-controlled, dose escalation