• 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.

  • Translational Research Advancing Therapy for ALS (TREAT ALS™) / The Lawrence and Isabel Barnett Drug Development Program - BRIDGE GRANTS

    Deadlines for submission:
    Letter of Intent: May 23, 2017
    Request to submit full application: June 20, 2017
    Submission of full application: September 4, 2017
    Notification of award: November

    Funding commences on receipt of all relevant signatures.

    Instructions for on-line submission.
    Platform open for letter of intent April 22, 2017.

    Step 1:
    Register using this link: http://alsa.spectrumportal.net/Accounts/Register

    Step 2:
    Login using this link http://alsa.spectrumportal.net/Accounts/Logon

    Step 3:
    Submit an abstract for an open call using the relevant link.
    Platform open for letter of intent April 22, 2017. E-mail researchgrants@alsa-national.org for queries.

    Program Description
    There is currently one FDA-approved drug for the treatment of Amyotrophic Lateral Sclerosis (ALS), riluzole (Rilutek), improving survival by two to three months. Other treatments are available that relieve the symptoms associated with ALS and improve the quality of life for patients living with the disease by providing comfort to the patient. However, there is an urgent need for improved therapies. With the recent progress in understanding ALS, the increased effort to develop tools to identify novel treatments for the disease and advances in technology, the opportunity to discover improved treatments for ALS could not be better. The ALS Association’s TREAT ALS™ program funds research from early target identification to preclinical research and early pilot clinical trials. As part of the program, The Association is requesting letters of intent for its drug development contract program, milestone driven research to develop new treatments for ALS. The program compliments the Department of Defense ALS research program and the translational programs at the National Institutes of Health and specifically aims to support studies that will de-risk and incentive industry partners to further pursue interesting targets.

    Contract Information
    This call supports the preclinical assessment of therapeutics for ALS. The proposed studies are expected to be product-driven and focused on therapeutics. It is anticipated that the agents and/or data generated from these awards will lead to the advancement of new therapies for ALS. The program is designed to support preclinical testing and development of therapeutics for ALS. Applications must include preliminary data relevant to the phase(s) of the preclinical development process covered by the proposed research. Applicants must clearly and explicitly articulate what impact the project may have on therapeutic development for ALS. Clinical trials will not be supported with this funding opportunity.

    The contracts are limited to the areas of programmatic interest listed below. Applications must focus on one or more of these areas to be considered for funding. Applications that do not focus on at least one of the following areas will be administratively withdrawn. Preliminary data supporting the choice of target for drug development for ALS must be provided both in the letter of intent and the full application. Priority is given to applications focused on developing compounds directed towards the most attractive targets for ALS with significant data to support the relevance of the chosen target for ALS therapy. Industry partnerships are encouraged.

    • Testing of compounds in mouse models of ALS. (NOTE: Design of mouse model study and approaches to measure target engagement will be critically assessed and relevant details must be included in the letter of intent. Additional studies in human tissue and/or IPS lines are encouraged to strengthen mouse data).
    • Development of pharmacologic agents through Adsorption, Distribution, Metabolism, Excretion, and Toxicity (ADMET) phase; This can be performed by a contract research organization in collaboration with the PI.
    • Screening of compound libraries to identify lead compounds. (This should be for an already established assay).
    • Design and implementation of full-scale, pilot current Good Manufacturing Practice (cGMP) production of therapeutics and/or delivery systems for use in advanced preclinical and initial clinical trials.
    • Industry partners are strongly encouraged.

    The preclinical drug development process will likely require resources beyond those available at a single organization. Therefore, the contracts are open to investigators participating in synergistic collaborations focused testing and developing lead agents for the treatment of ALS. Collaborations should be dedicated to a single, synergistic preclinical development project or study rather than an additive set of subprojects (i.e., the combined efforts of the collaboration must provide greater benefit than the sum of individual research initiatives). If a collaboration is proposed, letters confirming/supporting the collaboration are required. If the collaboration is multi-organizational, participating organizations will ensure the success of the collaboration by resolving potential intellectual and material property issues and by removing organizational barriers that might interfere with achieving high levels of cooperation. Biotechnology or pharmaceutical companies are encouraged to apply. Whether a biotechnology or pharmaceutical company applies for this mechanism as an individual applicant or as part of a collaboration, the company is expected to leverage its own resources to complement the funding provided by this contract.


    • The maximum period of performance is 2 years. Funding will be provided over a shorter time frame where appropriate milestones are reached.
    • The maximum allowable direct and indirect cost (maximum of 10% allowed) for the entire period of performance is $200,000.
    • More cost-effective studies that do not request the full available funding amount are encouraged. The applicant may request the entire maximum funding amount for a project that may have a period of performance less than the maximum 2 years. (time-lines must be clearly described with go/no go milestones).
    • Regardless of the period of performance proposed, the applicant may not exceed the maximum allowable costs.
    • Travel costs of up to $1,500 per year to attend scientific/technical meetings are allowed.
    • Salary for PI will not be supported.

    Payment structure is based on achievement of milestones which will be finalized jointly with the investigators and the scientific advisory board if the study is approved for funding.

    Letter of Intent: Due Date: May 23, 2017

    Review Criteria for letter of intent.

    1. Scientific merit: Priority of therapeutic target as compared with other proposals. Design of mouse study
    2. Research Strategy and Objectives: How the scientific rationale supports the project objectives and feasibility.
    3. Impact: How the project will make an important contribution to ALS therapeutic development.
    4. Personnel: How the qualifications of the PI and key personnel are appropriate to perform the proposed research project.
    5. Consultants and Collaborators: How the overall collaboration will be most effective at achieving milestones and progressing the therapeutic towards clinical trials.
    6. Industry partner will be favorably reviewed.
    7. Timeline for development of therapeutic to be ready for phase I clinical trial.

    Notification of Pre-Application Screening Results: June 20, 2017

    Following the pre-application screening, PIs will be notified as to whether or not they are invited to submit an application; however, they will not receive feedback (e.g., a critique of strengths and weaknesses) on their pre-application.

    Full Application due September 4, 2017

  • 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

  • Clinical Research Training Scholarship in ALS Research

    Application Deadline: October 1, 2017

    The American Brain Foundation and The ALS Association are pleased to support clinical research training in ALS. The AAN Research Program demonstrates the American Academy of Neurology Board of Directors’ dedication to promoting neurology and neuroscience research and training.

    Each award will consist of a commitment of $65,000 per year for two years, plus a $10,000 per year stipend to support education and research-related costs for a total of $150,000. Supplementation of the award with other grants is permissible, but to be eligible to apply for this award, the other grant source(s) cannot exceed $75,000 annually. For additional information regarding this policy, visit aan.com/research-and-awards/aan-research-program/frequently-

    Click here to read more about the scholarship program.