World’s First iPS Transplant Performed

A Japanese woman in her seventies has become the world’s first person to receive a transplant of iPS-derived cells.

AsianScientist (Sep. 18, 2014) – A Japanese woman in her seventies suffering from age-related macular degeneration (AMD) has become the first person in the world to be treated with induced pluripotent stem cells (iPS). The landmark surgery was performed at the Institute for Biomedical Research and Innovation in Kobe, Japan on September 12, 2014.

Although stem cells hold great promise for regenerative medicine, applications have been held back by concerns over immune rejection and ethical concerns regarding the destruction of embryos. Both these concerns may soon be historical, thanks to the discovery of iPS cells which won Professor Shinya Yamanaka the Nobel Prize in 2012. He found that a cocktail of four transcription factors was sufficient to reprogram any cell in the human body into a stem cell, paving the way for personalized stem cell therapy.

Since then, the technology has matured and scientists have been feverishly working on bringing iPS cells to the clinic. The team led by Professor Masayo Takahashi from the RIKEN Center for Developmental Biology (CDB) proceeded with the surgery just four days after receiving approval from a 19-member health-ministry committee, a move which has sparked envy among iPS researchers where clinical trials with iPS cells have yet to be approved.

Prof. Takahasi and her team took skin cells from the patient and transformed them into iPS cells. They then coaxed the cells into differentiating to retinal pigment epithelium (RPE) cells, successfully growing a 1.3 by 3 millimeter sheet which was then implanted into the patient’s retina. Although the procedure is not expected to completely restore vision, the researchers hope to see if it will prevent the further degradation of the retina and importantly, whether there are any serious side effects.

The main concern over the use of iPS cells is whether they contain genetic defects that could cause tumors. In order to receive approval for this novel procedure, the research team showed that the iPS cells did not cause tumors in both mice and monkeys. Furthermore, they also performed tests of genetic stability and found the cells to be safe.

The patient “took on all the risk that go with the treatment as well as the surgery”, Dr. Yasuo Kurimoto of the Kobe City Medical Center General Hospital, who performed the operation. “I have utmost respect for the bravery she showed in resolving to go through with it.”

Dr. Kurimoto also credited Professor Yoshiki Sasai for his pioneering efforts in stem cell research. The RIKEN CDB researcher recently committed suicide, apparently over his involvement in the STAP cell controversy.

“This project could not have existed without the late Yoshiki Sasai’s research, which led the way to differentiating retinal tissue from stem cells,” he said.

The success of the surgery comes as a breath of fresh air for RIKEN, which has recently received extensive negative press coverage over the retraction of the STAP cell papers and the subsequent fallout. In particular, it is reprieve for the beleaguered CDB, which will be halved in size in response to calls for a complete overhaul.

Aside from RIKEN and Japan, iPS scientists around the world are also eagerly awaiting the safety data to come out of the trial, which is eventually planned for six patients.

“If Masayo can demonstrate that these cells are safe in patients, that will have calmed some of the anxiety about the new cell type out there,” Dr. Kapil Bharti from the National Eye Institute in Maryland told Nature News.


Copyright: Asian Scientist Magazine; Photo: RIKEN.
Disclaimer: This article does not necessarily reflect the views of AsianScientist or its staff.

Rebecca did her PhD at the National University of Singapore where she studied how macrophages integrate multiple signals from the toll-like receptor system. She was formerly the editor-in-chief of Asian Scientist Magazine.

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