The grant to Dr. Ko at the University of Southern California will help us understand exactly where defects in SMA occur and the reasons for observed selectivity at different muscles. The grant to Dr. Monani at Columbia University will help identify new genes that are protective against lowered SMN levels and will lead to new drug targets. Finally the funded projects led by Dr. Kolb at The Ohio State University and Dr. Simard at the University of Manitoba will validate molecular biomarkers that could make future clinical trials more efficient.
Detailed descriptions of each project can be found at www.curesma.org
Our main goal is to accelerate the discovery of an effective therapy and cure for SMA by funding and advancing a comprehensive research program, using a three-pronged approach:
1) Basic Research to reveal the best ways of making SMA drugs,
2) Drug Discovery to make new drugs, and
3) Developing Clinical Trial infrastructure to help test new drugs.
Basic research is a critical component in finding a treatment for SMA. It provides fundamental information about what is going wrong in SMA by telling us when and where SMN protein is needed, and what SMN protein does in different cell types. This knowledge provides seed ideas for new ways of making drugs.
The FSMA basic research program is governed by our Scientific Advisory Board (SAB). The SAB carefully reviews all grant applications to ensure that we fund only the best quality research relevant to our mission.
The newly funded projects include:
Stem Cell Models of SMA: Molecular and Cellular Mechanisms
Christopher Henderson, Ph.D., Columbia University, $160,000 for 2 years.
New Neuromuscular Preparations for In Vivo Evaluations of Drug Efficacy in SMA
Chien-Ping Ko, Ph.D., University of Southern California, $70,000 for 1 year.
Validation of Spinal Muscular Atrophy Biomarkers in VALIANT Subjects
Stephen Kolb, M.D., Ph.D., The Ohio State University, $70,000 for 1 year.
Louise Simard, Ph.D., University of Manitoba, $25,000 for 1 year.
Identification and Characterization of Factors Critical in Regulating the Selective Vulnerability of Distinct Motor Neuron Pools in SMA Model Mice
Rashmi Kothary, Ph.D., Lindsay Murray, Ph.D., University of Ottawa, $120,000 for 2 years.
Exploring novel genetic determinants of disease severity in spinal muscular atrophy model mice.
Umrao Monani, Ph.D., Columbia University, $160,000 for 2 years.
Effects of SMN on mRNA transport and local protein synthesis in motor axons. Wilfried Rossoll, Ph.D., Claudia Fallini, Ph.D., Emory University, $140,000 for 2 years.
Since 2004, the FSMA Scientific Advisory Board has awarded 66 basic research grants for a total of $8,195,096 in funding. This is in additional to our drug discovery and clinical research funding. The grants have been awarded to 47 different principal investigators at 33 different institutions.
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Families of Spinal Muscular Atrophy is dedicated to creating a treatment and cure by:
- Funding and advancing a comprehensive research program
- Supporting SMA families through networking, information and services
- Improving care for all SMA patients