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Respiratory Drug Focus of New Clinical Trial for Parkinson’s

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Respiratory Drug Focus of New Clinical Trial for Parkinson’s

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Gilda

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Edited on 07/03/2016 at 17:08

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Gilda

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710 comments posted | 16 in the Parkinson's disease Forum

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A medication approved to treat various respiratory diseases and that has demonstrated neuroprotective effects in preclinical studies is the focus of a new clinical trial for Parkinson’s disease.

The trial is the latest to be launched as part of the Linked Clinical Trials (LCT) initiative, a program spearheaded by UK research charity The Cure Parkinson’s Trust in collaboration with Van Andel Research Institute (VARI), which is based in Grand Rapids, Michigan, USA.

“With this trial, we’re moving beyond treating Parkinson’s symptoms. We want to actually slow or stop disease progression,” said Patrik Brundin, M.D., Ph.D., head of LCT’s international scientific committee and director of VARI’s Center for Neurodegenerative Science. “This drug—ambroxol—has performed exceptionally well in preclinical studies for Parkinson’s and is already approved to treat other conditions.”

By focusing on existing medications that have have already passed the rigorous drug approval process and shown promise in preclinical laboratory studies for treating Parkinson’s, LCT aims to significantly reduce the time and cost required to bring new therapies to people with Parkinson’s.

“Linked Clinical Trials has real momentum and provides us with the prospect of having new, potentially breakthrough treatments being accelerated into the clinic within a relatively short period,” said Tom Isaacs, president and co-founder of The Cure Parkinson’s Trust. “This new trial provides real hope that we are on the cusp of something truly life-altering—new therapies that show promise to improve the quality of life for the seven to 10 million people worldwide who have Parkinson’s."

From respiratory remedy to neuroprotection

The trial is based at Royal Free Hospital in London and focuses on ambroxol, which is typically used to treat respiratory conditions. Earlier work conducted by the trial’s principal investigator Anthony Schapira, M.D., D.Sc., demonstrated that ambroxol improves the function of a protein that plays a key role in cellular “trash removal” processes. Recent evidence suggests that impairment of these processes are linked to neurodegenerative disease onset and progression. Additionally, mutations in the gene that encode this protein are considered to be the greatest genetic risk factor for developing Parkinson’s.

“Our preclinical work suggests ambroxol may be an effective Parkinson’s treatment thanks to its ability to correct a dysfunctional protein that is prevalent in people carrying a genetic mutation associated with inherited Parkinson’s cases,” Schapira said. “What is particularly interesting is the potential for ambroxol also to benefit Parkinson’s patients without these genetic mutations.”

A complex problem

There have been few major therapeutic breakthroughs for Parkinson’s in the last 50 years with the exception of levodopa, the current gold standard for drug treatment, and deep brain stimulation, a surgical option. Although these therapies may significantly improve quality of life, they do not slow or stop the progressive brain cell death that is the disease’s hallmark. LCT’s scientific committee, which comprises leading Parkinson’s experts and advocates from around the world, is charged with investigating additional compounds that not only treat symptoms but that may also stop disease progression.

In addition to the ambroxol trial, LCT also supports trials to explore the cholesterol drug simvastatin and the diabetes drug exenatide as potentially disease modifying treatments in Parkinson’s. In the coming months, additional trials focused on other medications, including other diabetes treatments, drugs to remove iron build-up in the brain, and treatments to address mitochondrial function, are expected to launch.

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Edited on 03/03/2016 at 10:37

Very interesting . Have had parkinsons 2 years . cant tolerate levodopa. On really low dose patches, now diagnosed coeliac. Go for research , keen for anything which stomach tolerates to help mobiliy.

Respiratory Drug Focus of New Clinical Trial for Parkinson’s https://www.carenity.co.uk/forum/parkinson/my-library-parkinson/respiratory-drug-focus-of-new-clinical-trial-f-825 2016-03-03 10:31:54

Unregistered member

03/03/2016 at 10:31

Very interesting s

Respiratory Drug Focus of New Clinical Trial for Parkinson’s https://www.carenity.co.uk/forum/parkinson/my-library-parkinson/respiratory-drug-focus-of-new-clinical-trial-f-825 2016-03-03 10:31:55

Barbarapotter

07/03/2016 at 17:08

Barbarapotter

Last activity on 07/10/2024 at 09:08

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My husband has had Parkinson's for 5 years, he is not yet on Levodopa as he keeps putting it off because of side effects, but is on border line now.

I just hope and pray that something is discovered soon before he has to go down that road as so many other people have to. It is so very frustrating watching him change from the person he use to be. He is such a patient tolerant man and must be so so frustrated himself when he can't deal with things like he use to, as so many people have to. I hope and pray there will be an answer for everyone that has to deal with PD sooner rather than later, regaining some quality of life again. It's so very hard when we can't really help our husbands/wives/relations, apart from just being there for them. Just hope 'if' there is a break through, cost won't dictate who or where you live before being prescribed it ( as happened with cancer patients) Keep fighting everyone

Respiratory Drug Focus of New Clinical Trial for Parkinson’s https://www.carenity.co.uk/forum/parkinson/my-library-parkinson/respiratory-drug-focus-of-new-clinical-trial-f-825 2016-03-07 17:08:48

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