The obstacles to bringing new Parkinsonís disease (PD) drugs to market are many, and they span all stages of the drug development pipeline.†
On average, the time it takes to bring one new drug to market is 15-30 years, with a cost of $1 billion. And this is for those few compounds that actually make it to pharmacy shelves. Most potential treatments die early, unable to secure the necessary support from the pharmaceutical industry to move research from the laboratory to the clinic.
A key part of what we do at The Michael J. Fox Foundation (MJFF) is to find ways to catalyze Parkinsonís disease (PD) research, by placing bets on ideas and therapies that face scientiﬁc or business obstacles. By funding research to overcome these obstacles, MJFF aims to make PD a more attractive investment to both pharmaceutical companies and other funding sources such as venture capitalists and the government.
This week, a team of Foundation staffers led by our CEO, Todd Sherer, PhD, published an article in the journal Movement Disorders, delineating the key obstacles in the field, as well as Foundation strategies for overcoming them.† Below, some salient points:
A major challenge to the development of novel therapies to treat some of the symptoms of PD, and the side effects of Parkinsonís medication, is that no clear regulatory path exists for bringing such drugs to market.† One reason: Dyskinesia and cognition-targeted drugs lack clinical scales to effectively measure progress in development studies.† MJFF has launched two studies to address this reality, in order to establish a clear clinical paradigm for measuring if new drugs are working or not.†
A significant hurdle to the development of a so-called ďcureĒ for PD is the lack of a biomarker to effectively measure biological changes taking place in Parkinsonís patients, as well as to gauge how treatments designed to address these processes are working.† MJFFís landmark biomarker study the Parkinsonís Progression Markers Initiative is addressing this search head-on.† A new partnership with the NIH National Institute of Neurological Disorders and Stroke, called BioFIND, is also dedicated to the search for PD biomarkers. †
Clinical trial recruitment
Clinical trial recruitment is a challenge across all diseases: Thirty percent of all clinical trials fail to recruit a single subject, and 85 percent of trials finish late due to recruitment troubles. PD is no exception, as less than 10 percent of PD patients participate in trials. Slow trial enrollment results in recruitment delays that lead to longer, more expensive trials. Increased trial costs can negatively impact industryís incentive for making PD a strategic priority, and affect future investment in the development of new Parkinsonís treatments.
To get to improved therapies and eventually a cure, more volunteers are needed.† For this reason, MJFF has launched a clinical trial matching tool Fox Trial Finder, to help willing volunteers find the trials that need them, fast.† Since officially launching earlier this year, more than 11,000 have already signed up.†
Finding the money to develop treatments
With that $1 billion price tag, itís clear that no one funding entity has the resources to bring a new drug to market.† This is why MJFF works diligently at brokering relationships between academia, industry, and the government.†
This past fall alone, two Foundation awardees announced critical follow-on funding to forward work into novel Parkinsonís treatments.† Jeff Conn, PhD, of Vanderbilt University, signed a major deal with Bristol-Myers Squibb to develop a symptomatic treatment for PD, and Jim Surmeier, PhD, of Northwestern University was given a multi-million award from the National Institutes of Health for work targeting calcium channel blocker-like drugs as potential disease-modifying treatments for PD.†