Our approach to research is focused on continually expanding the pipeline of potential drugs and therapies for SMA, cultivating new approaches and advancing promising ideas.
Every year, the pipeline gets broader and deeper. We’ve seen enormous growth over the past decade, and we expect that growth to continue in the coming years as more projects approach FDA approval.
2015 Drug Pipeline Update
In 2015, the SMA drug pipeline reached a number of new highs. There are now:
- 18 drug programs in development
- 7 drug programs being tested in clinical trials
- 16 companies investing in SMA drug development
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. As drug development and clinical trials continue to progress, we are focused on developing a diversity of approaches that can treat all different types, ages, and stages of SMA.
In addition, the generosity of our donors allowed us to fund $1.85 million in new research in the 2014-2015 grant year. And we are currently in the process of awarding grants that will fulfill nearly $2 million in new research funding for 2015-2016.
New Research Funding
At this year’s Annual SMA Conference, we announced plans to fund nearly $2 million in new SMA research.
Earlier this year, we opened up requests for proposals (RFP) in both basic research and in clinical care. In November, our Medical Advisory Council met to evaluate the clinical care proposals, and just this month, our Scientific Advisory Board met to evaluate the basic research proposals.
Both the MAC and the SAB evaluated all proposals to see which projects are the most intriguing, which have a well constructed study plan, and which match up with the most pressing unanswered questions about SMA. The best proposals will then receive a grant from Cure SMA.
We also recently opened an RFP for drug discovery research. This RFP will close early next year, at which time our Translational Advisory Council will review the proposals and select the best ones for funding.
Basic research and clinical care research grants will be announced in January 2016. Drug discovery grants will be announced in spring 2016.
In 2014-2015, we awarded $1.85 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 might benefit the SMA community.
- $140,000 to Sara Custer, PhD, at Indiana University
- $140,000 to Francesco Lotti, PhD, at Columbia University
- $140,000 to Mustafa Sahin, MD, PhD, at Boston Children’s Hospital
- $80,000 to Linda Lowes, PT, PhD, at Nationwide Children’s Hospital*
- $140,000 to Chad Heatwole, MD, at the University of Rochester*
- $330,000 to California Institute of Biomedical Research and their collaborators, led by Peter Schultz, PhD
- $445,000 to Brian Kaspar, PhD, at Nationwide Children’s Hospital
- $50,000 to Nilesh Mehta, MD, at Boston Children’s Hospital
- $50,000 to Walter Truong, MD, at Gillette Children’s
- $30,000 to Deborah Boroughs, RN, MSN, at BAYADA Home Health Care
- $50,000 to Rebecca Hurst Davis, MS, RD, CSP, CD, at University of Utah
- $50,000 to Martin Lemay, PhD, at Université du Québec à Montréal
SMA Researcher Meeting
- $200,000 to bring the leading SMA researchers together from around the world
*The grants to Dr. Heatwole and Dr. Lowes, both of which deal with clinical trial outcome measures, were jointly funded by basic research and clinical research grant money.
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.
- Regulation of HDAC5 phosphorylation by Cdk5 in SMA. Yong-Chao Ma, Ph.D., Northwestern University.
- The role of glia cells in SMA. Chien-Ping Ko, Ph.D., University of Southern California.
- Arginine Methylation as a Regulator of SMN Activities in Motoneurons. Jocelyn Côté, Ph.D., University of Ottawa (with funding from Canada).
- The when and where requirements of SMN in mild SMA. Christine DiDonato, Ph.D., Northwestern University.
- To Characterize the Role of SMN Protein in Myoblast Fusion. Barrington G. Burnett, PhD at Uniformed Services University of the Health Sciences.
- Multi-Center Electrophysiological Evaluation of Clinically Relevant Phenotypes in SMA Mouse Models. Laurent Bogdanik, PhD & Cathleen Lutz, PhD at The Jackson Laboratory.
- Astrocytes and Oxidative Stress in SMA. Allison Ebert, PhD at the Medical College of Wisconsin.
- Investigate Ubiquitination- Dependent SMN Transport. Ke-Jun Han, PhD at the University of Colorado.
- The Non-SMN Mediated Benefits of The HDAC Inhibitor Trichostatin A. Rashmi Kothary, PhD at The Ottawa Hospital Research Institute (with funding from Canada).
- Investigating The P53 Signaling Pathway in Pathogenesis of Mouse Models of SMA. Lyndsay Murray, PhD of the University of Edinburgh.
- The Isis Antisense Drug to Dr. Adrian Krainer at Cold Spring Harbor Laboratory.
- The SMA Patient Registry to aid in clinical trial recruitment.
- Parent Project MD for a collaborative study exploring expectations in clinical trials.
- Focus Group Project exploring clinical meaningfulness in SMA patient population.
- Timothy Lotze, MD at Texas Children’s Hospital.
- Matthew Halanski, MD at University of Wisconsin.
We thank the following families and foundations for their generous contributions toward these grants: The Jacob Isaac Rappoport Foundation toward Drs. Custer, Sahin, Lotti, and Kaspar; The Miller McNeil Woodruff Foundation toward Drs. Lowes and Kaspar; The Michael and Chandra Rudd Foundation toward Drs. Kaspar and Schultz; The Spinal Muscular Atrophy Research Team toward Dr. Heatwole; FSMA Canada toward Drs. Lemay, Cote, and Kothary.