Japan’s regenerative medicine & cancer know-how could help KO chronic diseases

Last month, a Japanese pharmaceutical company opened a “cell factory” in Osaka that’s been described as the world’s first to produce induced pluripotent stem (iPS) cells for commercial applications. It’s based on regenerative medicine technology that can be used to reprogram healthy cells so they replace diseased tissue.

The Sumitomo Dainippon Pharma Co. facility in Suita, Osaka, is one of the latest examples of Japan’s leading role in regenerative medicine. The field was given a huge shot in the arm when Kyoto University researcher Shinya Yamanaka won the Nobel Prize in physiology or medicine in 2012 with John Gurdon for discovering that mature cells can be reprogrammed to become stem cells.

Yamanaka, now director of the Center for iPS Cell Research and Application (CiRA) at Kyoto University, recently participated in a panel discussion about what advances in Japanese medical R&D could mean for patients around the world at the 2018 New Economy Summit (NEST) conference in Tokyo. Joining him was Hisataka Kobayashi, chief scientist at the National Cancer Institute’s Molecular Imaging Program, along with JANE Chair and Rakuten CEO Hiroshi Mikitani, who served as moderator.

“Theoretically, we can make iPS cells differentiate into any cell in the body,” Yamanaka said during an overview of cellular reprogramming, in which body cells are restored to their original stem cell state with the ability to differentiate into any type. “Diseased cells in each patient can be replaced.”

Last year, a Japanese man in his 60s became the world’s first person to receive retinal cells derived from iPS cells donated from another person. The transplant followed a 2014 operation in which a Japanese woman received retinal cells for age-related macular degeneration, which can cause blindness. In that case, however, the iPS cells were a reprogrammed version of her own skin cells.

iPS cells could also be used to help treat Parkinson’s disease, heart failure, spinal cord injury and other disorders. But Yamanaka noted that iPS cells can be studied to create new medicines. For instance, Kyoto University researcher Haruhisa Inoue managed to create motor neuron cells from iPS cells taken from people with amyotrophic lateral sclerosis (ALS), an incurable degenerative disease. CiRA has been testing over 50 new compounds on these motor cells and some compounds are preventing the cells from dying as they would normally.

“Of course, pluripotent cells are not a panacea,” said Yamanaka. “But they could produce many powerful tools for many of the difficult diseases that we have no cure for.”

Kobayashi of the National Cancer Institute gave attendees an overview of a new approach he developed to fight cancer. He first introduced the “science of destruction” involved in cancer treatment – that is, the use of surgery, radiation and chemotherapy to attack cancer cells in patients. Cancer is a leading cause of death in Japan and many other countries. Over 370,000 Japanese are estimated to have died of cancer in 2017, and anticancer therapies have changed little in decades; they often harm healthy tissue and cells.

Kobayashi, however, explained how a novel approach called near-infrared photoimmunotherapy (NIR-PIT) uses infrared light for targeted killing of cancer cells and boosts the immune action against tumor cells in different locations. Photoabsorbing chemicals are attached to antibodies and these are injected into patients. After the antibodies bind to the surface of tumor cells, it’s exposed to near-infrared light, damaging the cell membrane and leading to cell death. Nearby cells are not affected. Kobayashi’s team patented NIR-PIT, licensed the know-how to innovative San Diego biotech company Aspyrian Therapeutics Inc.—led by Hiroshi Mikitani who serves as Chairman—and has been using it in clinical trials.

“Twenty to 30% of patients can be helped completely with this and won’t relapse, but it’s not helpful in all cases,” Kobayashi said. “It’s quite important that we convert immune cells so that they can recognize tumors and we’re helping with this.”

“Cancer is no easy foe,” said Yamanaka, adding that he was shocked to first learn of the potential of PIT a few years ago.

“Pharmaceutical companies globally tested tons of antibodies against cancer but failed,” Yamanaka added. “However, maybe they can combine them with photoimmunotherapy, and if people working in pharmaceutical companies can overcome organizational silos and barriers and work together, maybe in a short period of time we can overcome cancer.”