• 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

  • Inaugural ALS Finding A Cure We Care Call for Proposals

    Due date for nominations is September 8, 2021


    ALSFAC-WE-CARE is led by Denis Rizzuto, whose wife Christie had ALS. Christie, a wife, mother, and friend to many, inspired all who knew her with her warmth, her internal and external beauty, and her deep devotion to caring for others. All members of the executive committee of ALSFAC are committed to supporting research to find the cures and to helping those living with ALS today. Over the past months, we met with several remarkable organizations who provide compassionate care to patients and families with ALS. We spoke to many patients and families and learned how much need there is for more care and for more coordination between the institutions that help those with ALS. These initial grants we hope are the first of many from ALSFAC-WE-CARE to ensure that everyone with ALS has the care they need.
    We heard for example of several loan closets and a need for a coordinated effort to link these so individuals with ALS can have what they need, no matter where they live. We also heard of amazing programs to help the children of individuals with ALS and to bring care and resources to the home. These programs are spectacular but often are restricted to limited geographies. There is no shortage of need nor of great groups trying to provide help. We want to make a difference to expand the programs that are working, create new programs to fill unmet needs, and to support efforts for coordinated delivery of key care help and resources.
    Therefore, applications from nonprofit organizations are requested for projects that will help patients and families with ALS and that will also help make these services available more broadly. We will bring the funded organizations together and over time will expand the team and care initiatives so that no person with ALS goes without the care resources they need. ALSFAC believes strongly in collaboration, creativity, open sharing of information, and elimination of duplication in efforts. As noted by the founder of ALSFAC, Mr. Leandro Rizzuto, we are only limited by our imagination.


    Timeline for Submission:
    Applications open: July 26, 2021
    Applications due: September 8, 2021
    Recipients notified: November 2021
    Submit to: https://app.box.com/f/0537c35341164eb6bda403035ed0f8d8


    Grants are for the support of 2-year proposals of up to $200,000 USD total (inclusive of a maximum of 15% in indirect costs). Support will not exceed 2 years and applicants should submit proposals that are compatible with a 2-year time frame. The continuation of funding within this period will be subject to the submission of satisfactory progress reports, which will be required at six-month intervals.

    Selection criteria:

    • Impact of Applicants project to increase and improve care for people with ALS
    • Clear success goals and metrics 
    • The project will address clear gaps in compassionate care 
    • Collaboration with other organizations to improve compassionate care and willingness to think boldly about gaps in care and how to address


    Application Process:

    • Please provide a cover letter addressed to ALS Finding a Cure-WE-CARE including:
      • Proposal Title
      • Total costs (200 K maximum over two years including15% overhead cap)
      • List of all investigators, foundations, and institutions 
      • Name and contact information for person(s) responsible for contracts 
      • Address for mailing of payments
    • Provide a proposal including: 
      • A brief abstract suitable for a media release to the lay public if the application is successful
      • Project Plan, Background, and Timeline (max. 7 pages). Please address the following questions:
        • What is your proposed care proposal for ALS patients?
        • In what way will your project improve the lives of individuals with ALS and their families?
        • What does success look like for your project?
        • How is your proposed care project different from or an expansion of what your organization already provides?
        • How will you collaborate and share knowledge and resources with other organizations to ensure synergies and increased access to compassionate care?
        • Why is ALS important to you and your foundation?
        • What regions will you help people with ALS in and how will you augment what is available in that area for people with ALS
      • Detailed itemized budget and budget justification
        • Description of study milestones, including the costs associated and timeline of specific deliverables  
        • Any funding from other funding sources that relate to the proposed work.
        • Please include information on your annual funding and what percentage goes to patient care and what percentage covers administrative expenses. Please note this grant does not support the awardee sending the funds to other foundations. 
    • Signed and dated investigator CVs or bios


    Please submit questions to info@alsfac.org
    To learn more about ALS Finding a Cure click here

  • The Sean M. Healey & AMG Center for ALS at MGH Lalji Family ALS Award

    Due date for nominations is September 10, 2021

    The Lalji Family ALS Award is a global prize celebrating excellence in research for an individual or team of investigators who are making transformative breakthroughs in the science of repair of neurological function in ALS. Our goal is to identify and recognize therapies and modalities to regain lost function in ALS patients. 

    Prize Criteria:

    The Lalji Family ALS Award is accepting nominations for individual or team of investigators who have made significant progress in the ALS field impacting the repair of neuronal degeneration in ALS. Examples include but are not limited to: 

    • Identification of key modulators of axonal health and degeneration
    • Discoveries to explore changes in axonal transport 
    • Novel methods to increase and improve endogenous neurogenesis 
    • Cell therapies to target and improve neuronal and/or glial regeneration
    • Restoring nerve muscle communication 
    • Immune system inhibition to decrease inflammation and improve neuronal regeneration
    • ·Discovery of intra-cellular mechanisms to enhance repair and regeneration  

    Timeline for Submission:

    • Release Date: July 19, 2021
    • Nomination Due Date: September 10, 2021
    • Notification to successful candidate: Fall 2021
    • Presentation of award: Virtual 32nd International Symposium on ALS/MND December 2021 

    Download Nomination Form here

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

    Award: $40,000 USD 

    To learn more about the Sean M. Healey and AMG Center for ALS click here.

  • The ALS Association Accepting Applications for ALS Clinical Drug Trials

    The ALS Association launched a new grant funding opportunity through the Clinical Trial Awards (CTA) Program. Letters of Intent are due on September 3, 2021 (5 pm US ET).  While there are only 2 weeks left, there are no organizational signatures required for the short Letter of Intent. 

    The CTA program provides support for early-stage interventional clinical trials to discover new treatments and cures for ALS.  Type of clinical trials supported through this program are early to mid-phase (phase 1, phase 2a) exploring safety or biomarkers to justify larger phase 2b studies (such as the Healey platform trial platform). The program is open to industry and academic investigators and includes development of pharmacological treatments, biological therapies or gene therapies. This program will award grants of up to $1,000,000 for 3 years. 

    The ALS Association is building this new program based on a successful path set by earlier funding programs for clinical studies. For example, collaboration with ALS Finding a Cure® for the ALS ACT clinical program led to the selection of and funding for Amylyx’s Centaur trial, a trial which showed positive efficacy on both function and survival. Similar partnerships have resulted in funding for Dr. Stan Appel’s T regulatory cell approach, Dr. Bob Brown's AAV10 gene therapy for SOD1, ALSTDI's CD40L targeting therapy, and Dr. Tim Miller’s TDP-43 imaging tracer efforts.

    Click here for details on The ALS Association's funding programs.

    For questions on this exciting announcement, please reach out to researchgrants@alsa-national.org

  • MGH Translational Clinical Research Fellowship at Pfizer

    Application must be submitted by November 1, 2021 for consideration 

    This fellowship provides a unique opportunity for a board-certified or board-eligible physician scientist trained in Neurology to spend a period of up to two years working with the Pfizer Early Clinical Development (ECD) organization in Cambridge, Massachusetts and performing clinical activities at Massachusetts General Hospital in Boston. The recipient of this fellowship will have an opportunity to work on different projects as a member of the Pfizer Innovation Research Laboratory (PflRe Lab). The mission of the PflRe lab is to deliver on an innovative digital platform to transform clinical development in drug trials. Digital medicine is an industry-leading mobile/digital technology initiative striving to utilize digital continuous monitoring of patient physiology and activity as novel endpoints for disease diagnosis and health state assessment. The fellow will also be trained in translational medicine and pharmaceutical industry drug development in preparation for a future role as an industrial clinical research leader or academic clinical researcher. Prior experience in drug development/translational research is not required. Computational experience including signal processing and machine learning is a plus, but not required. The clinician-scientists are selected by a joint Pfizer- Massachusetts General Hospital Search Committee and employed by the Massachusetts General Hospital. There is a 75% protected time commitment to the Pfizer ECD organization and a 25% commitment to clinical activities at Massachusetts General Hospital within the department of Neurology. The program provides annual salary support plus benefits. The awardee may receive additional support for tuition, travel, and supplies. Appointment begins on July 1st, 2022. 

    MINIMUM ELIGIBILITY REQUIREMENTS 

    • Physician scientist who is or will be a BC/BE Neurologist by July 1, 2022 

    • Fellow or Junior faculty in Neurology 

    • Ability to commit 75% of time to Pfizer and 25% of time to MGH 

    APPLICATION 

    • Letter of interest outlining career goals and why and how the program would advance these career goals (2 pages maximum) 

    • Curriculum Vitae (CV) 

    • Two letters of support 

    • NIH Other Support – current and pending grants 

    • Application must be submitted by November 1, 2021 for consideration 

    PROGRAM START DATE 

    July 1st, 2022 (2-year program) 

    DOWNLOAD APPLICATION FORM HERE

    Questions about the program can be directed towards:

    Andrew J. Cole, M.D. F.R.C.P. Program Director, 

    Massachusetts General Hospital 

    Cole.Andrew@mgh.harvard.edu 

     

    Xuemei Cai, MD 

    Program Director, 

    Pfizer, Inc.

    xuemei.cai@pfizer.com 

     

    Please submit your application as one PDF file to: 

    Erica Ratti 

    Program Coordinator, Massachusetts General Hospital

    eratti1@bwh.harvard.edu 

  • The Merit E. Cudkowicz MGH-Takeda Translational Neuroscience Fellowship

    Application must be submitted by November 1, 2021 for consideration

    This fellowship provides a unique opportunity for a board-certified or board- eligible physician scientist trained in Neurology to spend 50%of their time within the two-year fellowship working with the Takeda Neuroscience Therapeutic Area Unit in Cambridge, Massachusetts, while performing clinical activities at Massachusetts General Hospital / MGB in Boston. The recipient of this fellowship will have an opportunity to work on one of a number of diverse projects (see array below) that constitute a rich pipeline of new molecular entities for neurological diseases. 

    The fellow will also receive formal training in translational medicine, clinical trial design, general pharmaceutical industry drug development and leadership training. Prior experience in drug development/translational research is not required. 

    The clinician-scientists are selected by a joint Takeda-Massachusetts General Hospital / MGB Search Committee and employed by the Massachusetts General Hospital. There is a 50% protected time commitment to the Takeda Neuroscience Fellowship and a 50% commitment to clinical and/ or research activities at Massachusetts General Hospital / MGB within the Department of Neurology. The program provides annual salary support plus benefits. Appointment begins on July 1, 2022.

    MINIMUM ELIGIBILITY REQUIREMENTS

    • Physician scientist who is or will be a BC/BE Neurologist by July 1, 2022 

    • Fellow or Junior Faculty Member in Neurology 

    • Ability to commit 50% of time to Takeda and 50% of time to MGH 

    APPLICATION 

    • Letter of interest outlining career goals and why and how the program would advance these career goals (2 pages maximum) 

    • Curriculum Vitae (CV) 

    • Two letters of support, one to be from primary MGH/MGB mentor 

    • NIH Other Support – current and pending grants 

    • Application must be submitted by November 1, 2021 for consideration. 

    • Applications will be evaluated and acceptance may be offered on a rolling basis 

    PROGRAM START DATE 

    • July 1, 2022 (2-year program) 

    DOWNLOAD APPLICATION FORM HERE

    Please submit your application as one PDF file to: Erica Ratti
    Program Coordinator, Massachusetts General Hospital
    eratti1@bwh.harvard.edu

    QUESTIONS ABOUT THE PROGRAM CAN BE DIRECTED TOWARDS: 

    Andrew J. Cole, M.D., F.R.C.P. 

    Professor of Neurology and Program Director 

    Neurological Clinical Research Fellowships Massachusetts General Hospital 

    Tel 617-726-3311 

    Cole.Andrew@mgh.harvard.edu 

     

    Martin M. Bednar, M.D., Ph.D., FAANS 

    V.P., Neuroscience and Takeda Fellowship Director 

    Takeda Pharmaceuticals International Co. Tel 860-326-4357 

    martin.bednar@takeda.com 

     

    The Merit E. Cudkowicz MGH-Takeda Translational Neuroscience Fellowship honors the qualities of dedication, creativity, team building, relentlessness, and singular focus on developing novel effective therapeutics for serious diseases that Dr. Cudkowicz has personified and modeled throughout her career as a clinician-scientist and Neurologist at MGH.

  • University of British Columbia Seeks Clinician Leader

    UBC partners with ALS Society of British Columbia to accelerate life-changing research 

    Thanks to a gift of $5.3 million from the ALS Society of British Columbia, UBC is establishing an endowed professorship in ALS research.  

    UBC has committed funding through the President’s Academic Excellence Initiative to support the recruitment of a senior ALS clinician-scientist and bring clinical trials to patients with ALS faster.    

    “The partnership between the ALS Society and UBC is an historic leap toward meaningful change for all those living with ALS here in B.C. and beyond,” said Dr. Jon Stoessl, professor and head of UBC’s division of neurology. “Patients are counting on us to improve access to treatment, develop new therapies and move closer to a cure for ALS. Local patient participation in clinical trials will be critical to achieving these bold aims.”    

    For patients with ALS, clinical trials offer hope in the short-term and for future generations. There is a large unmet need in B.C. and globally to bring forward new therapies to patients as soon as possible through clinical trials. The ALS dedicated clinician-scientist will engage the scientific community in ALS research while remaining focused on the immediate needs of ALS patients.    

    “This collaboration with UBC is an important milestone for the ALS Society of British Columbia as we celebrate the society’s 40 year anniversary. PROJECT HOPE opens the door for future world-class research and clinical trials to benefit the patients and caregivers throughout British Columbia. This partnership with UBC is an important first step in bringing cutting-edge therapies to continue the fight for a cure for ALS right here in British Columbia. I would like to thank UBC, the BC Ministry of Health and the ALS Society of BC staff, volunteers, donors and directors for their unwavering commitment to see this dream become a reality.”   – Sheldon Cleaves, President, ALS Society of British Columbia    

    The ALS Society of BC raised $5.3 to establish the endowed ALS research professorship in collaboration with UBC. This support includes $2.3 million raised directly from ALS patients and their community of supporters, and $3 million from the B.C. Ministry of Health. The balance of the faculty member’s salary will be paid annually by UBC through the President’s Academic Excellence Initiative.    

    Recruitment of the clinician-scientist dedicated to ALS will begin this fall.    

    Approximately 400 British Columbians live with ALS, a fatal neurodegenerative disease where patients typically become unable to move, speak, swallow and breathe as the condition progresses.    

    This initiative complements UBC’s carefully developed plan for growth through an unprecedented faculty recruitment effort that will significantly enhance the university’s research capacity and impact.  

    Contact 

    Dr. Jon Stoessl
    Professor & Head, Division of Neurology
    UBC Faculty of Medicine
    Tel: 604-822-7967
    Email: jstoessl@mail.ubc.ca 

     

    www.med.ubc.ca/byline-date-only/ubc-partners-with-als-society-of-british-columbia-to-accelerate-life-changing-research/