Curing Brain Diseases by Growing New Cells?

June 22, 2005 — -- Scientists are getting closer to reaching one of the Holy Grails of medical research -- regenerating brain cells to wipe out a wide range of neurological diseases, including Alzheimer's, Parkinson's and epilepsy.

"We're not there yet," said Dennis Steindler, executive director of the McKnight Brain Institute at the University of Florida and one of the leaders of the global effort.

Steindler's group has reached a milestone that has set off a buzz throughout the world of regenerative medicine. It suggests that some time in the not-too-distant future scientists might be able to remove a few stem cells from the brain of a person suffering from a neurological disease, then produce a bucket full of new brain cells to replace those destroyed by the disease.

And ultimately, the research may point the way toward new drugs that will reset the chemistry in a diseased brain to make it produce the cells it needs to recover.

"We may not have to drill a hole in anybody's head at all," says Steindler. "We may be able to use drugs instead."

He and his colleagues have used chemicals to coax stem cells from the brain of an adult mouse to make other cells that, in turn, make brain cells, or neurons. Stem cells are primitive cells that develop into specialized cells, like neurons.

Blueprints to 'Neuron Factories?'

What distinguishes the Florida researchers is their ability to monitor every change along the way.

They also record the chemical and electrical activity during each change, and that has given them a comprehensive image of exactly where the process starts, how it changes, and the end results.

"We have a fingerprint of what the cell looks like, and what all of its children look like en route to making lots of new brain cells,'' he says. "That has never been done before."

What they have ended up with, he says, are "neuron-generating factories."

At this point, all this action occurs in a petri dish, and while other researchers have created brain cells in the lab, no one else has been able to document every event as thoroughly, thus shedding new light on how the process works.

"It's like an assembly line," says Bjorn Scheffler, a neuroscientist at the university and a member of the team. "We can basically take these cells and freeze them until we need them. Then we thaw them, begin a cell-generating process, and produce a ton of new neurons."

The researchers reported their findings in the June 13 issue of the Proceedings of the National Academy of Sciences.

Optimism Abounds

The research so far involves cells from the brain of a mouse, but Steindler thinks it won't be hard to move from mice to humans.

"I don't think it's going to be difficult at all," he says. Of course, as so often is the case, what works for mice may not work at all for humans, but it's clear that Steindler is having a tough time keeping his optimism in check.

Here's how he was quoted in an Irish publication:

"I'm quite optimistic we will translate this to human therapeutics in the very near future. Because advances in the field of regenerative medicine are occurring so quickly at the moment, it could be anytime. It could be next week, it could be 10 years. I'd like it to be next week."

During my interview he was less specific:

"I hope it is pretty quick."

Other scientists not associated with the research say that if further experiments confirm the findings, it could be very, very important.

"The ability to regenerate the needed cell type and place it in the correct spot would have major impact," says Eric Holland, a neurosurgeon at Memorial Sloan-Kettering Cancer Center in New York, who specializes in the treatment of brain tumors.

Detailed Developments

Where all this will lead is still debatable, but the research stands alone in terms of detail.

Using a powerful microscope, the researchers photographed the stem cells in the petri dish every five minutes for up to 30 hours. They ended up with a time-lapse movie that shows exactly what changed every time a new chemical was tried out on the cells.

Thus they were able to change the course of the development by chemical manipulation, and that's perhaps the most important aspect of all. That suggests that chemicals, not surgery, may be able to correct a diseased mind that is now almost impossible to treat.

It's a pot of gold at the end of a rainbow, and it all could be a mirage. Success would mean so much to so many people that it is almost impossible to hold one's enthusiasm in check. But many promising results in the past have proved disappointing.

There's still no cure for Alzheimer's disease, for example, despite decades of research. The progression of the disease can be slowed with some drugs, but only a little.

If a cure could be found, the financial problems that threaten to cripple Medicare would vanish, and families would no longer have to endure what has become known as "the long goodbye."

Steindler's group is already testing lab animals with neurological diseases to see if they can be coaxed into generating new brain cells to replace those destroyed by the diseases. That would be a major accomplishment, and if it works, "then we will begin to talk about human trials," he says.

It's worth hoping that Steindler and his colleagues are on the right track.

But only time, and much more research, will prove it.

Lee Dye's column appears weekly on ABCNEWS.com. A former science writer for the Los Angeles Times, he now lives in Juneau, Alaska.