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Therapeutic Pipeline Program


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The Michael J. Fox Foundation for Parkinson’s Research (MJFF) seeks to stimulate development of Parkinson’s disease (PD) therapeutics with potential for fundamentally altering disease course and/or significantly improving treatment of symptoms above and beyond current standards of care. Part of our annual Edmond J. Safra Core Programs for PD Research, the Therapeutic Pipeline Program is open to industry and academic investigators working at either pre-clinical or clinical stages of drug and therapeutic development. Proposals must focus on a treatment strategy with clear potential for PD and have a well-defined plan for moving toward clinical utility for patients. Both novel approaches as well as repositioning of approved or clinically safe therapies from other non-PD indications are of interest.

Applicant will be asked to justify the therapeutic targets relevant for PD, provide details of the therapeutic strategy, and clearly identify any suboptimal properties of their therapeutic strategy.  Considerations include:

The target and pathway proposed:

  • What is the relevance of the proposed target for PD?
  • What is the relationship of the proposed target within the pathway in which it is implicated to other potential targets? 
  • Why is the proposed target the most relevant in that pathway – in comparison to other targets in the same pathway?

 The therapeutic strategy:

  • Current drug development stage with clear justification
  • Provide research operation plan, including feasibility, timelines, and all assays to be performed
  • Provide a clear description (strengths and weaknesses) of the properties of the proposed therapeutic




Spring 2014 Review Cycle

  • Informational Conference Call*: September 18, 2013 at 12pm US ET
  • Pre-Proposals Due: October 30, 2013 – 6pm US ET
  • Full Proposal Invitations: November 20,  2013
  • Full Proposals Due (by invite only): January 15, 2014– 6pm US ET
  • Anticipated Award Announcement: March 2014
  • Anticipated Funding: April 2014

Fall 2014 Review Cycle 

  • Informational Conference Call*: March 26, 2014 at 12pm US ET
  • Pre-proposals Due: May 28, 2014 – 6pm US ET
  • Full Proposal Invitations: June 18, 2014
  • Full Proposals Due (by invite only): August 6, 2014 – 6pm US ET
  • Anticipated Award Announcement: October 2014
  • Anticipated Funding: November 2014

*MJFF will hold a 45-minute conference call on the dates and times listed above to clarify and explain the goals of this funding initiative and answer applicant questions.  To participate in the call and receive call-in details, please RSVP via email to



PD is a progressive neurodegenerative disease affecting nearly five million people worldwide, with significant prevalence growth expected due to an aging population.  Current therapies are effective in addressing only the mild-to-moderate motor symptoms of the disease and have significant long-term side effects.  There are few specific drugs available that target the numerous non-motor aspects of the disease or the underlying degenerative process.

The path from research discovery to the creation of a new treatment is long. New ideas about PD cause and progression lead scientists to propose numerous questions to further understand and refine these concepts. But for these ideas to translate into clinical significance for patients, work must move along a critical pipeline from target validation to preclinical drug development and ultimately to human clinical testing. Keeping new ideas flowing along this therapeutic pipeline is paramount to ensuring development of new treatments for people with PD.

MJFF is helping to speed up drug development for PD with innovative strategies, like our annually launched Edmond J. Safra Core Programs for PD Research, which identify roadblocks along the therapeutic development pipeline. By sharing the risk of drug development with academic and industry researchers alike, we hope to increase the movement of new treatments to the clinic.



The Therapeutic Pipeline Program supports PD therapeutic development along the entire preclinical and clinical path. Although MJFF is open to any creative therapeutic strategy, ideal applications should focus on efforts to address one of the following treatment challenges:

  • Strategies that protect or restore degenerating and/or dysfunctional neurons affected in PD.
  • Strategies to alleviate disabling motor and non-motor symptoms of PD. (Important non-motor symptoms include cognitive dysfunction/dementia, depression/mood disorders, autonomic dysfunction and sleep disorders.)
  • Strategies to alleviate complications of PD treatment including dyskinesias and other disabling side effects.

MJFF is also open to alternative strategies, including gene therapy, biological, surgical and non-invasive/non-pharmaceutical approaches that can have significant impact for patients. Moreover, as drug targets may be shared among multiple diseases and/or drugs may hit multiple disease targets. MJFF is also interested in drug repositioning of molecules for PD that have been approved or shown to be clinically safe for other indications.

As many traditional therapies involve pharmacological approaches, proposals focused on small molecules may seek support of key steps along the entire therapeutic pipeline including:

High-throughput screening

  • Primary assay development and validation
  • IC50 generation/validation in a second primary assay and chemistry support for hit ranking and clustering

Hit-to Lead and Lead Optimization

  • Chemistry support
  • In vitro and in vivo PK, PK/PD relationships, toxicity studies

GLP Toxicity

  • GMP scale-up of preclinical candidate
  • Large animal  toxicokinetics


  • Safety, tolerability, and efficacy

For non-pharmacological approaches, MJFF is willing to consider supporting similar stages of therapeutic development relevant to the particular approach.

Applicants are asked to develop a clear plan, including major ‘go/no go’ decision milestones, for moving a therapeutic strategy toward ultimate clinical utility in people with PD.  An emphasis should be placed on providing a detailed pre-clinical package regardless of the stage of development (e.g., a Phase II application should include detailed preclinical data).  Investigators new to PD research are encouraged to collaborate with experienced PD scientists and/or companies to ensure the greatest chance for success.



MJFF will commit up to $10 million to the Edmond J. Safra Core Programs for PD Research with the intention to support multiple awards. There is no set budget limit for proposals and applicants may request up to two years of funding for preclinical development or up to three years of funding for clinical development. No more than 25% (Academic institutions) or 10% (for-profit institutions) of direct costs may go to indirect costs. Please see the program instructions, Administrative Guidelines and our FAQ on MJFF indirect cost policy for details. MJFF reserves the right to reduce the duration and budget based on its review and final funding decision. All funding from MJFF is non-dilutive. To ensure the patient community and MJFF participate in commercial successes of MJFF Awardees, MJFF may request modest repayment of grant funds.


Eligibility Requirements

Applications may be submitted by:

  • U.S. and non-U.S. biotechnology/pharmaceutical companies or other for-profit entities, either publicly or privately held,
  • U.S. and non-U.S. entities, public and private non-profit entities, such as universities, colleges, hospitals, laboratories, units of state and local governments, and eligible agencies of the federal government.

As therapeutic programs may require many kinds of expertise, MJFF encourages industry and academic collaborations when appropriate. Given the significant coordination and leadership necessary for this program, post-doctoral fellows are NOT eligible to apply as PIs.



Please refer to our Administrative Guidelines.Note that any information listed above will supersede information contained in our general MJFF administrative guidelines.


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