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First-Ever Human Trial of an Induced Pluripotent Stem Cell Treatment Set To Begin

November 27, 2014

stem cells

What are Induced Pluripotent Stem Cells

Induced pluripotent stem cells (iPSCs) are adult cells that have been genetically reprogrammed to an embryonic stem cell-like state through being forced to express genes and factors important for maintaining the defining properties of embryonic stem cells.

Cells of this type have been considered to be promising for future treatments since their creation eight years ago, which in itself was a milestone. Research had been done, showing that it was known that iPSCs are already useful tools for drug development and modeling of diseases, and scientists were ultimately hoping to use them in transplantation medicine. Viruses were used to introduce the reprogramming factors into adult cells, and this process had to be carefully controlled and tested before the technique could lead to useful treatment for humans.

Why iPSCs are Unique

Induced pluripotent stem cells are special because they are not made from embryos. Rather, they come from gathering skin cells from adults and then treating those cells with genes that reverse the cell’s life stage back to its stem cell state. This means that scientists are able to make induced pluripotent stem cells from cells taken from a patient’s own body.

The cells that result should be well matched to the patient’s own genetics, however, it is possible that the “induction” part of the process introduces genetic aberrations to the cells.

The Future of Induced Pluripotent Stem Cells

It is finally being done: iPSCs are going to be used for human treatment. A Japanese student with a severe eye disease called macular degeneration is set to become the first person to be treated by Induced Pluripotent Stem Cells. Macular degeneration is a full-blown disease where people lose the light-detecting cells in the retinas in their eyes. Scientists have already tried using embryonic stem cells as a treatment for macular degeneration.

Stem cell therapies have been known to possibly carry the risk of tumors. In animal cases, the virus used to introduce the stem cell factors can sometimes cause cancers. However, the scientists in charge of the Japanese trial say that they found their treatment did not cause tumors in mice or monkeys. Stem cell therapies are just now being tried on humans, so there may be other risks to the treatment that scientists may not be aware of.

How It Will Work

The trial being conducted features a treatment created by Masayo Takahashi.

She is an ophthalmologist with a Japanese research institute called RIKEN. Takahashi has been making induced pluripotent stem cells and growing those cells into a sheet of replacement retinal cells. Then, she surgically attaches the sheet onto the retina.

According to Masayo Takahashi and her fellow scientists, this treatment has been previously demonstrated and shown to work positively in monkeys.

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