2016 Research Year-in-Review: An Historic Year for Our Community

This has been an historic year for the SMA community. On December 23, the FDA announced its approval of Spinraza, the first-ever approved treatment for SMA. 

In addition to the approval of Spinraza, in 2016 the SMA drug pipeline reached notable highs. There are now:

  • 18 active programs
  • 6 programs in clinical trials
  • 14 pharmaceutical partners

The drug candidates in the pipeline represent a variety of therapeutic approaches, from those that address the genetics of SMA, to those that target the muscles and nerves. Although the first-ever treatment for SMA, SPINRAZA, has been approved by the FDA, we continue to focus on developing diverse approaches that can treat all different types, ages and stages of SMA.

2016-2017 Research Funding

As other SMA drug programs reach new stages of the regulatory process, we remain focused on continued funding for basic research, clinical and regulatory research, and patient care initiatives.

At the 2016 Annual SMA Conference, we announced plans to fund nearly $2.5 million in new research projects for 2016-2017. As the SMA research landscape has developed and the drug pipeline has grown, we recently undertook a systematic review of our research funding priorities. Based on this review, we identified the following priorities:

  • Continued funding for basic research, to develop combination therapies for all types, ages, and stages of SMA.
  • Greater funding for clinical and regulatory research, including initiatives that will bring the patient voice into the process. Many of these projects are being carried out with our industry advisory group: seven different companies collaborating with Cure SMA on regulatory topics, education and clinical trial issues—sharing their data and their ideas to benefit our community.
  • Greater funding for patient care initiatives. As treatments are approved by the FDA, care will become even more important. We have the opportunity to both extend life and ensure the best possible quality of life.

2015-2016 Research Funding

In 2015-2016, we awarded $2 million in new research grants. If you missed any of our funding announcements, catch up on them in our news section using the links below. Each announcement includes a profile of the researcher who is receiving the grant, and explains more about their projects and how they may benefit the SMA community.


Drug Discovery

Clinical and Regulatory Research

  • $100,000 to help fund the launch of a collaborative industry consortium working on clinical trials, regulatory issues, and education

Clinical Care

SMA Researcher Meeting

Other Ongoing Funding

Many of our grants provide multiple years of research support, to ensure that scientists have the time and resources needed to complete their projects. In addition to the grants listed above, in 2015 Cure SMA also provided ongoing funding to the following grant recipients.

  • Transcriptional changes in a NSC-34 model of SMA. Sara Custer, PhD, Indiana University.
  • mTOR and protein synthesis in SMA. Mustafa Sahin, MD, PhD, Boston Children’s Hospital.
  • Role of sumoylation in SMN function and SMA pathology. Francesco Lotti, PhD, Columbia University Medical Center.
  • The development of a clinically relevant outcome measure for SMA therapeutic trials. Chad Heatwole, MD, University of Rochester.
  • Development of an innovative outcome measure to define disease progression in spinal muscular atrophy type 1 for use in the home or clinic. Linda Lowes, PT, PhD, The Research Institute at Nationwide Children’s Hospital.
  • Investigating the P53 signaling pathway in pathogenesis of mouse models of SMA. Lyndsay Murray, PhD, University of Edinburgh 

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