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Regenerative Therapies for Spinal Muscular Atrophy (SMA)

The goal of this request for proposals is to promote research with the potential to rapidly transform basic and translational regenerative medicine into treatments for SMA.

  • With support from the Spinal Muscular Atrophy (SMA) Foundation, Stanford’s Innovative Medicines Accelerator (IMA) is seeking proposals from Stanford faculty to address unmet needs in the drug development pipeline for SMA, a devastating neuromuscular disease.
  • SMA is caused by deletion or mutation of the SMN1 gene which leads to reduced levels of SMN protein and degeneration of motor neurons. The SMA Foundation drove the development of three FDA-approved drugs (Spinraza, Zolgensma, Evrysdi) that address the genetic cause of the disease. In its next phase, the Foundation is pursuing regenerative and cell-based therapies that will “rebuild” the nervous system and other relevant tissues such as muscle.
  • The Foundation’s goal is to seed up to ten projects at Stanford with the expectation that three will lead to new therapies and/or companies.

SMA is the perfect prototype and proof-of-concept trial for promising neuromuscular regenerative medicine discoveries.

  • In contrast to other neuromuscular disorders, the underlying cause of the disease has been corrected by SMA-specific drugs that prevent further degeneration in patients.
  • With the recent approval of these efficacious drugs, there is significant interest in regenerative medicine approaches for SMA from pharmaceutical companies, investors, and clinicians.
  • Next generation therapeutics developed for SMA will also be applicable to other indications such as neurodegenerative diseases, muscular dystrophies, cachexia, and ageing.

The RFP provides a unique opportunity to work with an experienced team with a track record for accelerating therapeutics development.

  • Since its inception, the SMA Foundation has funded over $150 million in research and therapeutics development and collaborated with leading academic medical centers (including Stanford), the federal government, contract research organizations, and many large pharmaceutical companies throughout the world.
  • Stanford has a longstanding relationship with SMAF and President Marc Tessier-Lavigne of Stanford University serves as the Scientific Advisory Board chair of SMAF.
  • When the SMA Foundation was established in 2003, there were no treatments for SMA. As a result of the Foundation’s investments and drug discovery programs, today there are three FDA-approved drugs; several more are in advanced preclinical and clinical development.
  • In addition to funding, SMA Foundation can provide its valuable research tools, expertise, and connections to resources for drug development, all of which will help expedite the selected projects’ progress to the clinic and to patients.
  • SMA Foundation can also foster partnerships between funded faculty and CROs or pharmaceutical companies.
Illustration of SMA associated with SMN1 gene mutation
SMA Foundation

Read More About the SMA Treatment Landscape

Please log into your Stanford Box or Stanford Medicine Box account to access the linked materials.

Support Provided: Selected projects will receive a university research award of up to $125,000 for one year. If a project achieves its goal and makes significant progress towards a therapy, robust additional funding and resources for preclinical development may be awarded.

Deadline: All application materials must be received by 5 pm, November 2, 2020.

Eligibility: All Stanford faculty with PI eligibility are welcome to apply.

Application Guidelines: Submit one PDF file containing the following materials in the order indicated below. All documents should be single-spaced, Arial 11-point font with 1-inch margins.

  1. Title page (1 page)
    •  “SMA 2020 Request for Proposals”
    • Project title
    • Investigator(s): Name, department, address, phone number, email address 
    • Application summary (150 words) – Provide a concise statement of the Significance and the Approach of the proposed project. Successful proposals will define a one-year milestone that, if achieved, will justify more intensive drug prototyping efforts.
  2. Proposal (3 pages maximum)
    • Describe the therapeutic hypothesis and its supporting evidence. If a therapeutic target or pathway has already been identified, provide a rationale for its choice. If the goal of the project is to identify a druggable target/pathway, explain how this objective will be achieved. If the goal is to engineer a cellular therapy, describe the characteristics of a drug prototype, and how they will be preclinically verified. Applicants are highly encouraged to engage clinicians and/or patients from the Stanford SMA clinic for input on proposals and throughout the duration of the grant. Please describe how you will incorporate this input in your proposal.
    • References – You may include citations for up to five relevant publications.
  3. NIH-format biosketch for each investigator

Applications should be submitted directly to ChEM-H through the SlideRoom portal. You do not need to submit your applications to your Research Process Manager (RPM) in RMG or through your Office of Sponsored Research (OSR) Contract and Grant officer (CGO) for their approval at this time.

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Selection Process: Proposals will be reviewed by a faculty panel knowledgeable in translational research and evaluated based on the strength and novelty of the therapeutic hypothesis in SMA and the proposed research plan. Preference will be given to projects that incorporate collaborations consisting of interdisciplinary teams across the university and that incorporate input from SMA clinicians and patients. Final selections will be contingent on shortlisted faculty presenting their proposals to the SMA Foundation.

Questions and Contact Info: Elizabeth Ponder, PhD, Director of Planning and Operations, Innovative Medicines Accelerator, eponder@stanford.edu