Scientists advance in the use of stem cells for incurable diseases

A group of scientists from the University of Colorado has achieved positive results in an experiment with stem cells extracted from the skin and that has the “potential” to successfully treat diseases that have hitherto been incurable.

Scientists have “reprogrammed” adult cells, healthy or diseased, into pluripotent stem cells (iPSC), which gives hope in the treatment of serious diseases and leads to the start of future clinical trials.

According to Ena Ganna Bilousova, from the Gates Center for Regenerative Medicine, at the Anschutz medical campus of the University of Colorado, and one of those responsible for the research, the tests developed have helped to resolve the inefficiency registered to date when creating stem cells from adult cells.

Bilousova said that currently, of every 1,000 adult cells “only one or two become iPSC”, which were discovered by Shinya Yamanaka in 2006 and that subsequently earned the researcher the Nobel Prize for Medicine.

“Researchers in Colorado have found a way to dramatically accelerate that process while improving the safety of this technology for clinical applications,” Bilousova said.

In this experiment, whose results were published on February 21 in the journal Nature Communications, researchers focused on skin diseases and reprogramming cells, healthy or sick, in iPSC.

Basically, the researchers focused on skin diseases and reprogramming healthy or diseased cells, that is, reactivating certain non-active genes in adult cells to transform those cells into iPSC.

The new method allows an unlimited number of cells belonging to the patient, to generate the iPSC outside the body, to manipulate them genetically, to convert them into cells of various types and to transplant them to the patient, or to use them for future medical research.

The breakthrough was achieved because no way was sought to improve existing methods, Bilousova said, but decided to use ribonucleic acid (RNA) molecules to accelerate the transformation (“reprogramming”) of adult cells.

“We were surprised to discover how simple manipulations of time and doses of RNA molecules can affect the efficiency of reprogramming,” said the researcher.

Photo courtesy, Sunday, February 25, 2018, by the Gates Center for Regenerative Medicine, the Anschutz Medical Campus of the University of Colorado, researchers Ganna Bilousova (back) and Igor Kogut, in Denver (USA). EFE

He added that the process is now less toxic and so precise that it can be applied to a single cell.

Dennis Roop, director of the university’s Gates Center and another of the research leaders, believes the breakthrough has the potential to help develop therapies based on adult stem cells “to cure diseases so far without cure, such as bullous epidermolysis” (EB), which make the skin more fragile.

This group of diseases affects between 25,000 and 50,000 people in the United States alone and half a million people in the rest of the world, according to DEBRA International, the global association that investigates bullous epidermolysis.

“There are no effective therapies for EB, and iPSC technology provides the opportunity to develop a permanent corrective therapy based on stem cells for these serious diseases that produce blisters on the skin,” Gates said in a statement released by the institution this week.

The University of Colorado estimates that the results of its research will allow the approval of the Food and Drug Administration (FDA) of the United States to begin clinical trials, which would be the first method based on iPSC that “leaves the laboratory” .

To accelerate the studies, the University of Colorado has signed a collaboration agreement with Stanford University and Columbia University, with which has established a consortium for the future effective treatment of epidermolysis bullosa diseases based on pluripotent cells. (efeusa)


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