10 breakthrough Parkinson’s therapies to watch out for


Author: Michael OkunPublished: 1 October 2015

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From disease-modifying drugs to coffee and marijuana, the landscape for breakthrough therapies in Parkinson’s is varied and exciting according to Michael Okun MD

A breakthrough is defined as a sudden increase in knowledge, improvement in technique, or fundamental advancement in understanding. Often breakthroughs occur when a formidable obstacle is penetrated. Breakthroughs are heralded as major achievements and they pave the road to meaningful progress in any disease.

I have spent my entire professional career focused on Parkinson’s and related diseases. I have been blessed to spend much of my time taking care of patients, and I have evolved to understand the critical need for all those suffering to have access to information on breakthrough therapies. I often emphasise to my patients that breakthrough ideas and therapies in Parkinson’s disease stretch far beyond a single drug or stem cell.

There is, in fact, a broader picture and more exciting portfolio of breakthroughs spanning drugs, cells, vaccines, devices, genetics, care, and behaviour. As such, patients and families with personal investments in Parkinson’s disease should be informed and updated about all of the potential breakthrough therapies.

“Every challenge you encounter in life is a fork in the road. You have the choice to choose which way to go: backward, forward, breakdown or breakthrough.”

– Ifeanyi Enoch Onuoha

Here is a list for patients and families of potential Parkinson’s breakthroughs to watch:

  1. Disease-modifying drugs and biomarkers

These are drugs that may slow disease progression; also imaging and fluid (e.g. blood, spinal fluid) markers that can track the effects of Parkinson’s drugs are needed and under development.

  1. Coffee, tea, exercise, interdisciplinary teams and carers

There is mounting evidence that these approaches have symptomatic and perhaps even other benefits.

  1. Extended-release/novel delivery systems for Parkinson’s disease drug and when to start drug therapy

Parkinson’s desperately needs longer-acting drugs to reduce the ‘number of pills’ burden.

  1. Marijuana and synthetic cannabinoids

The revelation that there are cannabinoid receptors in the brain has surprised some experts, and tickling them may be beneficial to some disease symptoms.

  1. New drugs for hallucinations, sleep, constipation and dizziness

These problems are frustrating for patients and carers, but several new drugs are poised to make a difference.

  1. Therapies while hospitalised and avoiding hospitalisation

We now know that the hospitalisation risk is very high in Parkinson’s disease, and that hospitals can be dangerous places for patients; the focus needs to shift toward avoiding hospitalisations.

  1. Advancing deep brain stimulation technology, earlier intervention and dopamine pumps

The technology has been refining our approach to the symptomatic management of Parkinson’s disease and new therapies are now reaching the bedside.

  1. Stem cells and stem cell tourism

There has been a shift away from the idea that stem cell transplants will cure Parkinson’s disease, however stem cells are proving powerful in drug screening.

  1. Prions, spreading proteins, vaccines and growth factors

Several novel approaches are in trial, and a Parkinson’s vaccine may be on the horizon. 

  1. The drug development pipeline

Understanding the drug development pipeline will help patients and families to gain access to the newest Parkinson’s disease therapies.

One of the most common questions I receive from people with Parkinson’s and their families is, “What will be the next breakthrough therapy?” Because of the complexity of the disease, each person possesses a different combination of bothersome symptoms. This complexity means that the answer to the next breakthrough therapy will vary patient to patient, and it could be that a combination of breakthroughs – for example, in care as well as possibly a new device or drug – is necessary.

Finally, remember the decision to apply new therapies and devices in Parkinson’s and other diseases should always be guided by the ethical principle that all new therapies, especially invasive therapies such as deep brain stimulation, should only be applied to ‘alleviate human suffering.’ Neurosurgeon Dr Kelly Foote and I addressed this topic in a TEDx talk, ‘Your brain controls everything, and we can control your brain.’

Michael’s new book ’10 Breakthrough Therapies for Parkinson’s Disease’ is out now

National Parkinson Foundation

Michael is the national medical director for the National Parkinson Foundation (NPF). The NPF runs the free international Ask the Doctor web-based forum, which helps them keep an accurate pulse on the field. Additionally, Michael’s blog called ‘What’s Hot in Parkinson’s Disease’ and his guest columns as the associate editor for the New England Journal of Medicine’s Journal Watch have provided the material, insight and inspiration for his new book. Collectively, these experiences have provided focus for uncovering the potential breakthroughs to watch.

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