Shinya Yamanaka on the Current State of iPSC Therapy

A few weeks ago, I had the pleasure of attending Nobel Prize winner Shinya Yamanaka’s talk on the first day of the UC San Diego-Kyoto University Joint Symposium. Although I’m not a stem cell researcher, I’m very much aware of the buzz surrounding induced pluripotent stem cell (iPSC) research—the excitement that began back in 2006 when Yamanaka’s lab developed the first iPSCs. Stem cells are categorized as undifferentiated cells capable of self-renewing (making more stem cells) as well as having the potential (potency) to give rise to various other cell types. Surprisingly, Yamanaka’s group discovered that mouse embryonic or adult fibroblasts can be transformed (or ‘induced’) into a pluripotent state in the presence of just four transcription factors: Oct3/4, Sox2, c-Myc, and Klf4. This discovery won Dr. Yamanaka the Nobel Prize in Physiology or Medicine in 2012. His talk at the symposium, “New Era of Medicine with iPS Cells,” was partially a plug for the importance of stem cell research funding, but also a general recap of the progression and current state of iPSC research, both in terms of successes and setbacks.
Currently, Dr. Yamanaka is the director of the Center for iPSC Cell Research and Application (CiRA) in Kyoto, Japan. In 2008, the institute received approval to begin generating and conducting research on disease-specific iPSCs. Patients who donated material for the derivation of iPSCs have diseases that include, but are not limited to, Duchenne’s muscular dystrophy, Amyotrophic Lateral Sclerosis (ALS), and Fanconi’s anemia. According to Dr. Yamanaka, the researchers at CiRA have now derived 140 iPSC lines, covering over 90% of the haplotypes found in Japan. These iPSCs went through rigorous testing and appear to have built up fewer mutations than what would naturally occur in humans. This is immensely important as, with any cell therapy approach, the threat of cancer is a harsh reality.

The audience at the talk ranged from seasoned scientists and graduate students (like myself), to members of the general public, some of whom were personally connected to the research . A young member of the audience was curious about the current state of iPSC research that focuses on spinal cord regeneration. She was disabled due to a traumatic spinal cord injury, so although she had no formal scientific training, she and her family have been closely following Dr. Yamanaka’s research over the last few years. Dr. Yamanaka shared that current spinal cord studies in chimps were going well, and with a cautiously optimistic tone, estimated that in a few more years treatment would move into humans. When the same audience member asked about the general success of iPSCs in the clinic, Dr. Yamanaka first paused, and then let out what appeared to be a nervous laugh before explaining a lengthy process that results in a very low success rate.

Although enthusiastic about the current set of researchers at CiRA, and the future of iPSC research and application, Dr. Yamanaka was honest about the discouraging degree of optimization that goes into making, maintaining, and utilizing these iPSC lines. He immediately struck me as someone who is wholly invested in his work, but in a very real, non-idealistic way. He is certainly humble for being so successful at such a young age (and by “young” I mean “scientist young,” i.e., a Nobel Prize before age 50). Although the focus of my PhD does not in any way relate to Dr. Yamanaka’s work, I will definitely be tuned in to the “iPSC buzz” in the coming years.

For more information:

CiRA: Center for iPS Cell Research and Application, Kyoto University (https://www.cira.kyoto-u.ac.jp/e/)

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