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Technique could offer relief to multiple sclerosis and leukodystrophy patients

The oligodendrocyte is a special brain cell that plays a vital role as caretaker of the fatty myelin sheaths that surround neurons.  Like insulation on a wire, the myelin ensures signal fidelity; lose the sheath and the signals become distorted or lost.

I. Cracking the Code to Produce Special Brain Cell

While multiple sclerosis is the best-known demyelination disease, a family of childhood diseases known as pediatric leukodystrophies also cause loss of myelin in up to 1,000 children born in the U.S. each year.

Using stem cells created from human skin cells, researchers at the University of Rochester and its affiliated Rochester Medical Center, located in Rochester, New York, were able to successfully cure leukodystrophy in lab mice.  So-called "human induced pluripotent stem cells" (hiPSCs) are a hot field of medical research as their origin from the patient's tissue reduces the risk of rejection and other complications.  

Human skin
hiPSCs can be derived from human skin and have the potential to treat a number of diseases.
[Image Source: Carry Fitness]

First discovered in 2007, the basic idea of hiPSCs is to take human cells -- from the skin or other tissues -- and then expose them to certain chemical signals that cause them to revert to special types of cell types that exhibit pluripotency (the ability to become the kinds of cells found in organs and other body tissues).

In the case of myelin disorders, the goal was to find the right chemicals to reprogram skin cells to become oligodendrocyte progenitor cells (OPCs), the cell class oligodendrocytes derive from.  

Oligodendrocytes are responsible for maintain neurons' insulation -- the myelin sheath.
[Image Source: Knowing Neurons]

After four years the RMC researchers managed to crack the code of how to trigger skin cells to become OPCs and then culture and purify the resulting cells.  Even when the process was complete, it took six months to grow a single batch of the special brain cells.

II. Animal Tests Are a Resounding Success

The next step was to test on an animal subject.  Rodents' brains are similar enough that human OPCs can serve as substitutes for the highly similar rodent cells.  Special mice were bred with leukodystophy to test the treatment potential.

The results were impressive.

Once injected into the brain, the hiPSC-derived OPCs quickly spread throughout the suffering rodent's brain and quickly began to morph into helper cells capable of producing myelin.  And while the factors used to induce pluripotency can raise a tumoregenic risk (risk of getting cancer), the rodents studied showed no tumors.

The treatment worked extremely well on mice. [Image Source: Gawker]

Organ-sourced stem cells proved less effective than the skin-derived OPCs.  Comments neurologist Steven Goldman, M.D., Ph.D., lead author of the study, '"The new population of OPCs and oligodendrocytes was dense, abundant, and complete.  In fact, the re-myelination process appeared more rapid and efficient than with other cell sources."

"This study strongly supports the utility of hiPSCs as a feasible and effective source of cells to treat myelin disorders," he adds.

The research was published in the peer-reviewed journal Cell Stem Cell.

New York State Stem Cell Science (NYSTEM) has agreed to a proposal by the Upstate MS Consortium -- a group of Rochester, Syracuse, and Buffalo researchers and clinicians -- to fund human clinical trials of stem cell derived OPCs on human multiple sclerosis patients.  Dr. Goldman, a key member of the group, says that while the initial study will focus on organ-derived stem cells, he expects hiPSCs to also be added to the mix, given the success.  The first trials are set to begin in 2015.

Sources: University of Rochester, Cell Stem Cell

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Why start with organ derived cells?
By NauticalStrong on 2/8/2013 1:55:29 PM , Rating: 2
If the cells derived from skin work as great as the article states, why are the human trials starting with organ derived cells? Something doesn't add up in my mind (humor intended).

RE: Why start with organ derived cells?
By kyuuketsuki on 2/8/2013 2:14:58 PM , Rating: 3
I'm guessing the research around organ-derived stem cells is simply more mature and/or they are easier to replicate. You did read the part where it took them four years to figure out how to reprogram the skin-cells, and then another six months to culture enough to do the study on mice?

By NauticalStrong on 2/8/2013 2:21:51 PM , Rating: 2
Good points. That makes sense. Thanks.

