The abilities to walk, talk and complete other simple tasks are capabilities most of us take for granted. The 1 million Americans living with Parkinson’s disease know the value of those faculties. Today, there are approximately 60,000 additional Parkinson’s diagnoses each year, making it the second-most common neurodegenerative disorder in the United States. It is important to pause and reflect on what we can do to combat this devastating disease so that its prevalence does not double by 2040, as is currently projected.
Parkinson’s is a neurodegenerative disease marked by protein misfolding, and cell dysfunction and loss. The known causes vary from genetic mutations to environmental exposures, with most cases not attributable to one cause. Symptoms, both motor and non-motor, increase in kind and severity over time. A cure has remained elusive; there is no treatment that can slow or stop the disease process. Current available therapies can’t treat all symptoms and can cause debilitating side effects.
In the midst of these challenges, researchers are working tirelessly to develop medicines to treat Parkinson’s and related conditions. About 50 Parkinson’s therapies are in clinical testing worldwide, with many more in the pre-clinical stages. A new report by the Pharmaceutical Research and Manufacturers of America (PhRMA) highlights 37 innovative new medicines either in clinical trials or under review by the U.S. Food and Drug Administration (FDA). These therapies from America’s biopharmaceutical research companies comprise 23 for Parkinson’s disease, 11 for related conditions and three diagnostics.
This quantity of new medicines is significant given they first have to be discovered or created, purified, described, and tested in labs. Of all the compounds in these early stages, only about one in 1,000 is promising enough to be tested in human trials. In totality, developing a new drug — from early discovery through FDA approval — takes well over a decade and has a failure rate of more than 95 percent. As a consequence, each success can cost upwards of $5 billion.
Vital to the work being undertaken to find treatments for Parkinson’s and other complex diseases is the ability to collaborate across industry, academia, non-profit patient advocacy organizations and foundations, and government. Collaboration harnesses the collective strength, resources and ingenuity of stakeholders to accelerate the discovery and development of therapies for these complicated diseases and increase the odds of success.
The Michael J. Fox Foundation for Parkinson’s Research (MJFF) promotes collaboration through varied ongoing approaches: consortia, studies and initiatives. Industry has been a partner of the Foundation’s since inception in numerous capacities, and MJFF has awarded more than $102 million to over 230 unique industry-led projects.
One of the most recent is a study from AstraZeneca researching treatment for a debilitating side effect of levodopa, the gold standard Parkinson’s medication. Known clinically as levodopa-induced dyskinesia (LID), these involuntary, fragmented or jerky movements greatly impact those living with the disease.
LID continues to be one of the most clinically challenging factors in long-term management of patients with Parkinson’s. Most patients develop LID within 10 years of the onset of Parkinson’s. Depending on factors including age, gender, duration of treatment and possibly genetic causes, the incidence of LID among Parkinson’s patients has been shown in studies to be between 30 and 80 per cent.
The MJFF grant will be used toward preclinical study of a novel “nicotinic receptor agonist” that would work with receptors to diminish LID. If this research and subsequent clinical trials prove successful, AstraZeneca hopes to deliver an innovative medicine that will reduce LID, enabling Parkinson’s disease patients to optimize their treatment with levodopa.
Innovative medicines play a vital role in improving the quality and value of our nation’s health care, improving patient outcomes and reducing overall health care costs. The innovations in science and medicine in recent decades have transformed many once deadly diseases into manageable conditions, improving and extending the lives of millions of Americans.
To further this advancement toward cures and better care options, we must continue to support the value of innovation, particularly in currently unmet medical needs like Parkinson’s disease. With millions suffering and $14.4 billion being spent a year on Parkinson’s, the human and economic cost is significant.
As we continue to research and develop new medicines for those living with Parkinson’s, collaboration and partnerships among non-traditional partners are essential to future innovation and meaningful advances in treatment discovery.
Sherer, PhD, is the chief executive officer of The Michael J. Fox Foundation for Parkinson’s Research (MJFF). Dunlop, PhD is vice president and head of Neuroscience for the Innovative Medicines & Early Development area of AstraZeneca Pharmaceuticals, based in Cambridge, MA.