AsianScientist (Oct. 13, 2014) – Imagine a world where we no longer need blood donors. A world where blood banks are filled to the brim with every possible blood type, coaxed from stem cells that originated from our skin. A world where artificial red blood cells (erythrocytes) are routinely used for blood tranfusions.
Such is the dream of Professor Shinya Yamanaka, Director of the Center for iPS Cell Research and Application (CiRA) at Kyoto University.
“Japan is an aging country; in ten to twenty years we will be short of blood donors,” said Yamanaka. “iPSC-derived red blood cells can be one of those alternatives [for blood transfusions].”
As ambitious as it may sound, these words were spoken by the man who won the Nobel Prize in Physiology or Medicine in 2012 for his central role in the development of induced pluripotent stem cell (iPSC) technology. iPSCs, unlike embryonic stem cells, are simple to produce, and do not require the use of human embryos.
Yamanaka was a panelist at a plenary session on global health at the recently concluded Science and Technology in Society (STS) Forum. The meeting, in its 11th year, took place from October 5-7, 2014, in Kyoto, Japan.
Other panel speakers included Dr. Shigeru Omi, World Health Organization regional director emeritus, Western Pacific office; Mr. Yasuchika Hasegawa, chairman of the Board and CEO of Takeda Pharmaceutical Company Limited; Dr. Klaus Lindpaintner, CSO of Thermo Fisher Scientific; and Dr. Richard Roberts, CEO of New England Biolabs.
Alluding to the recent successes of iPSC technology in the clinic, Mr. Henry McKinnell, Chairman of Moody’s Corporation and chairperson of the session, said that the world is due for a renaissance in medical research.
“I’m convinced the golden age of medicine is yet to come, and it will come in our lifetime,” McKinnell said.
“HIV (human immunodeficiency virus) is 100 percent preventable, but it is killing more than two million people a year. 164,000 people a year, most under five years of age, die from measles, when they could be easily vaccinated for less than one dollar per dose. There is an epidemic of cardiovascular diseases and cancers,” he added.
iPSC technology in the clinic
Aside from red blood cells, iPSCs could be differentiated into many other blood cell types, said Yamanaka. For example, along the white blood cell lineage, cytotoxic T killer cells could be used to kill tumor cells, while HIV-resistant CD4+ cells could be used in the treatment of HIV.
In other examples, Yamanaka described on-going projects that use iPSC-derived neurons to treat Parkinson’s disease, and iPSC-derived cardiomyocytes to treat heart failure.
Indeed, iPSC research has begun to address global health concerns, said Yamanaka. A recent clinical trial in Japan tested the safety and feasibility of taking iPSCs from adult patients to treat wet-type age-related macular denegeration (AMD), a blindness caused by progressive damage to the retinal pigment epithelium (RPE) from blood vessel growth.
Led by Professor Masayo Takahasi of the RIKEN Center for Development Biology, alongside doctors at the Kobe City Medical Center General Hospital, the team differentiated patient-derived iPSCs into RPEs, before transplanting the RPEs back into the patient’s eye. Six patients received iPSC transplants at a cost of approximately US$400,000 per eye.
A challenge worth taking
Despite inroads into the clinic for AMD treatment, the field of iPSCs is still very young, Yamanaka said. It is an important pursuit, he noted, as the gains from using iPSCs will largely overcome the many regulatory hurdles that still stand in the way. For example, opportunities exist to use iPSCs to speed up the drug development cycle.
“Drug development takes about ten years. In Phase I clinical trials, success is pretty low, at 20 percent or even 10 percent,” he said. “I believe that using iPSCs at various stages of drug development, we can double or even triple the success rate,” he concluded.
Juliana Chan was invited by the Japanese Society for the Promotion of Science and the New York Academy of Sciences to attend the 2014 Forum as one of its ten “Future Leaders”.
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Copyright: Asian Scientist Magazine.
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