Japanese team starts final-stage trial for iPS drug to treat Alzheimer's

A Japanese research team has started a final-stage clinical trial to treat familial Alzheimer's disease patients with a drug discovered using induced pluripotent stem (iPS) cells.

This is the first final-stage trial conducted in the field of iPS drug discovery, which uses iPS cells to discover new efficacies of new and existing drugs, the team said Tuesday.

The team includes members of Kyoto University's Center for iPS Cell Research and Application (CiRA) and Towa Pharmaceutical, which makes and sells generic drugs.

Through the trial, which began in May, the team aims to confirm the safety and efficacy of the treatment, with hopes of obtaining regulatory approval.

In 2017, CiRA professor Haruhisa Inoue and others carried out an investigation to find a compound that reduces amyloid beta, which causes Alzheimer's disease, by using iPS cells.

Their team found that bromocriptine, an existing drug used to treat Parkinson's disease, is effective in treating familial Alzheimer's disease patients with a specific genetic mutation.

The team conducted a clinical trial on eight patients from 2020 to 2022 and found that the patients did not display adverse reactions and that the deterioration of their cognitive functions as well as their behavioral and psychological symptoms tended to be suppressed.

So the team decided to increase the number of subjects to confirm the treatment's efficacy and safety through the final-stage trial.

The final stage will be conducted at medical institutions nationwide, including Mie University Hospital, until March 2028. The team hopes to have 24 patients participate.

"By using an existing drug, we'll be able to deliver a treatment to patients faster than if we have to develop a new drug," Inoue told a news conference.

Patients will be given an existing generic drug developed by Towa.

"As we're a manufacturer of generic drugs, it is our duty to utilize the many existing drugs," Towa President Itsuro Yoshida said. "We hope to provide treatment options for rare and special diseases."