Towa Pharmaceutical Co. and Kyoto University's Center for iPS Cell Research and Application have launched the final phase of a groundbreaking clinical trial testing bromocriptine, an existing Parkinson's disease medication, as a potential treatment for familial Alzheimer's disease. The trial, which began in May 2025, represents an innovative application of induced pluripotent stem cell technology in drug discovery.
Novel iPS Drug Discovery Approach
The research project utilizes a method called "iPS drug discovery," which employs induced pluripotent stem cells to identify promising treatments for neurological conditions. This approach allows researchers to test existing medications for new therapeutic applications, potentially accelerating the development of treatments for rare diseases.
Bromocriptine, the drug being tested, is currently used to treat Parkinson's disease, high prolactin levels, and type 2 diabetes. The medication works by lowering certain hormones through dopamine-like activity in the brain.
Trial Design and Patient Population
The clinical trial will run until March 2028 and aims to enroll 24 patients with familial Alzheimer's disease who carry mutations of a specific gene. The study follows a randomized, placebo-controlled design, with half of the participants receiving the active drug and the other half receiving a placebo.
The trial is being conducted at several medical institutions, including Mie University Hospital, focusing specifically on familial Alzheimer's disease, also known as early-onset familial Alzheimer's disease. This rare form of the condition typically affects individuals under the age of 60.
Preliminary Safety and Efficacy Signals
According to the research center, preliminary results from the limited patient population have shown promising signs. "Bromocriptine did not show any side effects specific to familial Alzheimer's disease," the center reported on Tuesday.
Additionally, researchers observed a "tendency for the progression of cognitive function and behavioral/psychological symptoms to be suppressed" in the group receiving the drug compared to the placebo group, suggesting potential therapeutic benefit.
Addressing Unmet Medical Need
Alzheimer's disease is a form of dementia that affects memory and other thinking skills, often making daily activities difficult for patients. While currently available medications can temporarily relieve symptoms, there is still no cure for the disease.
Familial Alzheimer's disease represents a particularly challenging subset of the condition, as it generally affects younger individuals and progresses rapidly. The current trial addresses a significant unmet medical need in this patient population, where treatment options remain limited to symptomatic management.
