Originally published on April 24, 2014.
Cure SMA invests in four research areas: Basic Research, Drug Discovery, Clinical Research, and Care Research. Cure SMA has invested $35 Million in research in the past decade alone. Right now we are providing funding for 24 different research projects. Later in 2014, new funding announcements will be made in each of these four areas for over $1.5 Million!
We believe that investment in all of these research areas is essential to finding effective SMA treatments and eventually a cure for the disease. Basic Research into SMA biology tells us what causes the disease. Understanding what causes SMA reveals new and better ways of making new drugs. Then Drug Discovery programs convert Basic Research findings into SMA drug candidates. Clinical Research gives the infrastructure needed to test drug candidates.
Care Research identifies the best care practices to improve the quality of life of SMA patients.
Curent Cure SMA Funding:
Basic Research to Understand SMA:
Today Cure SMA is providing funding for 12 different basic research projects on SMA, with $600,000 in new funding just announced in February of 2014. In mid-2014 we are planning to announce another RFP for additional funding in basic research. With these new grants, we will have invested over $10 Million for basic research since 2004. Our current basic research projects include:
1. To Characterize the Role of SMN Protein in Myoblast Fusion. Barrington G. Burnett, PhD, USUHS
2. The role of vehicle coat protein alpha-COP in models of SMA. Sara Custer, PhD, Indiana University
3. Investigating P53 Signaling Pathways in SMA Mice. Lyndsay Murray, PhD, University of Edinburgh
4. Non-SMN Mediated Benefits of Trichostatin A. Rashmi Kothary, PhD, Ottawa Hospital Research Institute
5. Investigate Ubiquitination-Dependent SMN Transport. Ke-Jun Han, PhD, University of Colorado
6. Astrocytes and Oxidative Stress in SMA. Allison Ebert, PhD, Medical College of Wisconsin
7. Electrophysiological Evaluation of SMA Mice. Laurent Bogdanik, PhD, Cathleen Lutz, PhD, Jackson Lab
8. Motor axon development in SMA. Charlotte Sumner, MD, Johns Hopkins University
9. Regulation of HDAC5 phosphorylation by Cdk5 in SMA. Yong-Chao Ma, PhD, Northwestern University
10. The role of glia cells in SMA. Chien-Ping Ko, PhD, University of Southern California
11. Arginine Methylation as a Regulator of SMN in Motoneurons. Jocelyn Côté, PhD, University of Ottawa
12. The when and where requirements of SMN in mild SMA. Christine DiDonato, PhD, Northwestern University
Drug Discovery to Identify New Therapies:
There are now 15 drugs in the SMA drug pipeline with 5 starting clinical trials in 2014. Cure SMA has been involved in two-thirds of all SMA drug programs and has invested over $19 Million since 2000. Cure SMA believes it is critical to build the SMA drug pipeline using multiple approaches, so there are back-ups if the current lead programs fail, as just 10% of drugs in clinical trials obtain FDA approval. Cure SMA plans a new round of drug discovery funding in 2014 and is currently funding 5 drug discovery programs on:
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. Chris Lorson at the University of Missouri
4. Muscle Enhancing Drugs to Dr. Jeff Jasper at Cytokinetics
5. Novel Small Molecules at to Dr. Peter G. Schultz at CALIBR
Care Research to Improve Quality of Life:
In 2013 Cure SMA initiated a new program in Clinical Care Research. The results of funded projects will build an evidence base for the clinical management of SMA, improving the quality of life for patients. We funded our first round of care research projects in June of 2013 with our second request for proposals to be released in 2014. Our current projects are:
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
Clinical Research to Effectively Test Drugs:
In 2001 Cure SMA started the Project Cure SMA Trial Network. The network developed clinical infrastructure and protocols to test candidate drugs for SMA. Overall they conducted 5 clinical trials. Cure SMA continues to fund collaborative projects to build clinical trial infrastructure and has invested $7 million in this area. Our 2014 clinical research projects include:
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