Japan’s Ministry of Health has granted conditional approval for two regenerative therapies based on iPS cells – Amchepry for the treatment of Parkinson’s disease and ReHeart for patients with severe heart failure caused by ischemic cardiomyopathy. The decision paves the way for the first commercial application of this technology in clinical medicine.
Amchepry, developed by Sumitomo Pharma in collaboration with Racthera, involves transplanting cells derived from induced pluripotent stem cells into the brain. These cells are transformed into precursors of dopamine-producing neurons – the neurotransmitter whose deficiency is responsible for the symptoms of Parkinson’s disease.
The regulatory decision was based on data from a small clinical study conducted by researchers at Kyoto University. The study involved seven patients aged between 50 and 69. Each patient received between 5 and 10 million cells implanted into both hemispheres of the brain. Participants were monitored for two years – no serious adverse effects were observed, and four patients showed improvement in disease symptoms.
The second therapy approved is ReHeart, developed by the startup Cuorips. The treatment involves implanting sheets of heart muscle cells produced from iPS cells. These structures are designed to support the formation of new blood vessels and improve heart function in patients with advanced heart failure.
The approval was granted under Japan’s accelerated regulatory pathway for innovative therapies. This framework allows treatments to enter the market conditionally after safety and preliminary effectiveness have been demonstrated, even with limited clinical data. However, companies must provide additional evidence confirming effectiveness within seven years in order to obtain full approval.
Induced pluripotent stem cells can transform into nearly any type of cell in the human body. The technology was developed by Japanese scientist Shinya Yamanaka, who received the Nobel Prize in 2012 for discovering a method of reverting mature cells to a pluripotent state.