The Yomiuri Shimbun
Clinical trial using iPS cells litmus test for promoting regenerative medicine
iPS細胞移植 再生医療普及への試金石だ

The latest clinical trial is the first step of epoch-making research that could evolve into regenerative medicine. After careful safety assessment, putting the latest result into practical use should be promoted.

A team led by RIKEN ophthalmologist Masayo Takahashi transplanted retina cells produced from induced pluripotent stem (iPS) cells into the eyes of a woman suffering from age-related macular degeneration. The patient is said to be doing well.

It was the world’s first clinical trial of transplanting iPS cells, which can develop into the cells of various types of tissues, into a human body.

This clinical trial can be considered a litmus test for regenerative medicine using iPS cells, which could eventually be used widely.

About 700,000 people are estimated to be suffering from the age-related macular degeneration, the symptoms of which include distorted vision.

The current treatment is symptomatic therapy to prevent the patient’s condition from further deteriorating. With the transplant of retina cells, the patient could make a full recovery. Expectations among patients over the effectiveness of such a transplant are believed to be running high.

Over the past seven years, since Prof. Shinya Yamanaka of Kyoto University successfully produced human iPS cells, the risk of the iPS cells becoming cancerous — a key issue to be tackled — has almost been resolved, while the efficiency of production has been enhanced.

The latest surgical procedure carries important implications as a clinical study to confirm the safety of medical technology using iPS cells. Is there an absolute guarantee the iPS cells will not turn cancerous? Are there any unknown risks? These issues will be examined over the next four years.

In an effort to have research results using iPS cells linked to regenerative medicine, the Education, Culture, Sports, Science and Technology Ministry plans to spend ¥110 billion from fiscal 2012 to 2022.

Faster approval needed

Clinical research using iPS cells in the treatment of Parkinson’s disease and serious cardiac failure are planned from next fiscal year.

Also under way is research to determine the mechanisms behind intractable diseases and develop new medicines for such diseases by producing iPS cells from patients with incurable diseases and transforming them into nerve and muscle cells.

It is hoped that with the successful retina cell operation, the transition from basic research to clinical research in regenerative medicine — in which Japan tends to lag behind other major countries — will advance smoothly.

Following the enforcement of the revised Pharmaceutical Affairs Law in November, the time needed to put cells and tissues for regenerative medicine into a practical use will be shortened from the current six years to about three years. If the safety of only a few trials is confirmed and their effectiveness deemed reliable, the ministry will give the go-ahead for practical use, although with conditions attached.

Takahashi has established her own venture firm for producing retina cells derived from iPS cells. Her firm plans to start clinical trials in 2016, with the aim of winning the ministry’s approval in 2018.

A faster approval system must function effectively so patients can receive treatment as quickly as possible.

The RIKEN Center for Developmental Biology, where the clinical trial was conducted, is scheduled to undergo a drastic shakeup. This will include halving the number of research labs due to fraudulent research articles over the so-called stimulus-triggered acquisition pluripotency (STAP) cells.

We hope RIKEN proceeds with its research on regenerative medicine steadily and makes an effort to regain public trust.

(From The Yomiuri Shimbun, Sept. 15, 2014)Speech