Severe heart failure is a solemn condition that worsens over time and the only treatment options are a heart transplant or heart-assisted artificial heart implantation. However, heart transplantation is hampered by a lack of donors and age restrictions, and artificial hearts carry the risk of infection and damage to cranial nerves, as well as long-term deterioration of quality of life.
ReHeart meets these unmet medical needs. The transplant is performed by surgery on 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 aid escalate blood flow and repair tissue. A nationwide, multicenter collaborative study of eight patients with severe heart failure found a trend toward improvement. has been confirmed in four patients, with peak oxygen consumption (peak VO2) increasing by more than 10 percent at 52 weeks after transplantation.
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, created from donor iPS cells. It is indicated for the relief of motor symptoms in patients with Parkinson’s disease who had an insufficient response to existing drug therapies, including levodopa.
Parkinson’s disease is a neurodegenerative disease that causes motor symptoms such as limb tremors and muscle stiffness, caused by the gradual loss of dopaminergic nerve cells in the brain. Current drug therapies are symptom-relieving treatments rather than primary approaches to replacing lost nerve cells.
AmShepla’s goal is to offer a novel treatment option by transplanting progenitor cells from lost dopamine-producing neurons directly into the brain. This transplant is performed using minimally invasive brain surgery. Diminutive holes are drilled in the skull, one on each side, and the cells are dispersed and injected into the capsule on both sides by three routes of administration.
In a study led by a doctor at Kyoto University Hospital, four out of six patients with Parkinson’s disease were analyzed. showed improved leisure time score (post-drug effect score) on the Motor Diagnostic and Treatment Symptom Rating Scale (MDS-UPDRS Part III) 24 months after transplantation. Scientists have confirmed that cells remained profitable in all six patients at the transplant site.
The world’s first production plant and cooperation between industry and academia
SMaRT, located in Suita City, Osaka Prefecture, is responsible for the production of Amshepri and is the world’s first commercial manufacturing facility for regenerative medicine and cellular medicines derived from donor iPS cells. iPS cells used as raw material for the product come from the warehouse provided by Kyoto University iPS Cell Research Foundationand differentiation induction and production technologies are based on proprietary technologies of Kyoto University and other institutions. For example, Eisai’s cell purification technology is used in part of the manufacturing process; the creation of the product was possible thanks to the cooperation of industry and academia with the support of various institutions.
It could also be argued that the fact that this landmark authorization came from Japan was structurally inevitable. This is because the entire supply chain was almost completely completed in Japan, from the development of the core technology by Shinya Yamanaka, winner of the Nobel Prize in Physiology or Medicine in 2012, to the supply of iPS cells by the iPS Cell Research Foundation of Kyoto University, the differentiation induction and production technologies developed by Kyoto University and other institutions, the establishment of the SMaRT commercial production facility, and even the development of university start-ups and the market entry of immense pharmaceutical companies.