Stem Cells Created From Skin May Help In the Fight Against Multiple Diseases

Author: Drucilla Dyess

Using a new method known as induced pluripotent stem (iPS), ordinary cells have been re-programmed to look and act like embryonic stem cells. Experts in stem cell research have now produced a collection of these master cells using skin and bone marrow cells from patients that will be shared freely with other researchers for a nominal cost to cover expenses.

In the journal Cell, the Harvard Medical School and Children's Hospital in Boston team write that the point is not to treat anyone yet, but to get as many researchers as possible experimenting with the cells in lab dishes for a better understanding of the diseases. These cells will have to be coaxed into tissues of different types. "That is where all of the science will go on over the next many, many years," said Dr. George Daley, principal member of the institute and senior author of the paper.

Per Doug Melton, the co-directory of Harvard Stem Cell institute, the lines of cells will be made available to researchers through an institute facility established at Massachusetts General Hospital. This new lab is up and running and prepared to shipping lines, according to Daley.

Embryonic stem cells have the ability to produce any type of tissue or blood cell. The new cells come from patients with 10 incurable genetic diseases and conditions, including Parkinson's, the paralyzing disease amyotrophic lateral sclerosis, or ALS, juvenile diabetes and Down's syndrome. Daley noted that this is only the first wave of diseases.

"This is a broader and more important collection of degenerative diseases for which there are no good treatments and, more importantly, no good animal models," Melton said. "The cells will allow researchers access for the first time to cell types of interest, to watch the disease progress in a dish, to watch what goes right or wrong. We'll see in the years ahead that this opens the door to a new way of treating degenerative disease."

Only last week, a team of scientists from the institute announced the successful transformation of skin cells from patients with Lou Gehrig's disease (ALS) into motor neurons genetically identical to patient neurons. This gives scientists the ability to create unlimited numbers of cells to better study the disease process.

Daley and Melton explained that every cell in the human body contains the same genetic instructions, and in people with inherited genetic diseases, every cell carries the same mistakes. Stem cells as well as the new iPS cells will grow virtually immortal in the lab, and given the right conditions, can be made to form any desired tissue, from heart muscle to brain cells. It is hoped that one day they can be used as tailor-made patches to repair diseased or damaged organs.

The number of lines that will be needed is dependent on factors such as how many ways a person can get a disease. For instance, if there are 50 ways to get type 1 diabetes, scientists will need to develop more stem cell lines. If there are only a couple of ways of acquiring the disease, fewer lines will be necessary.

Although the cells are an alternative to taking embryonic stem cells from a human embryo they do not replace them. The federal government severely restricts embryonic stem cell research because of moral objections. "Even though the iPS methodology gives us a facile way for making disease-specific lines, it does not eliminate the value or need for continuing to study human embryonic stem cells," Daley said. "Those are really the gold standard for pluripotent stem cell types. They have no genetic modifications and, at least for the foreseeable future, and I would argue beyond that, are going to be extremely valuable tools. Human embryonic stem cells allow you to ask questions that we never can ask with iPS cells."

The iPS method also requires the use of viruses, which limits the therapeutic potential of the lines. "Whether or not we're going to be able to figure out how to do it without viruses so we can use the cells therapeutically is, as of today, an unanswered question," Daley said. "I'm confident we're going to get there and that within the next year or two, we will have several strategies for reprogramming cells without viruses, and when that happens, we may have cells in our hands that may be valuable for cell replacement therapy."