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Embryonic stem cells: first clinical trials

Human embryos at various early stages of development
Human embryos at various early stages of development

Therapies based on human embryonic stem cells (ES), are likely to revolutionize medicine in the twenty-first century. Properly grown and oriented laboratoirees, these cells can be transformed at will into specialized cells (neurons, liver cells, heart, etc.).

In animals, injection of embryonic cells has already helped cure some genetic diseases. These cells can also be used to repair tissues that do not regenerate naturally (brain, spinal cord). In humans, the first controlled clinical trials are beginning to emerge. They are currently testing designed to prove the safety of treatments, a preliminary step before their effectiveness can be studied in turn. And unsurprisingly, the United States has already one step ahead in this area.

A second clinical trial is in effect this week. The company Advanced Cell Technology has been authorized to test the injection into the eye of retinal cells derived from embryonic stem cells in ten volunteer patients, suffering from the same incurable genetic eye disease. This procedure, applied initially to the elderly, seeks to prove the safety of treatment. Eventually, it should help care for children who lose their sight because of this inherited disease called Stargardt macular dystrophy.

In January 2009, the U.S. company Geron had already obtained a permit for a similar test for safety. It was now time to test the use of ES cells to repair severed spinal cord of injured people. This trial began in mid-October with the first patient in which two million of these stem cells (grown to become neurons) were administered. The results are not yet known. If prove the safety of treatment remains the primary objective, the researchers hope to observe the first positive effects on motor function of patients.

These three tests will raise several highly anticipated fears. Firstly, the risk of cancer induction. "Research on animals have shown that when all the injected stem cells had not been transformed into specialized cells previously, tumors were formed," warns George Uzan, director of an INSERM unit working on therapeutic applications stem cells. As for transplants, the risk of rejection of embryonic cells, since they necessarily come from foreign agencies, will also be studied carefully.

In case of bad results - and many disappointments had been with adult stem cells - (Induced pluripotent stem cells, iPS) cells offer only one more hope. Developed in 2004, these stem cells "made" from the skin include the ability to put aside the ethical issues related to the destruction of human embryos.


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