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New Parkinson’s Drug Comes to Market after Early Investment from The Michael J. Fox Foundation

People with Parkinson’s have a new treatment option due in part to funding from The Michael J. Fox Foundation (MJFF). On December 21, 2018, the U.S. Food and Drug Administration approved Inbrija, an inhaled levodopa powder, for “off” episodes, when Parkinson’s symptoms are not well controlled with oral medication. MJFF supported early clinical trials of the treatment, and this is the first regulatory approval of a Parkinson’s therapy directly funded by our Foundation.

“Our strategy of funding high-risk, high-reward projects with a focus on patient impact has paid off,” said MJFF CEO Todd Sherer, PhD. “Today people with Parkinson’s have a new option to manage life with the disease. Because of our Foundation’s investments, many more treatments to manage symptoms and to stop progression are moving closer to pharmacy shelves and patient hands.”

MJFF partially funded Phase I and II trials of Inbrija by biotechnology company Civitas Therapeutics in 2011and 2013 with two grants totaling $1.3 million. Acorda Therapeutics, Inc. acquired Civitas in 2014 and continued the development of Inbrija.

De-risking Investments for Larger Partners

We provide early-stage therapeutic projects with the capital needed to advance. Grantees use our funding to build evidence of safety and efficacy, which can attract larger partners with resources for later-stage testing, regulatory processes and commercialization. Our Foundation directs donor-raised funds to the areas of most pressing patient need: from better symptom management to treatments to slow, stop or even prevent Parkinson’s progression.

“The way The Michael J. Fox Foundation does it is really best in class. The lack of bureaucracy and obstacles encourages emerging companies to seek funding and engagement and ultimately advance their projects,” said Glenn Batchelder, co-founder and former CEO of Civitas Therapeutics and member of the MJFF Board of Directors.

This “de-risking” model has advanced dozens of Parkinson’s therapies. Treatments with potential to slow or stop the disease are marching through clinical trials with new partners after early MJFF funding. Therapies targeting individual symptoms are experiencing similar momentum, and the Food and Drug Administration is currently reviewing another MJFF-funded Parkinson’s treatment for “off” episodes: APL-130277 from Sunovion Pharmaceuticals.

Other efforts from our Foundation speed these studies, as well. Educating the Parkinson’s community on “off” episodes through our communications channels helps potential trial volunteers recognize these fluctuations and seek new options. Our study matching tool Fox Trial Finder helped some participants connect directly with these trials, and we held a meeting with payers (e.g., Medicare/Medicaid and insurance companies) to discuss the impact of Parkinson’s symptoms, including “off” episodes. That knowledge may impact decisions on medication coverage.

New Option for Unmet Patient Need

The newly approved Inbrija, which Acorda expects to become available to patients in the first quarter of 2019, helps quickly alleviate symptoms of tremor, slowness and stiffness. With long-term oral levodopa use and advancing disease, these aspects of the disease can re-emerge between medication doses. These “off” episodes can greatly impact quality of life, bringing uncertainty to one’s days and limiting ability to complete daily tasks.

A 2014 MJFF survey of more than 3,000 people with Parkinson’s disease found that more than 60 percent of respondents were in an “off” state for two or more hours per day and nearly 50 percent said their “off” episodes caused them to avoid or stop activities.

Acorda Therapeutics CEO Ron Cohen notes, “It was clear thanks to the work of MJFF that ‘off’ episodes were a serious symptom for many Parkinson’s patients. Acorda committed to addressing this, and the FDA approval means patients will soon have a new treatment option.”

Acorda and The Michael J. Fox Foundation are collaborating on a number of projects, including a study of how patients, care partners and doctors discuss “off” episodes to identify gaps in communication and thereby optimum care.

This article first appeared on the Michael J Fox Foundation’s Foxfeed Blog

MJFF and Blackfynn Collaborate on Parkinson’s Study to Discover Biomarkers

MJFF and PPMI

The Michael J. Fox Foundation (MJFF) and Blackfynn are joining forces to make optimal use of an initiative that could uncover Parkinson’s disease biomarkers and bring about new therapies.

Called the Parkinson’s Progression Markers Initiative (PPMI), the MJFF-backed effort is an ongoing observational study of more than 1,300 volunteer participants both with and without Parkinson’s to validate biomarkers and, over time, identify disease risk factors. Participants undergo a battery of tests and assessments.

Biomarker discovery is critical in the quest for therapies that can slow or halt Parkinson’s progression. Specifically, it would allow for earlier diagnosis, better disease tracking, and more efficient therapy tests. Current treatments only temporarily ease symptoms.

To glean as much as possible from patient data, Blackfynn will lead the PPMI Advanced Analytics Core, established to analyze biomarker discovery. The firm has a data integration and analysis platform it uses to drive development of therapies, and advance translational research for neurological diseases.

“The goal of the Advanced Analytics Core is to accelerate our analysis of the comprehensive within-subject data collected through PPMI,” Kenneth Marek, PPMI principal investigator, said in a news release.

“By combining this rich, multimodal data with Blackfynn’s platform and data science expertise, we hope to uncover insights into the determinants of [Parkinson’s disease] progression that will lead to new therapies and improved quality of life for patients.”

The Blackfynn infrastructure incorporates a full complement of medical and scientific data to enable swift assessment of data correlations and complex data visualizations. PPMI scientists plan to use the platform to interpret data, develop verifiable hypotheses, and better collaborate with the goal of more targeted translational research.

“Our work with MJFF and the PPMI investigators will maximize the value of this important patient dataset and help drive novel discovery with the potential to lead to the development of effective therapies for patients with PD,” said Amanda Christini, president of Blackfynn.

“Blackfynn will enable a new lens through which discoveries can be made, by looking at all raw and metadata together, in context, to find new patterns that are otherwise obscured when data are in isolation.”

Citing an unwieldiness caused by an abundance of Parkinson’s-related biomarker information, an editorial in the journal The Lancet Neurology heralded the efficiencies of collaborative research. Particularly, it applauded the Accelerating Medicines Partnership for its efforts to find a Parkinson’s cure.

As for the PPMI, five-year results have already shown that clinical trials in patients with early-stage Parkinson’s may benefit from assessing markers of disease progression — namely, changes in the Movement Disorder Society (MDS)-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) and dopamine transporter imaging.

Launched in 2010, PPMI is underway in the United States, Europe, Israel and Australia.

The MJFF is dedicated to finding a cure for Parkinson’s by funding research and ensuring the development of improved therapies for those living with the disease. It has funded more than $800 million in research to date.

The post MJFF and Blackfynn Collaborate on Parkinson’s Study to Discover Biomarkers appeared first on Parkinson’s News Today.

Linked Clinical Trials Initiative Boosts Number of Parkinson’s Clinical Studies

The Linked Clinical Trials (LCT) Initiative, a repurposing program that aims to identify existing medicines which can slow the progression of Parkinson’s disease, has resulted in a growing number of clinical trials. The partnership’s history, purpose and activities are the focus of an article, “The Linked Clinical Trials Initiative (LCT) for Parkinson’s disease,” that recently appeared in the European Journal of Neuroscience.

The International Parkinson’s LCT Initiative was conceived in 2010 by Tom Isaacs, late president and co-founder of the London-based Cure Parkinson’s Trust (CPT), and Richard Wyse, the organization’s current director of research and development.
CPT decided to support preclinical and clinical studies of potential disease-modifying therapies (DMTs) and of medications approved for other illnesses in therapy repurposing trials. Symptomatic treatments were not explored, as they were already being extensively investigated in industry-sponsored studies.

The Committee to Identify Neuroprotective Agents for Parkinson’s program greatly influenced the launch of LCT. Initiated in 2003, the program assessed more than 20 potential Parkinson’s therapies. Several were selected to enter clinical trials, but none met their primary clinical goals.

Yet given the ensuing discovery of new disease mechanisms and therapeutic targets, as well as the growing notion that some of the first events in Parkinson’s development occur outside the brain, it seemed “very reasonable to revisit the concept of drug repurposing in [Parkinson’s],” researchers wrote.
LCT’s initial focus was to gain insight from key opinion leaders on appropriate medications to test for potential therapies in Phase 2 trials of Parkinson’s. If successful, these trials would lead to Phase 3 studies and market possible new therapies.
The goal was to have multiple parallel trials comparing different therapies with a similar protocol. Yet the need to for different placebos and durations of treatment, among other factors, made this impossible.

Patrik Brundin, MD, then working at Sweden’s Lund University, agreed to chair the LCT scientific committee. Isaacs and Wyse approached Brundin because of his contributions to the Parkinson’s field and deep understanding of clinical and basic science. Overall, the scientists involved in LCT felt that studies should look at impaired processes — such as neuroinflammation or energy metabolism — and favor compounds able to affect more than one such process. Also, as there was no DMT for Parkinson’s, the disease was an attractive target for therapy repurposing, an approach that can be significantly time- and cost effective to find new medications.

The effort included eight scientists, including Brundin. Another scientist, Jeffrey Conn, PhD, of Tennessee’s Vanderbilt University Medical Center, was a global expert in blood-brain barrier penetration and pharmacology. The blood-brain barrier is a semipermeable membrane that protects the brain from the outside environment. Penetrating this barrier and reaching the brain is critical in treating neurological diseases.

Three patients, including Isaacs, attended the first LCT meeting in 2012 at the Van Andel Research Institute in Grand Rapids, Michigan. Also present were representatives from the Michael J. Fox Foundation for Parkinson’s Research, the Parkinson’s Foundation, and Parkinson’s UK. Since then, patient participation has increased, providing immediate access to their perspectives and discussions on trial design and continuation, as well as compliance with specific therapies.
The first LCT meeting led

Source: Parkinson’s News Today

Blame it on the Parkinson’s

Music can be therapeutic for Parkinson’s patients – listening, playing or composing. Watch and share the video below composed by PD patient Mitch Faile which reminds us all to Blame it on the Parkinson’s