Families of SMA is currently funding distinct drug discovery programs, Motor neuron replacement program at California Stem Cell and UCI is one of them. the total investment for these programs would be over $16 Million so far to build a California Stem Cell drug pipeline.
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;
- Educating health professionals and the public about SMA; Enlisting government support for SMA;
- Embracing all touched by SMA in a caring community.
The main vision of SMA families is a world where Spinal Muscular Atrophy is treatable and curable.
California Stem Cell (CSC) is preparing for a final FDA pre-IND meeting to take place in mid 2009. This is a critical step on the track to submitting a formal Investigational New Drug (IND) application to the FDA to begin clinical trials in SMA Type I.
It is the intent of CSC to gain approval to begin FDA-approved clinical trials for the use of these cells in development of a cell replacement therapy for SMA Type I.
Numerous pre-clinical efficacy studies have been completed, demonstrating that the cells work.
The pivotal animal safety study, required to support an application to begin clinical trials, was completed in October 2008.
According to Chris Airriess, Ph.D., Chief Operating Officer,
California Stem Cell, Inc., CSC is now preparing for a final FDA pre-IND meeting to take place in mid 2009, keeping us on track for a formal IND application to begin a Phase I/IIA clinical trial in SMA Type I.
Families of SMA has funded 5 multi-center clinical trials for existing drugs that have potential for SMA.
FSMA has directed and funded the leading new drug development program for a therapy specially designed to treat SMA.
Families of SMA is building a pipeline of drug discovery programs based on our investments in basic research.
FSMA has invested significant resources into alternative approaches that show promise to cure SMA.