Medication resembling a breath strip could treat Parkinson's disease 'off episodes'

A drug delivered via strip may ease "off episodes.''
Published June 1 2016
Updated June 2 2016

TAMPA — It happened once last year during a rehearsal for The Nutcracker at the Carrollwood Cultural Center.

Helen Michaelson, 66, felt her muscles suddenly get stiff. She became disoriented, too, causing her to slip down a crowded set of stairs and dislocate her shoulder.

"It was a pretty good fall," she recalled.

So-called "off episodes" can be a common occurrence for people like Michaelson who suffer from Parkinson's disease. In between doses of their regular medication, their bodies become rigid, their movements slow and labored.

But a treatment being pioneered in the Tampa Bay region could soon provide relief.

Dr. Robert Hauser, who oversees the Parkinson's Disease and Movement Disorders Center at the University of South Florida, is among three dozen researchers nationally investigating a medication that works like a breath strip and has been shown to quickly control off-episode symptoms.

If their research pans out — and the FDA gives its approval — the treatment could be on the U.S. market as early as 2017, said Anthony Giovinazzo, whose biotech company Cynapsus Therapeutics is driving the effort.

"You have 300,000 or 400,000 people losing between two and six hours of valuable time per day," Giovinazzo said. "It adds up to a lot of hours in the year. We're trying to get some of that time back."

Parkinson's disease is a neurologic disorder that affects nearly 1 million adults in the United States. It develops when the brain produces less dopamine, a chemical that helps the body regulate movement.

Common symptoms include tremor, stiffness and slowness.

People with Parkinson's usually take three or four daily doses of levodopa, an oral medication that is converted to dopamine in the brain. But sometimes, as the disease progresses, the drug becomes less effective, and doesn't last from dose to dose.

"That's when patients may be prone to falls and unable to do their activities," Hauser said. "It can be dangerous, depending on how old someone is or what they are trying to get done."

A shorter-lasting medication called apomorphine can serve as a bridge between doses. But it is only available in an injection.

That's where Hauser's research comes in.

The new treatment Hauser and other researchers are exploring would deliver apomorphine through a thin film not unlike a breath strip that patients would place under the tongue. Earlier studies show it helps control symptoms in most patients.

"It has the potential to be more reliable and to get (to the brain) more quickly," Hauser said, pointing out that, unlike levodopa, it doesn't have to travel through the small intestine.

Hauser says the new treatment could also provide relief to people who wake up experiencing slowness and stiffness, or those needing an "on-demand" treatment overnight.

Hauser recently helped complete a Phase 2 study of the treatment. He is now among the researchers involved in a larger Phase 3 study. The clinical trials are recruiting patients at 35 locations in 18 different states and in Ontario, according to the National Institutes of Health.

Giovinazzo, the Cynapsus CEO, says the project has been moving "extremely fast" because of strong relationships with the Michael J. Fox Foundation for Parkinson's Research and powerful U.S. investors.

"If we can navigate the regulatory waters and get approval for this approach to delivering the drug, it would impact a great number of patients' lives," he said.

Dr. Michael Okun, who chairs the Department of Neurology at the University of Florida and is national medical director for the National Parkinson Foundation, said the strips would give patients "access to a new formulation of medication without the need for an injection."

But Okun, who was not involved in the research, pointed out that the treatment may have some shortcomings.

"The onset time of 24 minutes was longer than what the average Parkinson patient would hope for," he said after reviewing Cynapsus's published data.

Michaelson, who experienced an off episode during The Nutcracker rehearsal last year, hopes the team wins approval quickly. She is a patient of Hauser's, but not a participant in the trial.

Although Michaelson is retired — she was the band director at several Hillsborough County middle schools and education director at the Carrollwood Cultural Center — she continues to volunteer as a community band leader. And off episodes limit her ability to conduct.

Michaelson isn't interested in the injection; she doesn't like the idea of giving herself a shot, she said. But she'd be open to a drug she could put under her tongue.

"It is exciting, believe me," she said.

Contact Kathleen McGrory at [email protected] or (727) 893-8330. Follow @kmcgrory.

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