Cure SMA


925 Busse Rd
Elk Grove Village, IL 60007
800-886-1762
https://registry.fastercures.org/organization-profiles/1005.html?user=10089


   

TRAIN Inventory Profile

Mission
Strategy/Approach
Research Portfolio
Financials
Key Accomplishments
Leadership
Ask Us About
 EXPAND ALL

Mission

Cure SMA leads the way to a world without spinal muscular atrophy, the number one genetic cause of death for infants. We fund and direct comprehensive research that drives breakthroughs in treatment and care, and we provide families the support they need for today. Cure SMA (then known as Families of SMA) was formed in 1984 by Audrey Lewis and a small group of families who joined together so they could fund research toward a treatment and cure for SMA, and find new ways to support each other.

Strategy/Approach

Cure SMA invests aggressively yet strategically in a comprehensive research portfolio. We invest in four different types of research: basic research to understand disease mechanisms, drug discovery to develop preclinical drug assets, clinical research to generate the tools needed to effectively test drug in patients, and care research to improve the quality of life of patients today. Like any investor with a diverse portfolio, we balance risk through this strategy. We also place emphasis on “seed funding” for early-stage research projects, to prove their worth as possible avenues to drug discovery. By doing this, we attract investment from pharmaceutical companies, leveraging our initial investment.

Cure SMA also offers a comprehensive family support and patient care programs—including information packets, care packages, an equipment pool, and the Annual SMA Conference. These programs first and foremost provide families with assistance and hope; however, they also provide incredible incentives to join together and build a strong and unified community. We are then able to offer our community as an extraordinary resource for drug developers, including access to patient data, assistance with clinical trial recruitment, and advocates for regulatory decisions.

Research Portfolio

Cure SMA invests across four research areas, plus additional funding for the annual SMA Researcher Meeting:

  • Basic Research. We fund basic research to investigate the causes and biology of SMA, often revealing new and more effective ways of making drugs. We also use this funding to develop tools that facilitate SMA research. Grants normally range from 1 to 2 years and from $80,000 to $160,000.
  • Drug Discovery. We fund drug discovery to convert basic research ideas into new drug candidates. Grants normally range from 2 to 4 years and from $300,000 to $1,000,000.
  • Clinical Trials. While our pharmaceutical partners fund the majority of clinical trials, we continue to invest where our dollars will be most effective—for example, to develop new or improved clinical trial outcome measures. Grants normally range from 1 to 2 years and from $80,000 to $160,000.
  • Clinical Care. We fund clinical care research to understand the issues that affect daily life for people with SMA, from breathing to nutrition, and to improve their quality of life today. Grants normally range from 1 to 2 years and from $50,000 to $100,000.
  • SMA Researcher Meeting. Each year, a portion of our research dollars are used to support the SMA Researcher Meeting. This is the largest SMA research meeting in the world, bringing together scientists to accelerate the pace of research through the sharing of new data and the building of collaborations. Funding for this program is approximately $300,000 each year.

Financials

Fiscal Year 2014, year ending 6/30/14

  • Revenue: $5,050,694
  • Assets: $3,380,394
  • Grants: $1,004,654
  • Gifts Received: $4,639,671
  • Total Expenditures: $4,835,421

Key Accomplishments

  • More than $59 million invested in our comprehensive research program. 
  • 18 active programs in the SMA drug pipeline, with more than two dozen programs cumulatively
  • Seven drug programs currently being tested in clinical trials.
  • Funded research that identified the genetic cause of SMA, as well as identified an SMA "backup gene" already in the body, which is now the foundation for over half of the active SMA drug programs.
  • Supported the first drug program for SMA from initial research in 2000 all the way to first clinical trials in 2011.
  • 15 biopharmaceutical partners investing in SMA drug development.
  • Founded—and continue to organize and run—both the largest annual conference for SMA families and the largest SMA research meeting in the world.
  • Established 34 local chapters across the US, with over 110,000 community and supporters within those chapters.
  • Annually reach 70% of all newly diagnosed families, with over 3,000 support items sent each year.

Leadership

Cure SMA is governed by a 13-member Board of Directors, plus seven additional committee members. Members of the board are drawn from across the SMA community.

In addition, research and care programs are governed by three different expert panels. The Scientific Advisory Board governs basic research, the Translational Advisory Council governs drug discovery, and the Medical Advisory Council governs patient care. All three scientific/clinical panels are drawn from leaders in both academic and industry research, as well as healthcare professionals across a number of disciplines—neurology, pulmonology, nursing, psychology, occupational and physical therapy, and more—that are relevant to SMA.

Staff

Ask Us About

  • Facilitating patient engagement in the R&D process
  • Maintaining a patient registry or biobank
  • Partnering with industry