Japan approves world’s first iPS stem-cell treatments for Parkinson’s and heart failure

Japan has cleared two pioneering stem-cell therapies, one for Parkinson’s disease and another for severe heart failure, according to the manufacturer and local media reports on Friday (March 6). The treatments could begin reaching patients within months. Pharmaceutical firm Sumitomo Pharma announced it had received approval to produce and sell Amchepry, a Parkinson’s therapy that involves transplanting stem-cell-derived cells directly into a patient’s brain.

At the same time, Japan’s health ministry approved ReHeart, a regenerative treatment created by medical startup Cuorips. The therapy uses sheets of heart muscle cells to stimulate the formation of new blood vessels and improve heart function in patients with severe heart failure.

Reports indicate the treatments could become available to patients as soon as this summer. If launched, they would be the first commercially available medical products based on induced pluripotent stem (iPS) cells. Japanese scientist Shinya Yamanaka received the 2012 Nobel Prize in Physiology or Medicine 2012 for discovering iPS cells, which can develop into almost any type of cell in the human body. "I hope this will bring relief to patients not only in Japan but around the world," health minister Kenichiro Ueno told a press conference. "We will promptly carry out all necessary procedures to ensure it reaches all patients without fail."

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In a statement, Sumitomo Pharma said it received "conditional and time-limited approval" to manufacture and market Amchepry under a regulatory framework designed to speed innovative treatments to patients. According to the Asahi Shimbun newspaper, the authorization functions as a provisional license. The safety and effectiveness of the therapy were evaluated using a smaller number of patients than typically required in standard drug trials. A clinical study conducted by researchers at Kyoto University suggested the treatment was safe and helped improve symptoms in some patients. The trial included seven Parkinson’s patients aged between 50 and 69. Each received either five million or 10 million cells implanted on both sides of the brain.

Researchers used iPS cells from healthy donors and converted them into precursor cells that produce dopamine—cells that are gradually lost in people living with Parkinson's disease. Patients were monitored for two years, and researchers reported no major adverse effects. Four of the seven participants showed measurable improvement in their symptoms.

Parkinson’s disease is a progressive neurological condition that affects movement and commonly causes tremors, stiffness, and difficulty with coordination.

Globally, around 10 million people are living with the condition, according to the Parkinson's Foundation. Current treatments mainly help manage symptoms but do not stop or reverse the progression of the disease. Induced pluripotent stem cells are produced by reprogramming mature, specialized cells back into a youthful, versatile state, allowing them to transform into many different cell types without the use of embryos. This technology remains one of the most promising areas of modern medical research.