Could Pinky the 'Super Mouse' End Alzheimer's?

In the animated series "Pinky and the Brain," Pinky is the witless sidekick who ends up frustrating Brain's plans of world conquest.

But would happen if you took Pinky and swapped some of his brain cells with human ones? As it turns out, what you might get is a really smart mouse.

But that's not all you'd get. You'd get a potential new therapy, capable of curing Alzheimer's, Lou Gehrig's disease. . .even multiple sclerosis.

University of Rochester Medical Center researchers transplanted a particular kind of brain cell, called a glial cell, into young mouse brains.

Glial cells aren't actually the "thinking cells" in the brain—that's the job of neurons—but they do perform important tasks for the cells that are handling processing. They help feed, support, insulate and protect neurons.

As the mice grew up, the population of glial cells multiplied in their brains, eventually numbering into the millions.

They took over duties from native mouse cells, and this made the mice more intelligent in cognitive tests than normal mice. That's probably because human glial cells are different than mouse ones. The human version of this cell type is at least 10 times larger and far more capable of supporting the ability of neurons to connect and communicate. With the help of human glial cells, the super mice passed cognitive tests with flying colors, showing memories that were four times better than those of ordinary mice.

In another experiment, researchers injected human glial cells into mice that had a problem with producing myelin, a protein that acts as an insulating layer around nerves. In function, this is akin to the insulation around a network or phone cable. If the insulation is poor, any signals crossing the link will be degraded. The glial cells transformed into a cell type that produces myelin to compensate for the defect.

These experiments do much more than just satisfy scientific curiosity — they point the way to new therapies.

A number of neurological disorders are associated with problems in the brain's supporting cells.

Multiple sclerosis, for example, is caused when the myelin sheaths around nervous system cells are progressively damaged. Other central nervous system diseases happen when special nervous system support cells die off. This then leads to the death of the cells they support.

Unfortunately, central nervous system (CNS) cells generally won't regenerate as easily as cells in other tissues. When you cut your skin or break a bone, you mend fairly well. Damage to CNS tissue, however, is often for good. There's not much we can do with existing marketed technology. From ALS, also known as Lou Gehrig's disease, to Parkinson's disease to Alzheimer's, we are largely limited to treating symptoms. The same is true for a traumatic spinal cord injury causing paralysis or stroke.

Finding ways to grow new replacement stem cells and then using them to repopulate damaged areas in the brain and nervous system is going to help solve the problem.

That's where Pinky the "super mouse" comes in.

Stem cell research is going to generate a wave of future therapies that will prevent permanent loss of CNS function from injury and disease.

The potential market opportunities for innovators like these are massive.

I'm following two companies right now with promising developments: International Stem Cell Corp. (ISCO:OTCBB) and Neuralstem (CUR:NYSE).

International Stem Cell Corp. owns a patented stem cell technology derived from donor-volunteered unfertilized human egg cells. The FDA has just approved ISCO's stem cell line for human clinical investigation. According to Stephen Petranek, editor of Agora Financial's Breakthrough Technology Alert:

"I am looking forward to an announcement from the company soon that it has submitted an investigational drug application to the FDA to move ahead with its Parkinson's therapy. That will help set up a Phase 1 study. Because of success in animal testing, the company believes that its neural stem cell line can be injected into the midbrain of human Parkinson's patients and the cells will generate dopamine and replace dead cells, mitigating the disease. The company is also pursuing testing of its neural stem cells as a therapy for stroke."

Neuralstem has cracked the code on producing pure lines of therapeutic stem cells for use in nervous system therapeutics. This is an extremely important safety consideration because you have to be confident the stem cells will mature into the right kind of adult cells needed in the tissue where they've been transplanted. The company is pursuing many CNS indications, with clinical trials underway in ALS and spinal cord injury. In 2010, the company initiated the first-ever FDA-approved human clinical trials for ALS neural stem cell transplants. Earlier this year, a 26-year-old woman paralyzed in a motor vehicle accident became the first human to receive an experimental spinal cord neural stem cell graft using Neuralstem's product in a new Phase 1 trial—again, the first trial of its kind ever cleared by the FDA.

Ad lucrum per scientia (toward wealth through science),

Ray Blanco, The Daily Reckoning