Cure SMA Funds 26 Research Projects in 2014

Our strategic research approach includes four areas—basic research, drug discovery, clinical trials, and clinical care—working together toward our vision of a world without SMA. Cure SMA has invested $57 million in research since 1984, with $35 million in the past decade alone. In 2014, we provided funding to 26 different research projects across these four areas.

Our Research Model

Basic Research

Basic research is the first step in developing a treatment and cure for SMA. Basic research projects investigate the biology and cause of SMA in order to identify the most effective strategies for drug discovery. In three decades of basic research funding, we’ve awarded 79 grants for a total of nearly $10 million.

Drug Discovery

Drug discovery converts what we have learned about the causes and biology of SMA into new drug candidates that can be tested in clinical trials. There are now 17 drugs in the SMA drug pipeline, with 6 in clinical trials. Cure SMA has been involved in over half of all SMA drug programs and has invested over $19 million since 2000.

Clinical Trials

Clinical trials test drug candidates for safety and effectiveness. Cure SMA has invested $6.5 million to conduct and prepare for clinical trials. Now, because of our successes, government and industry fund the majority of SMA clinical trials.

We continue to work with clinical partners on trial readiness and recruitment, building on experience and testing protocols we have developed. We also continue to earmark a portion of our research funding for clinical trials, so that we can opportunistically invest where our dollars will be most effective.

Clinical Care

Clinical care research is the fourth prong in Cure SMA’s research. 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. We won’t stop working toward a world without SMA, but until we have a treatment and cure, we’ll do everything we can to improve quality of life for children and families affected by the disease today.

Looking Ahead

Cure SMA will be committing another $1.8 million to new research funding over this year, including approximately $1 million in funding to be announced in the next three months.

Though there’s great promise in the research landscape, there’s also a pressing need for continued and growing investment. With the size of our community and the strength of our connections, we’re able to direct research at unparalleled scale and efficiency. We look forward to announcing our new funded projects, many of which will build on the successes of this year’s funded projects.

2014 Funded Projects

Basic Research

1. Regulation of HDAC5 phosphorylation by Cdk5 in SMA. Yong-Chao Ma, Ph.D., Northwestern University.
2. Motor axon development in SMA. Charlotte Sumner, M.D., Johns Hopkins University.
3. The role of glia cells in SMA. Chien-Ping Ko, Ph.D., University of Southern California.
4. Arginine Methylation as a Regulator of SMN Activities in Motoneurons. Jocelyn Côté, Ph.D., University of Ottawa (with funding from Canada).
5. The role of vehicle coat protein alpha-COP in new models of SMA. Sara Custer, Ph.D., Indiana University.
6. The when and where requirements of SMN in mild SMA. Christine DiDonato, Ph.D., Northwestern University.
7. To Characterize the Role of SMN Protein in Myoblast Fusion. Barrington G. Burnett, PhD at Uniformed Services University of the Health Sciences.
8. Multi-Center Electrophysiological Evaluation of Clinically Relevant Phenotypes in SMA Mouse Models. Laurent Bogdanik, PhD & Cathleen Lutz, PhD at The Jackson Laboratory.
9. Astrocytes and Oxidative Stress in SMA. Allison Ebert, PhD at the Medical College of Wisconsin.
10. Investigate Ubiquitination- Dependent SMN Transport. Ke-Jun Han, PhD at the University of Colorado.
11. The Non-SMN Mediated Benefits of The HDAC Inhibitor Trichostatin A. Rashmi Kothary, PhD at The Ottawa Hospital Research Institute (with funding from Canada).
12. Investigating The P53 Signaling Pathway in Pathogenesis of Mouse Models of SMA. Lyndsay Murray, PhD of the University of Edinburgh.

Drug Discovery

1. Gene Therapy to Dr. Brian Kaspar at Nationwide Children’s Hospital.
2. The Isis Antisense Drug to Dr. Adrian Krainer at Cold Spring Harbor Laboratory.
3. New Antisense Drugs to Dr. Arthur Burghes at OSU and Dr. Christian Lorson.
4. Drug Screens in Motor Neurons to Dr. Lee Rubin at Harvard.
5. Muscle Enhancing Drugs to Dr. Jeff Jasper at Cytokinetics.
6. Novel Small Molecules at to Dr. Peter G. Schultz at CALIBR.

Clinical Trials

1. The SMA Patient Registry to aid in clinical trial recruitment.
2. Stephen J. Kolb MD at OSU for patient recruitment for the NIH NeuroNext Biomarker Trial.
3. Parent Project MD for a collaborative study exploring expectations in clinical trials.
4. Focus Group Project exploring clinical meaningfulness in SMA patient population.

Clinical Care

1. Oscar Mayer, MD at The Children’s Hospital of Philadelphia.
2. Timothy Lotze, MD at Texas Children’s Hospital.
3. Matthew Halanski, MD at University of Wisconsin.
4. Kathryn Swoboda, MD at University of Utah.