IPSC against macular degeneration: the first clinical study

The Japanese Ministry of Health has allowed the first trial of pluripotent cells in humans

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The Special Committee of the Japanese Ministry of Health on Safety has given the go-ahead to launch the world's first clinical trials of the medical potential of induced pluripotent stem cells (iPSC), the journal Nature (Next-generation stem cells cleared for human trial) reports. It is expected that the first transplant operation for a patient with wet age-related macular degeneration of retinal pigment epithelium cells obtained from his skin cells will take place in the coming days.

Age-related macular degeneration (macular degeneration), characterized by damage to the central area of the retina, the macula, is the leading cause of irreversible vision loss among the population of developed countries aged 50 years and older. In the wet form of the disease, new blood vessels begin to grow behind the retina, affecting the pigment epithelium, which leads to impaired photoreceptor nutrition and blindness.

A group led by ophthalmologist Masayo Takahashi from the Center for Developmental Biology at the RIKEN Institute officially began recruiting study participants on August 1, 2013.

To obtain a graft that will restore the degenerated retinal pigment epithelium, Takahashi and her colleagues took a peppercorn-sized skin sample from the shoulder of each of the six selected patients and reprogrammed these cells with the help of certain proteins into induced pluripotent cells capable of turning into cells of different specialization. The addition of other factors turned them into retinal cells. This process took about 10 months.

A small layer of the obtained retinal cells is now planned to be placed under the damaged area of the retina of the first participant, where, if everything goes well, they will begin to grow and restore the pigment epithelium. Since the new retinal cells will be genetically identical to the patient's own retinal cells, the authors of the method hope that problems with rejection will be avoided. In any case, preliminary studies conducted by Takahashi's group on monkeys did not reveal such a risk. Another concern about the safety of clinical use of iPSC is the possibility of uncontrolled malignant growth of introduced cells, however, the results of experiments conducted by Takahashi's group on mice and monkeys did not reveal an increased risk of such a development of events. In addition, in order to avoid the possibility of dangerous mutations appearing in the process of reprogramming cells, additional tests for their genetic stability were carried out.

All this allowed the Takahashi group to obtain permission to start clinical trials from a special committee of the Ministry of Health of Japan. The presentation of the technique was reportedly attended by Shinya Yamanaka, who, together with John B.Gurdon, received the 2012 Nobel Prize in Physiology or Medicine for the discovery of iPSC.

It is expected that the stage of engraftment of new retinal cells will last about eight weeks. Then, during the year, the researchers will monitor the process of restoration of the pigment epithelium.

Portal "Eternal youth" http://vechnayamolodost.ru 11.09.2014