A Cell That Acts Like a Pill: Using iPSC-Derived Cholinergic Neurons to Treat Alzheimer’s Disease

Alzheimer’s disease (AD), the most common subtype of dementia, affects approximately 44 million people worldwide. One of the most major and direct causes of AD, as proposed by the cholinergic hypothesis, is a severe deficiency of cholinergic neurons located in the basal forebrain. This paper proposes a novel approach of using replacement therapy to treat Alzheimer’s disease with stem cell technology. The overarching idea is to inject new cholinergic neurons, which are developed from induced pluripotent stem cells (iPSC), into the brains of AD patients to reverse the cholinergic deficit outlined in the cholinergic hypothesis. The proposed idea is founded on a similar study carried out recently by a team lead by Jeffrey S. Schweitzer that used iPSCs to create dopaminergic neurons which were injected into the brain of a Parkinson’s disease patient and has been proven successful. The proposed treatment development plan would consist of three stages- development of induced pluripotent stem cells and differentiation into basal forebrain acetylcholinergic progenitor cells (APCs); animal studies where researchers inject APCs into the basal forebrain of AD mice models and track progress and improvement in cognitive behavior; and clinical trials involving volunteer AD patients when the procedure of APC development and injection will be repeated, and results will be analyzed. The resulting effects of this treatment can be expected to yield medical, economic, and social benefits.