The Japanese government has approved the world's first regenerative medicine therapies using induced pluripotent stem cells (iPS cells).
The Ministry of Health, Labour and Welfare said on the 6th that it approved manufacturing and sales for two iPS cell therapies targeting heart disease and Parkinson's disease.
The approved therapies are ReHeart, a severe heart failure treatment developed by Cuorips, a startup founded at Osaka University, and Amchepry, a Parkinson's disease treatment developed by Sumitomo Pharma.
This is the first time iPS cell therapies have been commercialized. iPS cells are cells created by introducing specific genes into adult cells such as skin or blood so they can differentiate into various cell types like embryonic stem cells. They have been seen as a new technology that could overcome the limits of disease treatment by creating needed cells to help repair damaged organs or tissues.
Amchepry, the Parkinson's disease therapy approved by Japan, was developed using this principle. Blood cells are collected from a cell donor and reverted to iPS cells, then differentiated into precursor cells of dopamine-producing neurons. The cells are then transplanted into the brains of patients with Parkinson's disease to help restore the function of damaged dopamine neurons. Parkinson's disease is a representative neurodegenerative disorder in which dopamine-producing neurons in the brain gradually disappear, leading to tremors, muscle rigidity, and movement disorders.
ReHeart, the severe heart failure therapy, is made by differentiating iPSCs into cardiac muscle cells. About 100 million heart cells are cultured into a coin-shaped tissue and transplanted onto the surface of the heart of patients with ischemic cardiomyopathy to help restore cardiac function.
The two therapies approved this time were cleared under Japan's conditional approval system introduced for regenerative medicine.
The system grants priority approval based on fewer patient data than conventional drugs to deliver regenerative medicine products to patients more quickly. In fact, ReHeart was evaluated using clinical trial data from eight patients, and Amchepry from seven patients. The corporations must conduct additional trials after launch to re-verify efficacy and safety, and they must obtain full approval within a set period.
Going forward, additional trials are planned for 75 patients for ReHeart and 35 patients for Amchepry. For the Parkinson's disease therapy, 30 of these will be evaluated in patients age 65 or younger.
With the therapies approved, the Japanese government will decide on drug pricing and whether to include them under health insurance coverage. In Japan, drug prices for regenerative medicine products are typically set within four to five months after approval. As a result, the two therapies could be used to treat patients as early as this summer.
iPS cells are a technology developed by Yamanaka Shinya of Kyoto University. He received the 2012 Nobel Prize in Physiology or Medicine for establishing a method of "cell reprogramming" that reverts adult cells to a stem cell state. This year also marks the 20th anniversary of Yamanaka first creating iPS cells in mice in 2006.