• ALS Platform Trial

    NEALS is excited to announce that the Sean M. Healey & AMG Center for ALS at Mass General, in partnership with TackleALS, is committed to establishing the first Platform Trial initiative for ALS and will support the first three treatment approaches. The Northeast ALS Consortium (NEALS) and Berry Consultants will be joining the collaboration for the design and conduct of the ALS Platform Trial. The successful partners will work closely with a Trial Design Team to tailor the arm of the Platform Trial according to the experimental treatment. This is an ongoing effort, and we fully anticipate that in addition to these first three treatments, more treatments will be added to the platform, supported by pharma, foundation partners, federal and other fundraising initiatives.

    To read the original announcement, please click here.

    More information regarding site selection and the final design will be announced shortly. In the meantime, please consider applying! Investigators who submit a proposal that becomes part of the platform trial will act as the PI of that arm.

    To participate in an upcoming informational webinar about the application process please contact Susie Hawkins at slhawkins@mgh.harvard.edu or 617-643-1807. Please contact Susie for queries regarding the application and review process or how to contact the ALS Trial Design Team.

    Timeline for Submission: 

    Application Due Date: Wednesday May 8th, 2019                

    Notification of therapy selection: End of May or early June 2019    

    Click here for CDA template Submit completed CDA form to healeycenterforals@mgh.harvard.edu

    Click here for ALS Platform Trial Therapy Application Form

    Submit completed application form to healeycenterforals@mgh.harvard.edu

  • The Healey Center International Prize for Innovation in ALS

    The Healey Center International Prize for Innovation in ALS is an annual global prize celebrating excellence in research for an individual or team who catalyze exceptional discoveries leading to a transformative advance in therapy development in ALS.

    The last decade has seen unprecedented advances in our understanding of the biology of the disease, an exponential increase in novel targets and innovative approaches to developing therapies. The Healey Center International Prize for Innovation in ALS is an annual global prize celebrating excellence in research for an individual or team who catalyze exceptional discoveries leading to a transformative advance in therapy development in ALS.

    Sean M. Healey, executive chairman of Affiliated Managers Group, Inc. (AMG), joined AMG when it was a start-up in 1995, and together with his founding colleagues built the firm into one of the largest global asset management companies with over $830 billion under management—AMG is NYSE-listed and a component of the S&P 500. Prior to AMG, Mr. Healey began his career as an investment banker in the Mergers and Acquisitions Department at Goldman Sachs.

    Mr. Healey was diagnosed with ALS in May 2018. Following his diagnosis, he took a step back from his long-time role of chairman and CEO at AMG in order to aggressively pursue treatment at the guidance of his doctor, Merit Cudkowicz, MD, MSc, chief of neurology at Massachusetts General Hospital. Together, with his company and friends, he established the Sean M. Healey & AMG Center for ALS at Mass General.

    Prize Criteria

    Investigator or team who made a significant advancement in the ALS field that impacted the development of novel therapies for ALS, including, but not limited to the following:

    • A global, multiple disciplinary collaboration that leads to new insights into the biology of the disease and translates into novel approaches for therapy development
    • A novel outcome measure to improve efficiency in clinical trials in patients with ALS
    • A technology to advance therapy development for ALS
    • A technology to identify novel targets for drug development
    • Tools or targets to stratify the ALS population and improve clinical trial design

    Timeline for Submission

    Release date: May 5, 2019

    Nomination due date: July 9, 2019

    Notification to successful candidate: August 2019

    Presentation of award: At the International Symposium on ALS/MND in Perth, Australia on December 6, 2019

    Make a Nomination

    Download nomination form here

    Submit completed nomination form and supporting documents as a single PDF to healeycenterforals@mgh.harvard.edu

    Award: $50,000 USD and travel reimbursement up to $2,500 USD to attend the International Symposium on ALS/MND

    To visit the Healey Center International Prize for Innovation website, click here.

  • 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.