World's first approval of iPS cell therapies for commercial use

Japan's health authorities have granted conditional approval to two therapies developed from induced pluripotent stem cells (iPSCs), to treat Parkinson's disease and heart failure.

One therapy, Amchepry (also called raguneprocel) targets Parkinson's disease and involves the transplantation of dopamine-producing cells into patients' brains (see BioNews 975 and 1258). The other therapy, ReHeart, is designed to treat heart failure by using stem cell-derived cardiac cells to support weakened heart muscle (see BioNews 1033). The approval of both treatments represents the first time such therapies have been formally approved for clinical use under a regulatory pathway that allows conditional marketing, rather than being restricted for use within research trials.

'Reaching this milestone exactly twenty years after the first announcement of mouse iPS cells is deeply meaningful, and I am delighted to see such progress toward real-world application,' said Professor Shinya Yamanaka, director emeritus of the iPS Cell Research Institute at Kyoto University, Japan. 'At the same time, establishing this as a medical treatment will require examining safety and effectiveness in many more clinical cases'.

Both therapies build on the work of Professor Yamanaka, who first demonstrated that adult cells could be reprogrammed into a pluripotent state, giving them an embryonic-like capacity to differentiate into any cell type in the body. This discovery was first made in mouse cells in 2006, and later in human cells. Professor Yamanaka was jointly awarded the Nobel Prize in Physiology or Medicine in 2012 alongside Sir John Gurdon of the University of Cambridge (see BioNews 676).

This approval allows the therapies to be manufactured and used clinically, but only under strict post-marketing surveillance requirements – meaning that their developers must continue to collect safety and efficacy data over several years before full authorisation can be granted. The decision reflects Japan's accelerated regulatory pathway for regenerative medicine, enabling earlier patient access while still requiring ongoing evaluation of long-term outcomes.

However, some experts have cautioned that the approval has been granted on the basis of limited data. 

'The current evidence supporting these approvals remains very limited. The clinical studies involved only seven to eight patients and follow-up periods of one to two years, without randomised control groups,' Dr Hiroshi Kawaguchi, an orthopaedic surgeon at Nadogaya Hospital in Chiba, Japan, told Medical News Today.