Japan's health ministry panel on Thursday approved the commercialization of two regenerative medicine products derived from iPS cells, marking a global first. These treatments target patients with severe heart failure and Parkinson's disease, under a conditional approval requiring data collection for up to seven years. Shinya Yamanaka, pioneer of iPS cell research, expressed delight at this milestone.
Japan's health ministry expert panel on Thursday recommended that the minister approve the production and sale of two regenerative medical products using induced pluripotent stem (iPS) cells, poised to be the world's first of their kind.
One is ReHeart, developed by Cuorips Inc., a startup from Osaka University. It targets patients with severe heart failure due to ischemic cardiomyopathy. Heart muscle sheets derived from human iPS cells are placed on the heart's surface to promote new blood vessel formation and restore function. In 2020, an Osaka University team performed the world's first such transplant. By 2023, trials on eight patients confirmed safety and efficacy, showing reduced symptoms like exhaustion and palpitations, plus improved heart function and physical fitness.
The other is Amchepry, from Sumitomo Pharma Co. and Racthera Inc., for Parkinson's disease. iPS cells are differentiated into dopaminergic neural progenitor cells and injected into the brain. Trials from 2018 to 2023 at Kyoto University Hospital and others involved seven patients in their 50s and 60s; six were assessed. The cells produced dopamine, and four showed motor function improvements after two years of observation. No serious side effects occurred, with better results in younger patients with milder symptoms.
iPS cells were pioneered by Shinya Yamanaka, who generated mouse iPS cells in 2006 and human ones in 2007, earning the 2012 Nobel Prize in Physiology or Medicine. Using patient-derived cells aims to minimize rejection, though long-term safety remains a challenge. The approvals are conditional based on small trials, with rollout pending discussions on pricing, insurance, and manufacturing. Cuorips aims to launch sales this year.
Yamanaka said, "I am very happy to see the first big step toward its societal implementation, 20 years since it was announced." The panel set a seven-year approval term; if safety is later confirmed, full use without restrictions will be allowed.
