Japan Approves the World’s First Treatment Made With Reprogrammed Human Cells | WIRED

Save Story Save this story Save Story Save this story On March 6, Japan’s Ministry of Health, Labor and Welfare officially granted conditional and time-limited marketing authorization to two regenerative medical products derived from reprogrammed iPS cells , marking exactly 20 years since the creation of mouse iPS cells . These will be the world's first practical application of iPS cell-derived products. “We are very pleased to have taken a major step toward social application on the 20th anniversary of the announcement of mouse iPS cells,” said Shinya Yamanaka, director emeritus of the iPS Cell Research Institute at Kyoto University. “To establish it as a medical treatment, it is essential to go through the process of confirming its safety and efficacy in many more cases. I believe it is important that we continue to move forward steadily, step by step, with scientific caution and without getting carried away.” What Are iPS cells? The initialism stands for induced pluripotent stem cells. These are adult cells, such as skin or blood cells, that have been reprogrammed to function like embryonic stem cells. They can divide indefinitely and can take the form of any cell type. Thanks to this ability, they can be used to test new drugs and for research in regenerative medicine. This type of cell allows for scientific advancement without the ethical problems that arise from working with embryonic stem cells. Sheets of Cells Attached to the Wall of the Heart One of the regenerative medicine products that has been approved is ReHeart, from Qualipse , a startup based at Osaka University. It's a sheet of specialized heart cells called cardiomyocytes differentiated from iPS cells of healthy donors, and it generated considerable buzz when it was presented at the Osaka-Kansai Expo. It's intended for patients with severe heart failure due to ischemic cardiomyopathy who don't recover sufficiently despite standard treatment, including drug therapy and invasive procedures. Qualipse's iPS cell-derived cardiac muscle sheet. It was also the focus of much attention when it was exhibited at Expo 2005 Osaka-Kansai. Severe heart failure is a serious condition that worsens over time, and the only treatment options are a heart transplant or a heart-assisted artificial heart. However, heart transplantation is hampered by a shortage of donors and age restrictions, and artificial hearts also carry the risk of infection and cranial nerve damage, as well as long-term decline in quality of life. ReHeart addresses these unmet medical needs. The transplant is performed via a surgery in the left side of the chest, with three sheets of cardiomyocytes attached to the surface of the heart. Signaling proteins secreted by the transplanted cells help increase blood flow and repair tissue. In a national multicenter collaborative study that included eight patients with severe heart failure, a trend toward improvement was confirmed in four patients, with peak oxygen consumption (VO2 peak) increasing by more than 10 percent at 52 weeks post-transplant. Neurons “Implanted” Directly Into the Brain The second approved product is Amusepri (generic name: laguneprocell) from Sumitomo Pharma and Racthera. It consists of precursor cells destined to become dopamine-producing neurons made from donor iPS cells. It is indicated for improving motor symptoms in patients with Parkinson's disease who have had an inadequate response to existing drug therapies, including levodopa-containing preparations. Parkinson's disease is a neurodegenerative disorder that causes motor symptoms such as tremors in the limbs and muscle rigidity due to the gradual loss of dopaminergic nerve cells in the brain. Current drug therapies are treatments to alleviate symptoms, not a fundamental approach to replacing lost nerve cells. AmShepli aims to offer a new treatment option by transplanting progenitor cells from lost dopamine-making neurons directly into the brain. This transplant is performed using a minimally invasive type of brain surgery. Small holes are drilled in the skull, one on each side, and the cells are dispersed and injected into the capsule on both sides via three delivery routes. In a physician-led trial conducted at Kyoto University Hospital, four of the six Parkinson's disease patients analyzed showed an improvement in their off-time score (the score when the drug's effect has worn off) on the Motor Symptoms Diagnostic and Treatment Rating Scale (MDS-UPDRS Part III) 24 months after transplantation. Researchers confirmed that the cells remained viable in all six patients at the transplant site. World’s First Manufacturing Facility and Industry-Academia Collaboration SMaRT, located in Suita City, Osaka Prefecture, is responsible for the production of Amshepri and is the world's first commercial manufacturing facility dedicated to regenerative medicine and cell-based drugs derived from donor iPS cells. The iPS cells used as raw