Families of Spinal Muscular Atrophy Research Funding Leads to the Publication of 17 Journal Articles in 2010. December 16, 2010.
The research goal at Families of Spinal Muscular Atrophy is to accelerate the discovery and development of a treatment and cure for SMA. To help achieve this we fund basic research grants, along with later-stage drug discovery programs and clinical trials. FSMA research funding contributed to the results in 17 published articles in 2010. Please click here for a list of the 2010 publications supported by FSMA funding.
Basic research allows us to solve critical unanswered questions in SMA biology. This information then reveals new and more effective ways of making SMA drugs which helps us build a robust and diverse SMA drug pipeline.
Some examples of the important results from our basic research funding over the past 25 years are:
-Mapping and cloning of the SMA gene, SMN1.
-Identification of the SMN protein and its roles in the cell.
-Discovery of the back-up SMA gene, SMN2 that provides a unique and straightforward approach to developing a treatment.
-Development of animal models to better understand SMA disease pathology and to test SMA drug candidates.
-Determination that HDAC inhibitors can enhance SMN2 gene expression.
-Identification of the nucleic acid sequence being used to correct SMN2 splicing by ISIS Pharmaceuticals.
Specific funding was provided from Families of SMA to the following 19 institutions which published these results in 2010: University of California Irvine, The Ohio State University, Rosalind Franklin University, University of Utah, University of Pennsylvania, CBSquared, University of Ottawa, University of Milan, University of Cologne, Emory University, Northwestern University, Harvard University, Cambria Biosciences, The Ohio State University, and The Project Cure SMA Clinical Trial Network (which included work at University of Utah, University of Wisconsin-Madison Health Sciences, Wayne State University. The Ohio State University, Johns Hopkins Medical School, Ste. Justine Hospital, Northwestern University, University of Manitoba, and CBSquared).
One useful metric to assess whether our basic research funding is accomplishing what it should is the number of peer-reviewed research articles published in scientific journals. Scientific publication of the basic research that we fund indicates that our investments are leading to new and important discoveries about SMA.
In order to fund research at Universities across the world, FSMA has a Research Grant Program that is administered by our Science Advisory Board (SAB). Please click here to read their bios.
There are three main research objectives of this Research Grant Program:
1) Learning basic biological information about SMA to enable more effective therapy development,
2) Generating research tools to enable SMA research, drug development, and clinical trials, and
3) Identifying and then validating new and better ways of making SMA drugs.
The overall goal for our basic research grant program is to identify seed ideas that will form the basis of full-scale industrial drug discovery programs.