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Researchers say Parkinson's cure may lie in the human nose

Tuesday June 26, 2012

Laura Ungar

USA Today - University of Louisville researchers hoping to find a cure for Parkinson's disease have discovered an unlikely potential treatment — stem cells from the human nose.

Videos from a laboratory at Louisville reveal the promise: One shows a rat with a brain damaged to mimic Parkinson's continually circling the bottom of a bowl in one direction, unable to do anything else. Another shows a similar rat injected with nasal stem cells moving normally and trying to climb out.

The research — which uses an adult patient's own cells — is outlined in this month's issue of the journal Stem Cells Translational Medicine.

"I think it would be wonderful to have thought of something … that could help people. That's what I'm in this for," said Louisville neuroscientist Fred Roisen, chief science officer and co-founder of a company based on the technology called RhinoCyte.

Parkinson's — which afflicts about a million Americans, including Louisville-born boxing legend Muhammad Ali— is a progressive neurological disorder that mostly strikes people over 50, causing tremors, slow movement and other problems.

It occurs when nerve cells in the brain that produce dopamine, a chemical that helps control muscle movement, are slowly destroyed.

It "is a terrible disease," Roisen said. "And as our population ages, there are gonna be more and more people with Parkinson's."

John Baumann, a 51-year-old Shelby County man diagnosed with it a decade ago, said he finds the research "interesting and coincidental" because his sense of smell was the first thing to go when he began developing Parkinson's. Loss of smell is considered an early warning sign.

"Anything that could lead to a cure is wonderful news," said the lawyer, author and inspirational speaker. "Stem cells have always scared me, since there's so much opportunity for something to go wrong. But when it's done in a moralistic and disease-related way, I'm all for it."

Spurring improvement

Roisen said nasal stem cells are not a cure for Parkinson's but do seem to spur improvement in some research animals. The study says about 35% of rats getting the cells experienced "improved behavioral recovery."

But Scott Whittemore, a stem cell researcher and vice chairman for research in the Department of Neurological Surgery at Louisville, pointed out that about two-thirds of the rats getting nasal stem cells didn't experience recovery.

"This is an intriguing initial study," said Whittemore, who was not involved in the research. "But the success rate needs to be increased before it would be a potentially viable therapeutic option."

There are medicines to treat Parkinson's symptoms, but they don't stop the progression of the disease.

Baumann said he takes medication but still suffers from fatigue and hand tremors and would love to take advantage of a more permanent treatment.

In Roisen's research, he uses a tiny bit of tissue from the olfactory neurosensory epithelium, removed during outpatient surgery from a nickel-sized region high in a nasal passage that's responsible for the sense of smell. The procedure doesn't harm the patient's ability to smell.

Roisen said these cells are unique, regenerating themselves every 30 to 60 days.

"Stem cells are there to replenish lost or damaged cells," he said. "The excitement is, you can use a stem cell to replace damaged or lost cells in the body."

After the tissue is removed, it's grown for six to 10 weeks, after which progenitor cells, or specialized cells with a tendency to turn into neural cells, are isolated and grown.

The cells are then injected during surgery into the brain region affected by Parkinson's. Or, they are frozen for future use.

Roisen said nasal stem cells have advantages over other types of cells -- including the fact that they don't carry the moral and scientific baggage of embryonic stem cells, which are from human embryos and have the potential to cause tumors. And since nasal stem cells come from the patient, no anti-rejection drugs are needed.

Twenty-four weeks after getting the cells, about 35% to 40% of rats with Parkinson-like symptoms demonstrated not only behavioral recovery but also "better mobility and coordination," Roisen wrote in a paper on the research. "In contrast, the animals receiving no (nasal stem cells) or a cellular control group showed no significant improvement."

Human trials in 2014

Roisen said he's excited about the research on nasal stem cells, and is stepping down as chairman of university's Department of Anatomical Sciences and Neurobiology at the end of the month to devote more time to it.

He and his team are also studying the use of nasal stem cells to treat spinal cord injury; they announced results of that research in 2006.

If everything goes well, Roisen said human clinical trials involving people with spinal cord injuries could begin next year, and human clinical trials involving Parkinson's patients could begin in 2014. Roisen said the treatment could become widely available by 2019 or even before.

Steve Gailar, acting chief executive officer of RhinoCyte, said Roisen's research has been funded since 2006 with about $4.5 million from investors, including venture firms and the University of Louisville Foundation.

He said the company is now trying to raise $10 million to take it through early stage clinical trials for spinal cord injury and to enter into the first early-stage human trials for Parkinson's.

Joy Cavagnaro, a consultant to RhinoCyte on regulatory matters, said she's not aware of any other scientists working on using nasal stem cells in this way.

She said it may be easier for Roisen to gain popular support than scientists working with embryonic stem cells, since the nasal cells don't have the same potential for creating tumors and don't raise the ethical and religious issues embryonic cells do.

Whittemore agreed that nasal stem cells have benefits but he said cell therapies in general "are still not the first option for Parkinson's." Symptomatic treatments such as medicines or surgical deep brain stimulation remain higher on the list.

And while the recent study shows potential for nasal stem cells, Whittemore said, "that's still a ways off from being a therapy."