Why I'm sure human stem cell trial will be safe – 05 …

Posted: Published on February 9th, 2014

This post was added by Dr. Richardson

Read more: Click here to see the original, longer version of this article

You will soon start the first ever human study using induced pluripotent stem (iPS) cells. Why is this such a big deal?

Stem cells can become any other type of cell. If we can use them to replace damaged cells it has huge implications for treating degenerative diseases. Trials of embryonic stem cells are under way, but their use can be ethically controversial. Often, too, patients need to take drugs to prevent their immune systems from rejecting this type of cell. Since iPS cells are made from a patient's own body, it removes the ethical issues as well as the need for immunosuppressant drugs.

How would treatment with iPS cells work?

You create iPS cells by injecting adult cells taken from the body with "reprogramming" genes that make them rewind to an embryonic state. Then, by injecting certain proteins, we can make iPS cells differentiate into liver, retina or any other type of cell. These can be inserted in the body to replace damaged cells. We are still 20 years from clinical treatments, but the potential is exciting.

Are there pitfalls with iPS cell treatments?

We have to be very careful because iPS cells multiply endlessly. So, if any undifferentiated iPS cells were accidentally put into someone, they could cause tumours. That's why this pilot study is so important to confirm the safety of putting cells derived from iPS cells into humans.

Who are the participants in the study?

Six people with age-related macular degeneration in their eyes, a condition that weakens central vision. For these patients, it is caused by the deterioration of the retinal pigment epithelium (RPE) the layer of cells that clears away debris on the retina. We aim to replace damaged parts of this layer by taking skin cells from the patients' arms, reprogramming them to iPS cells, then differentiating them into the required RPE cells.

Are you confident the pilot will be a success?

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Why I'm sure human stem cell trial will be safe - 05 ...

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