• MDA Request for Applications: Drug Development in Neuromuscular Disease

    MDA Venture Philanthropy

    MDA Venture Philanthropy (MVP) is the Muscular Dystrophy Association's drug development program, which operates within MDA's Translational Research program. MVP is exclusively focused on funding the discovery and clinical application of treatments and cures for neuromuscular diseases.

    Adapting elements of the venture capital model, the MVP business plan is characterized by an emphasis on measurable results along with deep involvement by its scientific and industry advisers. MVP evaluates and makes targeted investments in for-profit and not-for-profit companies and academics developing therapeutics for neuromuscular diseases.

    Building upon MDA's long-term investment in research and health care, MVP is designed to complement MDA's ongoing programs of health care, lifesaving services, advocacy, basic and clinical research, and professional and public health information. MVP also benefits from MDA's other research programs that support basic research, clinical trials and research infrastructure.

    Business Plan

    A strategy for lowering barriers to therapeutic development: MVP leverages MDA's years of expertise in research into nerve and muscle biology to create a unique environment to foster translation of research into therapies. Dedicated staff scientists track research developments from MDA's basic research program and from the scientific community as a whole and match the most promising ideas with drug development companies capable of developing such therapies. Where companies have promising therapeutics, MVP helps match the company with disease experts to expedite and streamline development. Where academic projects need more data before they will be attractive to a corporate partner, MVP helps match the researcher with experts in drug development. As a "partner" and not just a funder, MVP will help to cultivate follow-on investors, while its access to patients, experts and research infrastructure can offset some costs of drug development.  

    Bridging the high-risk stages of therapy development: MVP makes targeted investments in projects for which the therapeutic of interest is unlikely to advance via traditional funding avenues due to the perceived risk of a small market, lack of a clear regulatory path or new technology. MVP funds are leveraged to aid getting projects through the critical stages of development to ensure that successful therapies attract funding from other sources and get to market. Co-investors and follow-on funders with complementary skills are cultivated actively in the form of venture capital groups, other nonprofits, and larger biotechnology and pharmaceutical companies. Exit points for MVP funding may include the situations in which 1) a therapeutic progresses to phase III or is approved; or 2) significant funding from follow-on investor(s) is received. Return on investment strategies for MVP may include revenue sharing from licensing agreements or commercialization of the therapeutic, or less commonly, sales of preferred stock.

    MVP uses elements of the venture capital model: MVP uses an iterative evaluation process that reviews the science, management, financials, legal issues and intellectual property of the company or project. MVP holds monthly teleconferences open to its advisers at which decisions to advance projects to the next stage of evaluation are made. In addition to the diverse qualifications of MVP's program staff, advisory committees and ad hoc scientific experts, MVP may utilize well-established outside contractors for formal financial and legal diligence. MVP manages its grants as if they were investments such that funding commitments are structured as milestone-driven contracts.

    A unique blend of staff, contractors and volunteers: MVP has access to a unique set of expertise that it uses to select projects to invest in and to manage those projects. The scientific advisers consist of experts in neuromuscular disease research, neuromuscular medicine, biotechnology venture capital investment and drug development. MVP staff scientists work with these volunteers to select projects with the best possible chance of success, and to act as advisers as those projects move forward. MVP uses its business advisers and outside contractors for legal and financial diligence and for contract negotiation, ensuring that MVP invests only in projects likely to have the greatest possible impact. The business advisers are volunteers with expertise in mergers and acquisitions, biotechnology investing, investment banking and corporate development.

    For information, visit the MDA website: https://www.mda.org/research/mda-venture-philanthropy

    For funding inquiries, contact Grace Pavlath, Ph.D., Senior Vice President & Scientific Program Director at gpavlath@mdausa.org.

  • ALS Center Directorship position available

    ALS Center Directorship position available.  This position is open to both new neuromuscular fellowship graduates and established clinicians.

    The Providence ALS Center is a large ALS Association Certified Center of Excellence, providing services to up to 300 ALS patients.  The center has a well-established multidisciplinary clinic that meets twice a week and participates in numerous clinical research trials. The current director, Kimberly Goslin M.d.Ph.D. is stepping down and transitioning to half time.  Dr. Goslin will provide mentorship to a new neuromuscular fellowship graduate or backup to a more established ALS Clinician.

    Please share this information with neuromuscular specialists at all levels

    Please contact Dr. Goslin if interested for more information:  Kimberly.goslin@providence.org

  • Anne B. Young Fellowship in Therapeutic Development

    Massachusetts General Hospital is pleased to announce that we are accepting applications for a joint MGH/Biogen Idec training program in translational neuroscience. The two-year, clinician scientist program is open to young neurologists who will receive an exceptional training experience for drug development, capitalizing on the expertise of those at Biogen Idec and Mass General. Applications from other institutions are encouraged.

    The objective is to train clinician-scientists to rapidly and efficiently translate advances in neuroscience into treatments for people with neurological disorders. The awardee will learn about the entire life cycle of therapy development from industry and academic perspectives. This includes, but is not limited to, therapy selection, biomarker development, trial design and operations and regulatory science. This didactic, hands-on training program will increase the number of clinical investigators who are well trained and passionate about drug development in the neurosciences.

    Each awardee will develop a research project under the guidance of a mentoring committee that includes at least one Biogen Idec and one Mass General mentor. Awardees are expected to present their work in regular meetings and to publish results of their research in peer-review journals. Awardees will spend time both at Biogen Idec and Mass General.

    The program provides up to 80,000 annual salary support plus fringe benefits. The awardee may receive additional support for course tuition, travel, computer and supplies. Additional support of his/her clinical research project may be available depending on the research project.

    This is a 2 year program.

    Positions Available:

    • 1 in 2018

    Program Start Date:

    • July 1, 2018


    • Physician scientist pursuing a career path in neuroscience translational research.
    • Fellow or junior faculty in Neurology.
    • Identified a mentor at Mass General with whom applicant will develop a research project during the program.
    • Availability of 60-80% effort to dedicate for the fellowship

    Applications must include the following:

    • Anne B. Young, MD, PhD Neuroscience Program application form.
    • Current Curriculum Vitae (CV).
    • Letter of interest outlining career goals, an overview of the proposed project, and how the fellow ship would advance these career goals (2 pages maximum).
    • Letter of support from mentor confirming the availability of mentorship, space and resources.
    • Two additional letters of reference.
    • Other support report - Current and pending grant support information.
    • Non-US Citizens must have a green card or include proof of a valid Visa status.

    Applications Due November 1, 2017

    Candidates will be selected by a joint selection committee comprised of members from both MGH and Biogen.

    Please submit your applications in PDF format by November 1, 2017 to: Jill Cafaro.
    Applications will be treated as confidential documents.
    Questions about the program can be directed towards: Nazem Atassi, MD, MMSc Program Director

    For more information, please visit our MGH website: http://www.massgeneral.org/neurology/education/fellowship.aspx?id=204

    Download Neuroscience Translational Medicine Fellowship Brochure

    Download Anne B. Young Application

  • ALS ACT Request for Proposals: Early phase clinical development of novel, high-potential treatments for people with ALS

    Release Date: 16 October 2017
    Letter of intent due: 10 November 2017
    Please download the LOI form
    Upload LOI and also email it to researchgrants@alsa-national.org
    Link to online LOI submission: http://alsa.spectrumportal.net/IndividualApplicants/Application/0/25
    ALSA ALS ACT Clinical Phase I & II RFP 2017

    Questions about the RFP, ALS ACT, NEALS or The ALS Association are encouraged. Inquiries may be directed to:

    Merit Cudkowicz, MD, MSc
    Director NCRI
    Executive Committee Member NEALS
    Chief Medical Office, ALSFAC

    Lucie Bruijn, PhD MBA
    Chief Scientist, The ALS Association

    ALS Finding a Cure (ALS FAC), the ALS Association (ALSA) and the Northeast ALS Consortium (NEALS) are pleased to announce a call for phase I/II clinical trial applications for novel, high-potential treatments in Amyotrophic Lateral Sclerosis – ALS ACT. The call for clinical study proposals is intended for academic-industry partnerships, including pharmaceutical, biotherapeutic / biotechnology companies, academic members of NEALS, and ALS scientists throughout the world. Up to USD $1,500,000 (up to 10% indirect costs can be included in this amount) in ALS ACT clinical research support is available. Industry partnership applications are strongly encouraged with shared funding proposals.

    Potential early phase clinical trials (Phase I and II) should include, where possible, therapeutic interventions that have:

    • A pharmacodynamics marker that can measure whether pathway of interest is affected
    • A plan to collect samples for biomarker studies
    • A treatment planned in people with ALS

    Applications will be reviewed by an ALS ACT steering committee and will be judged on:

    1. Scientific rationale and merit, novelty, and the value of the project.
    2. Availability of appropriate facilities, regulatory approvals, and the technical ability to carry out the clinical study.
    3. If co-funding is needed, availability of funding must be demonstrated.

    The successful applicant will retain control of the trial as well as intellectual property relating to the therapeutic agent being investigated. Contacting ALSFAC/ALS Association prior to their grant submission is recommended to discuss opportunities and budget for The NCRI Core facilities that can be utilized in the trial. Applicants may request the full $1,500,000 in research support or may request a smaller amount, depending on the appropriate needs of the proposed study. A maximum of 10% indirect costs is allowable and should be included in the $1,500,000. Should applicants require more than the allowed budget for the trial, match funds should already be secured at the time of the application.

    Letter of Intent:
    November 10, 2017

    Notification to submit full application:
    December 1, 2017

    Full Application:
    January 12, 2018

    Recipients Announced:
    March 15, 2018

    Award starts once all necessary paperwork and administrative forms have been signed off by the institution(s) and the funding agencies.

    There is an urgent need for better ALS treatments and therapeutic agents. In the United States, ALS affects one in approximately 40,000 people, with 5,000 new diagnoses each year. There are currently two FDA-approved treatments for ALS, riluzole (Rilutek) and edaravone (Radicava) that modestly change the disease course. The goal of this Request for Proposal is to expedite the process of bringing new treatments forward for testing in people with ALS and to measure if that therapeutic agent is reaching its target.

    About ALS ACT
    ALS ACT is a novel academic-foundation-industry partnership to accelerate treatments for people living with ALS. In partnership with The ALS Association and The ALS Finding a Cure Team, composed of researchers from General Electric (GE) Healthcare and four academic sites, ALS ACT will enact a multi-pronged approach to expediting clinical trials in ALS.

    About The ALS Association
    The ALS Association is the only national non-profit organization fighting Lou Gehrig’s Disease on every front. By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, The Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure. For more information about The ALS Association, visit our website at www.alsa.org

    About ALSFAC
    The sole purpose of the ALS Finding A Cure®, a program of The Leandro P. Rizzuto Foundation, is funding research to find a cure. For more information about ALSFAC, visit our website atwww.alsfindingacure.org

    About NEALS
    The Northeast ALS Consortium (NEALS) is an international, independent, non-profit group of researchers who collaboratively conduct clinical research in Amyotrophic Lateral Sclerosis (ALS) and other motor neuron diseases. NEALS mission is to translate scientific advances into new treatments for people with ALS and motor neuron disease as rapidly as possible. NEALS has over 100 member sites in the United States, Canada, Ireland, and Israel. For more information about NEALS, visit our website at www.neals.org.

    Selection Criteria
    The ALS ACT steering committee will evaluate proposals based on the following criteria:

    1. Significance: Does the proposed study bring forward a new potential therapy for people with ALS? Can the proposed clinical trial be initiated expeditiously? Does the study have a pharmacodynamics marker that can measure whether pathway of interest has been affected? Is there a proposed plan to collect samples for biomarker studies? Applicants should demonstrate that there is sufficient preclinical data (pharmacology and toxicology) to support initiation of the proposed trial.
    2. Approach: Are the conceptual framework, design, methods, and analyses adequately developed, well-integrated, and appropriate to the aims of the study? Have potential problem areas and alternative approaches been considered?
    3. Innovation: Does the clinical trial employ novel concepts, approaches, and/or methods?
    4. Investigator/Sponsor: Is the investigator or sponsor appropriately trained/qualified to carry out the study?
    5. Environment/Collaborative Potential: Does the scientific environment in which the work will be done contribute to the probability of success? Does the proposed study take advantage of useful collaborative arrangements? Will the study utilize resources available through available the clinical trial cores? Partnerships with Industry for partial support are encouraged.
    6. Applicants are not required to but can partner with two NEALS Cores: the MGH Neurological Clinical Research Institute and the Barrows Neurological Institute to access:
    • Project Management
    • Grants & Contracts Management
    • Data Management
    • Study Monitoring
    • Outcome Measure Development and Training
    • Biostatistical Support
    • Site Selection, Start Up, Regulatory Document Review, and Ongoing Site Management
    • Site Trainings: Good Clinical Practice, Regulatory Compliance, and Site Management

    Should you be invited to submit a full application, application forms will be provided and applications must include the following. Applications will be treated as confidential documents.

    1. Research Plan
      Specific Aims
      Background and Significance
      Preliminary Studies
      Research Design and Methods (study design; participant description; data to be collected; plan of analysis)
      Supportive Documentation including a proposed timeline demonstrating that the trial will be conducted expeditiously
      Appendix materials are allowed including material to demonstrate that the treatment is ready for testing in humans. For studies requiring an IND, please indicate the status of the IND application in the Request for Proposal application.
    2. Budget and Ancillary Documents
      Applicants are strongly encouraged to contact ALS FAC prior to submission to discuss budget planning. Lucie Bruijn, Ph.D. MBA; lucie@alsa-national.org, Merit Cudkowicz, MD; mcudkowicz@partners.org
      Budget and budget justification – direct and indirect costs
      Biographical Sketch of key personnel

    Conditions of the Grant

    1. Proprietary Rights
      The Principal Investigator/Sponsor will maintain full control of scientific work and all corresponding responsibilities. A scope of work will be agreed upon by all parties prior to initiation of funding. If necessary, confidentiality agreements will be incorporated.
    2. Reporting
      For all projects, quarterly reports and a final report are required.
    3. Presentation
      Awardees are invited to make a platform or poster presentation at the Annual NEALS Meeting in 2018.
    4. Publications
      All publications, as well as abstracts of presentations at scientific meetings, posters, or any other form of publication that results from a study supported by this award must carry the following acknowledgement: ‘This research was in part supported by a grant from the ALS Association, and ALS Finding a Cure® Foundation’.
    5. Funds
      Funds will be restricted until evidence of IND (or IND exemption) is presented. Additional restrictions will be in place until IRB approval is obtained.