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Friday, March 24, 2017

How close are we to a Parkinson’s breakthrough? Three experts have their say

Author: Almaz OhenePublished: 23 March 2017

This year marks 200 years since Dr James Parkinson wrote his essay defining the condition we now know as Parkinson’s disease – but there is still no cure. For this debate piece in our special #UniteForParkinsons series, we spoke to Parkinson’s experts around the world to find out how close we could be to a big leap in the search for better treatment and a cure

A ‘breakthrough’ is defined as a sudden increase in knowledge, improvement in technique, or fundamental advancement in understanding. But what breakthroughs are Parkinson’s researchers and campaigners working towards? And how close are we to achieving those breakthroughs? Here, three world-renowned experts – Dr Michael Okun, Professor Baastian Bloem and Susanna Lindvall – share their thoughts and predictions.

“Breakthrough ideas and therapies stretch far beyond a single drug”

Dr Michael Okun, Adelaide Lackner professor and chairman of neurology at the Univeristy of Florida and medical director at the US-based organisation the Parkinson’s Foundation

“The point that I emphasise with my patients is that breakthrough ideas and therapies in Parkinson’s disease stretch far beyond a single drug or stem cell. There is, in fact, a broader and more exciting picture and portfolio of breakthroughs spanning drug, cell, vaccine, device, genetics, care, and behaviour. Patients and families with personal investments in Parkinson’s disease should be informed and updated about all of the potential breakthrough therapies.

Andy Siderowf and colleagues at the University of Pennsylvania’s National Parkinson Foundation (NPF) Center of Excellence, took another approach to constructing an economic model. The Penn investigators were interested in the ‘economic consequences of slower rates of Parkinson’s progression’. They used a technique called a Markov model and predicted the potential savings associated with slowing Parkinson’s disease progression. Slowing progression by 20% saved the health care system US$60,657 per patient. Stopping disease progression saved US$442,429 per patient.

It is now clear that the weather forecast will be predicting a future storm of Parkinson’s disease cases. This storm will be accompanied by a downpour of large hospital bills and an insurmountable economic burden. The newly diagnosed Parkinson’s disease cases, when added to the existing patients, will have a social and economic impact on all countries and cultures. It will be important that we advocate for a strategic investment from all nations of the world.

One of the most common questions I receive from Parkinson’s disease patients and 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 a breakthrough in care as well as possibly a new device or drug. We will need to expand our notion of breakthroughs, and we will need to harness this knowledge to reduce the burden of Parkinson’s disease.”

“I see a much greater role for informed, engaged patients”

Professor Bastiaan Bloem, MD, PhD, medical director, Parkinson Center Nijmegen

“Breakthroughs in the area of Parkinson’s disease will come from two main directions: the cure and the care. My own research and innovation focus is exclusively on care issues. The major breakthrough that I foresee here is a much greater role for fully informed and engaged patients as equal partners in healthcare, who have easy access to integrated healthcare networks of well-trained professionals who work according to a value-based healthcare concept.

“Care will be delivered largely outside institutions and within the patient’s own home environment, and increasingly via telemedicine approaches. This and other technological developments will help to drive these aforementioned innovations. Implementing this into the western world will take at least 10 more years, and unfortunately, much longer in less developed countries.”

“In the next 10 years I hope we will have effective methods for early diagnosis”

Susanna Lindvall, vice-president of the European Parkinson’s Disease Association (EPDA)

“Until now, Parkinson’s treatments have focused on symptom management in terms of drug-based treatments and surgical treatments such as deep brain stimulation. Now, there are several drugs in the pipeline – such as MSDC-0160, a drug originally created to treat Type-2 diabetes and the Transeuro trial of cell therapy (transplantation).

“In the coming 10 years I hope we will have effective methods for early diagnosis. Right now, researchers have developed a new test that is able to detect abnormal alpha-synuclein in the spinal fluid of people with Parkinson’s with remarkable specificity and sensitivity, at an early stage of the disease. It is early days, but the test is promising. There is also a new neuro-protective strategy where LRRK2 kinase inhibitors are being trialed as therapies for Parkinson’s disease. Researchers have discovered an interaction in neurons that contributes to Parkinson’s disease, and they have shown that drugs currently in development may block the process. The hope is that these will bring major improvements in the quality of life for people with Parkinson’s – life expectancy will improve and the progression of the condition may be slowed or even stopped.”

Michael Okun, MD

Michael is an internationally renowned neurologist and leading researcher. He is currently the Adelaide Lackner professor and chairman of neurology at the University of Florida and is the medical director at the US-based organisation, the Parkinson’s Foundation. He was honoured at the White House in 2015 as a Champion of Change for Parkinson’s disease.

Professor Baastian Bloem

Bas Bloem is a consultant neurologist at the Department of Neurology, Radboud University Nijmegen Medical Centre, the Netherlands. In 1994, he obtained his PhD degree at the Leiden University Medical Centre, the Netherlands. In 2002, he founded and became medical director of the Parkinson Centre Nijmegen (ParC), which was recognised from 2005 onwards as centre of excellence for Parkinson’s disease. Together with Dr Marten Munneke, he also developed ParkinsonNet, where people with Parkinson’s “co-design” their treatment programme in collaboration with a dedicated, multi-disciplinary team– accessed via ParkinsonNet’s online database of expert practitioners.

Susanna Lindvall

Susanna has been the vice-president of the European Parkinson’s Disease Association since 2005. She was born in Romania but has lived in Sweden since 1962, where she graduated as a BSc in organic chemistry at the University of Stockholm. From 1996 to 2002 she was President of the Swedish Parkinson’s Disease Association (SPA). She worked for many years as an information specialist at dairy company Arla Foods until her retirement in 2010.