TOKYO -- Fujifilm will apply for permission as soon as this fiscal year to run clinical trials on a treatment using a type of stem cell derived from adult tissue, aiming to become Japan's first company to test the cells for practical use in regenerative medicine.
The Fujifilm Holdings unit aims to start testing the therapy, aimed at patients with severe complications from leukemia treatment, in 2019, and hopes to win approval to manufacture and market the therapy in 2022. Preliminary discussions with oversight agencies are nearly complete.
The therapy uses induced pluripotent stem cells, or iPS cells -- adult cells reprogrammed to an essentially embryonic state from which they can be used to produce any type of body cell. Such cells are being heavily researched for their promise in regenerative medicine, and companies are working to build businesses around the cells, as has been done with drugs and medical devices.
Fujifilm's trials would target tens of patients suffering from acute graft-versus-host disease, a condition affecting about 40% of bone marrow transplant recipients. The condition can cause inflammation in the skin and liver, as well as diarrhea and vomiting, and occasionally death. There are about 1,000 cases in Japan, with more than 10,000 internationally, including leading Western countries.
The Japanese company aims to exploit the experience of Australian venture Cynata Therapeutics, in which Fujifilm holds a stake. Cynata has clinical trials underway in the U.K., with 14 of 15 patients so far fully recovered or showing improvement.
The therapy involves using iPS cells to make mesenchymal stem cells -- a variety that can form tissues like cartilage or fat. Injecting patients with such cells is expected to suppress their immune systems and keep immune cells in transplanted marrow from attacking their bodies.
Down the road, Fujifilm plans to apply for trials in the U.S. and to carry them out around the world. It would mass-produce mesenchymal stem cells made from iPS cells, prepare them as pharmaceuticals, and market them to medical institutions and others.
Transplants of mesenchymal stem cells are seen to be effective in treating such conditions as strokes; ulcerative colitis, which can cause severe diarrhea and bloody stool; and critical limb ischemia, which can result from hardened arteries or severe diabetes. Transplants of the cells are a topic of research in a variety of countries.
Japanese research institutions are running trials on iPS-related treatments, though these efforts are more focused on safety testing than realizing practical use. The government-backed Riken laboratory is carrying out transplants using iPS cells for patients with incurable eye conditions, while Osaka University has plans for serious heart failure, and Kyoto University has begun physician-led trials in using the cells to treat Parkinson's disease.