As this report details, 2015 was marked by a groundswell not only of therapeutic pipeline activity, but also of Parkinson’ s community engagement. Thanks to the generosity and commitment of our worldwide network of supporters, we funded $ 87.8 million in research programs last year— a single-year record for our organization. And our investments continue to bear fruit in the form of tremendous activity in the Parkinson’ s therapeutic development pipeline of both symptomatic and potentially disease-modifying treatments.
Today, no fewer than seven disease-modifying therapies( a treatment that could slow, stop or even reverse progression of Parkinson’ s disease, something no current treatment has been proven to do) are advancing through clinical testing.
Genetics remains the leading path to critical breakthroughs in this arena. In 2015, two Foundation-funded investigations targeting alpha-synuclein, the gene whose protein clumps in the brains of people with PD, moved into human trials. One of these already is planning for a Phase II study.( Read more on page 20.) Meanwhile, mutations of the gene known as LRRK2— the most common known genetic cause of PD— remain the focus of intense research interest. We are cautiously optimistic that our partners working toward the first LRRK2 inhibitor drugs could initiate clinical trials as soon as 2017.
On the symptomatic front, as many as four new therapies are positioned to come to market in the next two years, mostly rescue drugs to address disabling motor complications, including“ off” periods. In 2015, two new treatments, Rytary and Duopa— novel reformulations of carbidopa / levodopa designed to reduce“ off” periods— were approved by FDA and took their place on pharmacy shelves. And as this report went to print, we shared news of the U. S. regulatory approval of Nuplazid( pimavanserin), the first treatment for psychosis( hallucinations and delusions) in Parkinson’ s.
We join patients and families in celebrating these important wins. But we remain sober about the work still to be done, and we continue to look to the Parkinson’ s community as vital partners in the continuing advancement of Parkinson’ s therapeutic development as a whole.
As more drugs enter late-stage testing, the need for validated, objective biomarkers intensifies. Biomarkers of Parkinson’ s would make clinical trials more efficient, optimize the selection of patient populations for clinical testing, and clarify regulatory pathways to approval for breakthrough treatments. MJFF’ s landmark Parkinson’ s Progression Markers Initiative( PPMI), launched in 2010, has grown to become the field’ s premier vehicle for biomarker development. Last year, the study expanded its genetic cohorts and extended longitudinal observation of the original cohort. PPMI has studied a total of nearly 1,000 individuals to date, and its unparalleled dataset has been accessed more than 500,000 times by researchers worldwide. Today, studies using PPMI biosamples are yielding never-beforepossible insights into the natural history of PD. For example, in September 2015, leaders of the study’ s genetics core published an analysis of PPMI data outlining five clear criteria that could for the first time allow researchers to screen for Parkinson’ s risk and eventually even predict who will get PD.
As evidenced in part by patients’ and families’ tremendous willingness to take part in PPMI, people with Parkinson’ s have long been eager to engage in a true dialogue with scientists. In 2015, the Foundation launched Fox Insight, an online observational study and portal for patients to share their experiences and concerns directly with researchers, helping ensure that R & D priorities are reflective of the most pressing unmet medical needs of people with PD. Fox Insight aims to collect and analyze data from thousands of individuals on measurable features of Parkinson’ s, such as slowness of movement, tremor and sleep quality, enabling researchers to assemble a better picture of the clinical progression of Parkinson’ s and track its relationship to molecular changes. To date, more than 4,000 participants have enrolled; many more are needed.( Read more on page 46.)
And because virtual research is most effective when it complements traditional“ brick and mortar” studies, our Foundation continues to grow Fox Trial Finder, our online clinical trial matching tool. Last year, Fox Trial Finder crossed the 50,000-mark in registered volunteers, expanded scientifically to include studies focused on atypical parkinsonisms underrepresented in research, and enlarged its international footprint to include France. This brings the total number of countries worldwide in which it is matching volunteers and trials to 31.
In addition to tools for research engagement, our Foundation hears every day from patients,
4 The Michael J. Fox Foundation