Stem Cell Treatment Found Effective For Rare Brain Disorder

sciencehabit writes "Four young boys with a rare, fatal brain condition have made it through a dangerous ordeal. Scientists have safely transplanted human neural stem cells into their brains. Twelve months after the surgeries, the boys have more myelin—a fatty insulating protein that coats nerve fibers and speeds up electric signals between neurons—and show improved brain function, a new study in Science Translational Medicine reports. The preliminary trial paves the way for future research into potential stem cell treatments for the disorder, which overlaps with more common diseases such as Parkinson's disease and multiple sclerosis."

Re:Flowers For Algernon

[ColdWetDog]

These boys were said to have taken immunosuppressants for nine months before beinig injected with the stem cells. Given this, and that the disorder is genetic, I'm assuming the stem cells are from an external source.

From the Fine Linked Article, the stem cells were allogenic - ie, not from the patient.

Since the stem cells are turning into neurons, I wonder how this will affect them in the future. Would the neurons remain without immunosuppressants? Or would the boys slowly lose these foreign cells growing up, and ultimately revert back to their original selves.

Good question, likely they will be on suppressants the rest of their lives.

The nervous sytem is a dangerous thing to manipulate. The effects could range from nothing to the boys taking on traits of their donor. While it's great stem cells can provide relief for this disorder, I hesitate to call it a cure. And if things go south later in their lives, it may very well be a curse.

That's why they are doing this on an invariably fatal disease. They are going to die of this disease (and quite early on IIRC) without treatment. So it is considered a 'compassionate' protocol (not withstanding philosophical discussions on whether or not this really is a compassionate thing to do). So you get to do things that are much more dangerous than your average clinical trial. But this really is the only way to approach it - well, the only way our consistent with current ethical guidelines in the US.

Re:Flowers For Algernon

[reverseengineer]

The stem cells aren't turning into neurons, actually, despite coming from "neuronal stem cells." The intent of the treatment is for the stem cells to differentiate into oligodendrocytes, which are a type of glial cell (which in turn are several types of cells that provide support functions to neurons). Oligodendrocytes are interesting cells because they wrap around neurons like insulation around a wire (which is exactly their purpose). These cells play an important role in nerve conduction and in overall brain function, but they're just tubes filled with fat.

Re:Once again

[reverseengineer]

These happen to only be "adult stem cells" in the sense that they are not totipotent embryonic stem cells (cells from the very earliest stages after fertilization that can differentiate into any cell type), but I'd like to point out that doesn't mean they come from an adult brain. They're only "adult" by the meaning of having matured to the multipotent stage (neuronal stem cells can differentiate into neurons or glia, but not muscle cells or liver cells, for instance). The biotech company that provided these cells, StemCells, Inc. [stemcellsinc.com] cultures them from donated fetal brain tissue.

The first production step comprises a proprietary method for purification of HuCNS-SC cells from donated fetal brain tissue procured from an FDA-registered, not-for-profit agency, in compliance with Good Tissue Practice (GTP) and all other applicable state and federal regulations. As part of the purification process, cells from the tissue are “tagged” with a monoclonal antibody that recognizes human neural stem cells. High-speed Fluorescence Activated Cell Sorting (FACS) is then used to isolate the cells tagged by the monoclonal antibody. The FACS-isolated HuCNS-SC cells are then placed in cell culture.